## Protocol Section
### Identification Module
**NCT ID:** NCT02460679

**Acronym:** EPI-589

**Brief Title:** Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)

**Official Title:** A Phase 2A Safety and Biomarker Study of EPI-589 in Subjects With Amyotrophic Lateral Sclerosis

#### Organization Study ID Info
**ID:** EPI589-15-001
#### Organization
**Class:** INDUSTRY
**Full Name:** PTC Therapeutics
### Status Module
#### Completion Date
**Date:** 2018-02-23

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2020-10-14

**Type:** ACTUAL

**Last Update Submit Date:** 2020-09-23

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2018-02-23

**Type:** ACTUAL

#### Results First Post Date
**Date:** 2020-09-23

**Type:** ACTUAL

**Results First Submit Date:** 2020-08-26

**Results First Submit QC Date:** 2020-08-31

#### Start Date
**Date:** 2016-01-14

**Type:** ACTUAL

**Status Verified Date:** 2020-09

#### Study First Post Date
**Date:** 2015-06-02
**Type:** ESTIMATED
**Study First Submit Date:** 2015-05-21
**Study First Submit QC Date:** 2015-05-29
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** PTC Therapeutics

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** This is an open label study with 30-day run in phase to establish baseline parameters, 90-day treatment phase, and a 90-day withdrawal phase to determine long-term effects, duration of treatment response, and potential effects of EPI-589 therapy on known trajectory.

### Conditions Module

**Conditions:**

- Amyotrophic Lateral Sclerosis

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 19

**Type:** ACTUAL

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Participants will receive EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues.

**Intervention Names:**

- Drug: EPI-589

**Label:** EPI-589

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- EPI-589

**Description:** An immediate release film-coated table at a 250 mg dosage strength will be administered per dose and schedule specified in arm.

**Name:** EPI-589

**Other Names:**

- (R)-troloxamide quinone

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.

**Measure:** Number of Participants With Drug-Related Serious Adverse Events (SAEs)

**Time Frame:** Baseline (Day 0) to Month 6

#### Secondary Outcomes

**Measure:** Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12)

**Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3

**Measure:** Maximum Observed Plasma Concentration (Cmax)

**Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3

**Description:** ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task).

**Measure:** Change From Baseline in ALSFRS-R Total Score at Month 6

**Time Frame:** Baseline, Month 6

**Description:** Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation.

**Measure:** Change From Baseline in Vital Capacity at Month 6

**Time Frame:** Baseline, Month 6

**Description:** MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength.

**Measure:** Change From Baseline in MIP at Month 6

**Time Frame:** Baseline, Month 6

**Description:** Respiratory rate is the rate at which breathing occurs.

**Measure:** Change From Baseline in Respiratory Rate at Month 6

**Time Frame:** Baseline, Month 6

**Description:** The heart rate measures the number of times the heart beats per minute.

**Measure:** Change From Baseline in Heart Rate at Month 6

**Time Frame:** Baseline, Month 6

**Description:** SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen.

**Measure:** Change From Baseline in SpO2 at Month 6

**Time Frame:** Baseline, Month 6

**Description:** ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation.

**Measure:** Change From Baseline in ETCO2 at Month 6

**Time Frame:** Baseline, Month 6

**Description:** The failure to thrive as measured by body weight was defined as weight loss of more than 5% from baseline

**Measure:** Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6

**Time Frame:** Baseline, Month 6

**Description:** Participants were observed and timed swallowing water as well as solid foods in accordance with a standardized protocol.

**Measure:** Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6

**Time Frame:** Baseline, Month 6

**Description:** The strength of designated muscle groups was measured using handheld dynamometry.

**Measure:** Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion)

**Time Frame:** Baseline, Month 6

**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.

**Measure:** Change From Baseline in Number of Words Participant Read at Month 6

**Time Frame:** Baseline, Month 6

**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.

**Measure:** Change From Baseline in Time Spent in Reading at Month 6

**Time Frame:** Baseline, Month 6

**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.

**Measure:** Change From Baseline in Number of Words Per Minute Read at Month 6

**Time Frame:** Baseline, Month 6

**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.

**Measure:** Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility

**Time Frame:** Month 6

**Description:** Glutathione lowest limit of quantification (LLOQ) = 0.089 micromoles (uM) and upper limit of quantification (ULOQ) = 13.916 uM in plasma.

**Measure:** Level of Disease-Related Biomarker (Glutathione) in Plasma

**Time Frame:** Baseline up to Month 6

**Description:** Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF.

**Measure:** Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)

**Time Frame:** Baseline up to Month 3

**Description:** Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine.

**Measure:** Level of Disease-Related Biomarker (Glutathione) in Urine

**Time Frame:** Baseline up to Month 6

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Diagnosis of possible, probable, laboratory supported probable, or definite ALS by E1 Escorial Criteria
* Forced vital capacity (FVC) ≥ 70% of predicted
* Weakness onset within 3 years
* Agreement to use contraception if within reproductive years
* Willingness and ability to comply with study procedures
* Stable regimen of dietary supplements and /or riluzole for at least 30 days prior to enrollment
* Abstention from use of other investigative or non-approved drugs
* Participants must be able to swallow 0.375 \* 0.700 inch tablets

Exclusion Criteria:

* Allergy to EPI-589
* Use of ventilation
* Participation in other intervention studies
* Diagnosis of any other neurologic disease
* Malignancy within the past 2 years
* History of stroke
* History of brain surgery
* Hepatic insufficiency with liver function tests (LFTs) greater than 3 times upper limit of normal (ULN)
* Renal insufficiency requiring dialysis
* End stage cardiac failure
* Participation in a trial of a device, drug, or other therapy for ALS within 3 months of screening or during the trial

**Maximum Age:** 70 Years

**Minimum Age:** 21 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Los Angeles

**Country:** United States

**Facility:** Cedar's Sinai

**State:** California

**Zip:** 90048

**Location 2:**

**City:** San Francisco

**Country:** United States

**Facility:** California Pacific Medical Center

**State:** California

**Zip:** 94115

**Location 3:**

**City:** Portland

**Country:** United States

**Facility:** Providence Brain and Spine Institute ALS Center

**State:** Oregon

**Zip:** 97213

#### Overall Officials

**Official 1:**

**Affiliation:** PTC Therapeutics

**Name:** Matthew B Klein, MD, FACS

**Role:** STUDY_DIRECTOR

## Document Section

### Large Document Module

#### Large Docs

- Date: 2017-02-17

- Filename: Prot_000.pdf

- Has ICF: False

- Has Protocol: True

- Has SAP: False

- Label: Study Protocol

- Size: 586189

- Type Abbrev: Prot

- Upload Date: 2020-08-31T16:28

- Date: 2018-03-20

- Filename: SAP_002.pdf

- Has ICF: False

- Has Protocol: False

- Has SAP: True

- Label: Statistical Analysis Plan

- Size: 650327

- Type Abbrev: SAP

- Upload Date: 2020-09-23T08:11

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000010335

- Term: Pathologic Processes

- ID: D000019636

- Term: Neurodegenerative Diseases

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000009468

- Term: Neuromuscular Diseases

- ID: D000013118

- Term: Spinal Cord Diseases

- ID: D000002493

- Term: Central Nervous System Diseases

- ID: D000057177

- Term: TDP-43 Proteinopathies

- ID: D000057165

- Term: Proteostasis Deficiencies

- ID: D000008659

- Term: Metabolic Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M15415

- Name: Sclerosis

- Relevance: HIGH

- As Found: Sclerosis

- ID: M18879

- Name: Motor Neuron Disease

- Relevance: HIGH

- As Found: Lateral Sclerosis

- ID: M4024

- Name: Amyotrophic Lateral Sclerosis

- Relevance: HIGH

- As Found: Amyotrophic Lateral Sclerosis

- ID: M21558

- Name: Neurodegenerative Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12411

- Name: Neuromuscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M15915

- Name: Spinal Cord Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M5742

- Name: Central Nervous System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M28759

- Name: TDP-43 Proteinopathies

- Relevance: LOW

- As Found: Unknown

- ID: M28747

- Name: Proteostasis Deficiencies

- Relevance: LOW

- As Found: Unknown

- ID: M11639

- Name: Metabolic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T349

- Name: Amyotrophic Lateral Sclerosis

- Relevance: HIGH

- As Found: Amyotrophic Lateral Sclerosis

- ID: T4699

- Name: Primary Lateral Sclerosis

- Relevance: HIGH

- As Found: Lateral Sclerosis

### Condition Browse Module - Meshes

- ID: D000016472

- Term: Motor Neuron Disease

- ID: D000000690

- Term: Amyotrophic Lateral Sclerosis

- ID: D000012598

- Term: Sclerosis

### Misc Info Module

- Version Holder: 2024-05-24

## Results Section

### Adverse Events Module

**Description:** Safety population included any participant who received at least 1 dose of EPI-589.

#### Event Groups

**Group ID:** EG000

**Title:** EPI-589

**Deaths Num At Risk:** 19

**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.

**ID:** EG000

**Other Num Affected:** 15

**Other Num at Risk:** 19

**Serious Number At Risk:** 19

**Title:** EPI-589

**Frequency Threshold:** 0

#### Other Events

**Term:** Aortic valve incompetence

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Cardiac disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Vertigo

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Ear and labyrinth disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Eye pain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Eye disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Vision blurred

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Eye disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Abdominal pain lower

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Abdominal pain upper

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Constipation

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Diarrhoea

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Nausea

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Salivary hypersecretion

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Vomiting

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Asthenia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Chest pain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Chills

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Fatigue

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Hypersensitivity

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Immune system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Seasonal allergy

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Immune system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Clostridium difficile infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 15.0

**Term:** Nasopharyngitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 15.0

**Term:** Urinary tract infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 15.0

**Term:** Contusion

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 15.0

**Term:** Fall

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 15.0

**Term:** Post lumbar puncture syndrome

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 15.0

**Term:** Upper limb fracture

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 15.0

**Term:** Alanine aminotransferase increased

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 15.0

**Term:** Aspartate aminotransferase increased

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 15.0

**Term:** Blood pressure increased

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 15.0

**Term:** Blood testosterone decreased

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 15.0

**Term:** Decreased appetite

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Back pain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Muscle spasms

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Muscular weakness

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Musculoskeletal pain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Pain in extremity

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Benign hepatic neoplasm

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)

**Source Vocabulary:** MedDRA 15.0

**Term:** Aura

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Balance disorder

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Dizziness

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Dysarthria

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Headache

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Hypoaesthesia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Hypokinesia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Paraesthesia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Tension headache

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Depression

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Psychiatric disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Disorientation

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Psychiatric disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Dyspnoea

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Nasal congestion

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Oropharyngeal pain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Productive cough

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 15.0

**Term:** Acne

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 15.0

**Time Frame:** Baseline (Day 0) to Month 6

## Results Section - Baseline Characteristics Module

### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Units:** Participants

### Group

**ID:** BG000

**Title:** EPI-589

**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 8.25

**Value:** 55.9

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 5

**Category Title:** Female

#### Measurement

**Group ID:** BG000

**Value:** 14

**Category Title:** Male

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 2

**Category Title:** Hispanic or Latino

#### Measurement

**Group ID:** BG000

**Value:** 17

**Category Title:** Not Hispanic or Latino

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** Unknown or Not Reported

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** American Indian or Alaska Native

#### Measurement

**Group ID:** BG000

**Value:** 1

**Category Title:** Asian

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** Native Hawaiian or Other Pacific Islander

#### Measurement

**Group ID:** BG000

**Value:** 1

**Category Title:** Black or African American

#### Measurement

**Group ID:** BG000

**Value:** 17

**Category Title:** White

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** More than one race

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** Unknown or Not Reported

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 6.19

**Value:** 37.4

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 18.14

**Value:** 97.7

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 34.53

**Value:** 68.2

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 3.32

**Value:** 15.1

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Sitting

#### Measurement

**Group ID:** BG000

**Spread:** 3.71

**Value:** 14.7

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 13

**Class Title:** After 5 minutes supine

#### Measurement

**Group ID:** BG000

**Spread:** 3.28

**Value:** 14.7

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 15

**Class Title:** After 10 minutes supine

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 10.99

**Value:** 68.9

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Sitting

#### Measurement

**Group ID:** BG000

**Spread:** 13.46

**Value:** 63.8

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 13

**Class Title:** After 5 minutes supine

#### Measurement

**Group ID:** BG000

**Spread:** 12.30

**Value:** 65.3

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 15

**Class Title:** After 10 minutes supine

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 1.65

**Value:** 96.9

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Sitting

#### Measurement

**Group ID:** BG000

**Spread:** 2.79

**Value:** 95.4

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 13

**Class Title:** After 5 minutes supine

#### Measurement

**Group ID:** BG000

**Spread:** 2.70

**Value:** 95.1

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 15

**Class Title:** After 10 minutes supine

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 16.8444

**Value:** 24.833

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Sitting

#### Measurement

**Group ID:** BG000

**Spread:** 15.9007

**Value:** 30.964

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 13

**Class Title:** After 5 minutes supine

#### Measurement

**Group ID:** BG000

**Spread:** 14.5895

**Value:** 31.186

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 15

**Class Title:** After 10 minutes supine

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 5.465

**Value:** 12.46

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 3.698

**Value:** 4.68

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 20.630

**Value:** 26.41

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 17

**Class Title:** Grip - Left

#### Measurement

**Group ID:** BG000

**Spread:** 18.667

**Value:** 26.86

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 18

**Class Title:** Grip - Right

#### Measurement

**Group ID:** BG000

**Spread:** 8.218

**Value:** 13.94

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 17

**Class Title:** Shoulder Flexion - Left

#### Measurement

**Group ID:** BG000

**Spread:** 9.850

**Value:** 14.42

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 17

**Class Title:** Shoulder Flexion - Right

#### Measurement

**Group ID:** BG000

**Spread:** 12.796

**Value:** 22.11

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Knee Extension - Left

#### Measurement

**Group ID:** BG000

**Spread:** 13.224

**Value:** 22.13

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Knee Extension - Right

#### Measurement

**Group ID:** BG000

**Spread:** 11.832

**Value:** 17.32

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Knee Flexion - Left

#### Measurement

**Group ID:** BG000

**Spread:** 12.987

**Value:** 18.02

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Knee Flexion - Right

#### Measurement

**Group ID:** BG000

**Spread:** 16.229

**Value:** 20.13

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Hip Flexion - Left

#### Measurement

**Group ID:** BG000

**Spread:** 12.738

**Value:** 20.82

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** Hip Flexion - Right

#### Measurement

**Group ID:** BG000

**Spread:** 9.074

**Value:** 17.16

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 17

**Class Title:** Elbow Flexion - Left

#### Measurement

**Group ID:** BG000

**Spread:** 10.508

**Value:** 16.52

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 17

**Class Title:** Elbow Flexion - Right

#### Measurement

**Group ID:** BG000

**Spread:** 10.514

**Value:** 17.51

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 17

**Class Title:** Elbow Extension - Left

#### Measurement

**Group ID:** BG000

**Spread:** 10.482

**Value:** 15.36

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 18

**Class Title:** Elbow Extension - Right

#### Measurement

**Group ID:** BG000

**Spread:** 12.188

**Value:** 16.47

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 18

**Class Title:** Ankle Dorsi Flexion - Left

#### Measurement

**Group ID:** BG000

**Spread:** 10.898

**Value:** 13.04

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 18

**Class Title:** Ankle Dorsi Flexion - Right

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 0.45

**Value:** 100.3

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 23.51

**Value:** 48.5

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 45.4527

**Value:** 143.529

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 19

**Class Title:** 

**Denomination Units Selected:** 

## Results Section - Baseline Characteristics Module

### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Ethnicity (NIH/OMB)
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race (NIH/OMB)
**Unit of Measure:** Participants
### Measure 5
**Description:** ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in amyotrophic lateral sclerosis (ALS). Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task).
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score
**Unit of Measure:** units on a scale
### Measure 6
**Description:** Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation.
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Vital Capacity
**Unit of Measure:** percentage of capacity
### Measure 7
**Description:** MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength.
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Maximum Inspiratory Pressure (MIP)
**Unit of Measure:** centimetres of water at (cm H2O)
### Measure 8
**Description:** Respiratory rate is the rate at which breathing occurs.
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Population Description:** 'Number analyzed' signifies participants evaluable for specified categories.
**Title:** Respiratory Rate
**Unit of Measure:** breaths/minute
### Measure 9
**Description:** The heart rate measures the number of times the heart beats per minute.
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Population Description:** 'Number analyzed' signifies participants evaluable for specified categories.
**Title:** Heart Rate
**Unit of Measure:** beats/minute
### Measure 10
**Description:** SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen.
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Population Description:** 'Number analyzed' signifies participants evaluable for specified categories.
**Title:** Oxygen Saturation (SpO2)
**Unit of Measure:** percentage of hemoglobin
### Measure 11
**Description:** ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation.
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Population Description:** 'Number analyzed' signifies participants evaluable for specified categories.
**Title:** End-Tidal Carbon Dioxide (ETCO2)
**Unit of Measure:** percentage of CO2
### Measure 12
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Average Solid Swallowing Time
**Unit of Measure:** seconds
### Measure 13
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Average Water Swallowing Time
**Unit of Measure:** seconds
### Measure 14
**Description:** Handheld dynamometry parameters included grip strength, shoulder flexion, knee extension, hip flexion, elbow flexion, elbow extension, and ankle dorsi flexion.
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Population Description:** 'Number analyzed' signifies participants evaluable for specified categories.
**Title:** Muscle Function, as Assessed by Handheld Dynamometry Parameters
**Unit of Measure:** kilograms
### Measure 15
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Number of Words Participant Read
**Unit of Measure:** Words
### Measure 16
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Time Spent in Reading
**Unit of Measure:** seconds
### Measure 17
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Number of Words Per Minute Read
**Unit of Measure:** words/minute
**Population Description:** Efficacy intent-to-treat (EITT) population or safety population included any participant who received at least 1 dose of EPI-589.
## Results Section - More Information Module
### Certain Agreement
**Other Details:** The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Point of Contact
**Email:** medinfo@ptcbio.com
**Organization:** PTC Therapeutics, Inc.
**Phone:** 1-866-562-4620
**Title:** Patient Advocacy
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
### Outcome Measure 6
### Outcome Measure 7
### Outcome Measure 8
### Outcome Measure 9
### Outcome Measure 10
### Outcome Measure 11
### Outcome Measure 12
### Outcome Measure 13
### Outcome Measure 14
### Outcome Measure 15
### Outcome Measure 16
### Outcome Measure 17
### Outcome Measure 18
### Outcome Measure 19
### Outcome Measure 20
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 721.1
- **Upper Limit:** 
- **Value:** 7762.7
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 10
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 401.1
- **Upper Limit:** 
- **Value:** 6878.8
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 9
**Units:** Participants
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 930.3
- **Upper Limit:** 
- **Value:** 5179.0
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 10
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 642.2
- **Upper Limit:** 
- **Value:** 4167.8
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 9
**Units:** Participants
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 4.03
- **Upper Limit:** 
- **Value:** -4.1
**Title:** 
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 9.36
- **Upper Limit:** 
- **Value:** -3.1
**Title:** 
#### Outcome Measure 6
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 24.83
- **Upper Limit:** 
- **Value:** 2.4
**Title:** 
#### Outcome Measure 7
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 3.45
- **Upper Limit:** 
- **Value:** 0.3
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 5.57
- **Upper Limit:** 
- **Value:** -0.6
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 11
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 4.32
- **Upper Limit:** 
- **Value:** 0.0
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
#### Outcome Measure 8
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 10.74
- **Upper Limit:** 
- **Value:** 5.2
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 7.49
- **Upper Limit:** 
- **Value:** 7.1
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 11
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 8.39
- **Upper Limit:** 
- **Value:** 7.2
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
#### Outcome Measure 9
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 1.71
- **Upper Limit:** 
- **Value:** -1.0
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 4.64
- **Upper Limit:** 
- **Value:** -0.8
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 11
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 3.55
- **Upper Limit:** 
- **Value:** -0.1
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
#### Outcome Measure 10
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 8.2343
- **Upper Limit:** 
- **Value:** 2.384
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 5.0262
- **Upper Limit:** 
- **Value:** 0.605
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 11
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 9.6591
- **Upper Limit:** 
- **Value:** 7.112
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
#### Outcome Measure 11
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
**Title:** 
#### Outcome Measure 12
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 11.718
- **Upper Limit:** 
- **Value:** 4.80
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 20.950
- **Upper Limit:** 
- **Value:** 6.65
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
#### Outcome Measure 13
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 15.330
- **Upper Limit:** 
- **Value:** -9.69
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 7.207
- **Upper Limit:** 
- **Value:** -7.28
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 6.922
- **Upper Limit:** 
- **Value:** -4.53
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 12
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 8.915
- **Upper Limit:** 
- **Value:** -5.43
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 12
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 10.254
- **Upper Limit:** 
- **Value:** -5.42
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 11.505
- **Upper Limit:** 
- **Value:** -5.05
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 8.237
- **Upper Limit:** 
- **Value:** -4.63
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 9.170
- **Upper Limit:** 
- **Value:** -4.01
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 12.579
- **Upper Limit:** 
- **Value:** -6.84
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 9.627
- **Upper Limit:** 
- **Value:** -5.60
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 8.377
- **Upper Limit:** 
- **Value:** -4.84
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 8.186
- **Upper Limit:** 
- **Value:** -3.28
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 10.511
- **Upper Limit:** 
- **Value:** -5.77
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 9.048
- **Upper Limit:** 
- **Value:** -4.28
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 7.609
- **Upper Limit:** 
- **Value:** -6.12
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 7.529
- **Upper Limit:** 
- **Value:** -4.89
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 13
**Units:** Participants
#### Outcome Measure 14
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 0.00
- **Upper Limit:** 
- **Value:** 0.0
**Title:** 
#### Outcome Measure 15
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 11.78
- **Upper Limit:** 
- **Value:** 5.5
**Title:** 
#### Outcome Measure 16
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 26.7792
- **Upper Limit:** 
- **Value:** -147.535
**Title:** 
#### Outcome Measure 17
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 10
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 6
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 6
**Title:** 
#### Outcome Measure 18
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 0.41
- **Spread:** 
- **Upper Limit:** 2.15
- **Value:** 1.34
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 0.83
- **Spread:** 
- **Upper Limit:** 11.74
- **Value:** 2.73
**Title:** 
#### Outcome Measure 19
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 0.04
- **Spread:** 
- **Upper Limit:** 0.31
- **Value:** 0.13
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 0.06
- **Spread:** 
- **Upper Limit:** 0.31
- **Value:** 0.13
**Title:** 
#### Outcome Measure 20
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 0.03
- **Spread:** 
- **Upper Limit:** 0.46
- **Value:** 0.12
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 0.02
- **Spread:** 
- **Upper Limit:** 0.82
- **Value:** 0.17
**Title:** 
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Safety population included any participant who received at least 1 dose of EPI-589.
**Reporting Status:** POSTED
**Time Frame:** Baseline (Day 0) to Month 6
**Title:** Number of Participants With Drug-Related Serious Adverse Events (SAEs)
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 2
**Dispersion Type:** Standard Error
**Parameter Type:** MEAN
**Population Description:** EITT population or safety population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable at specified timepoints.
**Reporting Status:** POSTED
**Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3
**Title:** Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12)
**Type:** SECONDARY
**Unit of Measure:** hours*nanograms/milliliter
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 3
**Dispersion Type:** Standard Error
**Parameter Type:** MEAN
**Population Description:** EITT population or safety population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable at specified timepoints.
**Reporting Status:** POSTED
**Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3
**Title:** Maximum Observed Plasma Concentration (Cmax)
**Type:** SECONDARY
**Unit of Measure:** ng/mL
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 4
**Description:** ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task).
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in ALSFRS-R Total Score at Month 6
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 5
**Description:** Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Vital Capacity at Month 6
**Type:** SECONDARY
**Unit of Measure:** percentage of capacity
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 6
**Description:** MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in MIP at Month 6
**Type:** SECONDARY
**Unit of Measure:** cm H2O
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 7
**Description:** Respiratory rate is the rate at which breathing occurs.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Respiratory Rate at Month 6
**Type:** SECONDARY
**Unit of Measure:** breaths/minute
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 8
**Description:** The heart rate measures the number of times the heart beats per minute.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Heart Rate at Month 6
**Type:** SECONDARY
**Unit of Measure:** beats/minute
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 9
**Description:** SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in SpO2 at Month 6
**Type:** SECONDARY
**Unit of Measure:** percentage of hemoglobin
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 10
**Description:** ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in ETCO2 at Month 6
**Type:** SECONDARY
**Unit of Measure:** percentage of CO2
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 11
**Description:** The failure to thrive as measured by body weight was defined as weight loss of more than 5% from baseline
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6
**Type:** SECONDARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 12
**Description:** Participants were observed and timed swallowing water as well as solid foods in accordance with a standardized protocol.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable for specified categories.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6
**Type:** SECONDARY
**Unit of Measure:** seconds
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 13
**Description:** The strength of designated muscle groups was measured using handheld dynamometry.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion)
**Type:** SECONDARY
**Unit of Measure:** kilograms
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 14
**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Number of Words Participant Read at Month 6
**Type:** SECONDARY
**Unit of Measure:** words
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 15
**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Time Spent in Reading at Month 6
**Type:** SECONDARY
**Unit of Measure:** seconds
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 16
**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Number of Words Per Minute Read at Month 6
**Type:** SECONDARY
**Unit of Measure:** words/minute
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 17
**Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
**Reporting Status:** POSTED
**Time Frame:** Month 6
**Title:** Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility
**Type:** SECONDARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 18
**Description:** Glutathione lowest limit of quantification (LLOQ) = 0.089 micromoles (uM) and upper limit of quantification (ULOQ) = 13.916 uM in plasma.
**Dispersion Type:** Full Range
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 6 glutathione in plasma data.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to Month 6
**Title:** Level of Disease-Related Biomarker (Glutathione) in Plasma
**Type:** SECONDARY
**Unit of Measure:** µM
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 19
**Description:** Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF.
**Dispersion Type:** Full Range
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 3 glutathione in CSF data.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to Month 3
**Title:** Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)
**Type:** SECONDARY
**Unit of Measure:** µM
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
#### Outcome Measure 20
**Description:** Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine.
**Dispersion Type:** Full Range
**Parameter Type:** MEAN
**Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 6 glutathione in urine data.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to Month 6
**Title:** Level of Disease-Related Biomarker (Glutathione) in Urine
**Type:** SECONDARY
**Unit of Measure:** µM
##### Group
**Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues.
**ID:** OG000
**Title:** EPI-589
### Participant Flow Module
#### Group
**Description:** Participants received EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues.
**ID:** FG000
**Title:** EPI-589
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Investigator Decision
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 4
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 19
##### Milestone
**Type:** Received at Least 1 Dose of Study Drug
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 19
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 14
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 5
**Has Results:** True