## Protocol Section
### Identification Module
**NCT ID:** NCT06414954

**Acronym:** SYNAPSE-MG

**Brief Title:** Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis

**Official Title:** A Phase 2b, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 3 Dose Levels of NMD670 Over 21 Days in Adult Patients With AChR/MuSK-Ab+ Myasthenia Gravis

#### Organization Study ID Info
**ID:** NMD670-02-0002
#### Organization
**Class:** INDUSTRY
**Full Name:** NMD Pharma A/S
#### Secondary ID Infos
**Domain:** EUCTR
**ID:** 2023-507539-40
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2025-11

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-16

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-10

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2025-11

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-05

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-16
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-10
**Study First Submit QC Date:** 2024-05-10
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** NMD Pharma A/S

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** This Phase 2 proof-of-concept, dose range finding study aims to evaluate the safety and efficacy of 3 dose levels of NMD670 vs placebo in adult patients with MG with antibodies against AChR or MuSK, administered twice a day (BID) for 21 days.

### Conditions Module

**Conditions:**

- Myasthenia Gravis

- Myasthenia Gravis, MuSK

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** TRIPLE

**Who Masked:**

- PARTICIPANT

- INVESTIGATOR

- OUTCOMES_ASSESSOR

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 84

**Type:** ESTIMATED

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Intervention Names:**

- Drug: NMD670

**Label:** NMD670 high dose

**Type:** EXPERIMENTAL

#### Arm Group 2

**Intervention Names:**

- Drug: NMD670

**Label:** NMD670 mid dose

**Type:** EXPERIMENTAL

#### Arm Group 3

**Intervention Names:**

- Drug: NMD670

**Label:** NMD670 low dose

**Type:** EXPERIMENTAL

#### Arm Group 4

**Intervention Names:**

- Drug: Placebo

**Label:** Placebo

**Type:** PLACEBO_COMPARATOR

### Interventions

#### Intervention 1

**Arm Group Labels:**

- NMD670 high dose

- NMD670 low dose

- NMD670 mid dose

**Description:** Tablets taken twice a day for 21 days

**Name:** NMD670

**Type:** DRUG

#### Intervention 2

**Arm Group Labels:**

- Placebo

**Description:** Tablets taken twice a day for 21 days

**Name:** Placebo

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Scale goes from 0-36 and higher score indicates worse symptomatology

**Measure:** Change from baseline to day 21 in QMG total score for NMD670 vs placebo

**Time Frame:** Baseline to day 21

#### Secondary Outcomes

**Description:** Scale goes from 0-24 and higher score indicates worse symptomatology

**Measure:** Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo

**Time Frame:** Baseline to day 21

**Description:** Scale goes from 0-50 and higher score indicate worse symptomatology

**Measure:** Change from baseline to day 21 in MGC total score for NMD670 vs placebo

**Time Frame:** Baseline to day 21

**Description:** Scale goes from 0-30 and higher score indicate worse quality of life

**Measure:** Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo

**Time Frame:** Baseline to day 21

**Description:** Scale goes from 8-40 and higher score indicate worse symptomalogy

**Measure:** Change from baseline to day 21 in Neuro-QoL Fatigue Short Form

**Time Frame:** Baseline to day 21

**Description:** Summarised per treatment

**Measure:** Incidence of treatment emergent adverse event

**Time Frame:** Over 21 days of dosing

**Description:** Summarised per treatment

**Measure:** Incidence of serious treatment emergent adverse events

**Time Frame:** Over 21 days of dosing

**Description:** Summarised per treatment

**Measure:** Incidence of clinically significant abnormalities on physical examinations

**Time Frame:** Over 21 days of dosing

**Description:** Summarised per treatment

**Measure:** Incidence of clinically significant abnormalities on safety laboratory parameters

**Time Frame:** Over 21 days of dosing

**Description:** Summarised per treatment

**Measure:** Incidence of clinically significant vital signs abnormalities

**Time Frame:** Over 21 days of dosing

**Description:** Summarised per treatment

**Measure:** Incidence of clinically significant ECG abnormalities

**Time Frame:** Over 21 days of dosing

**Description:** Summarised per treatment

**Measure:** Incidence of Suicidal Ideation or Suicidal Behavior

**Time Frame:** Over 21 days of dosing

**Description:** Summarised per treatment

**Measure:** Incidence of clinically significant abnormalities on opthalmological examinations

**Time Frame:** From screening (day -28 to day -1) until follow up (day 28)

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Participant must be a male or female being 18 to 75 years (both included), at the time of signing the informed consent
* Diagnosis of MG, MGFA class II, III or IV
* Documented positive AChR or MuSK antibody test.
* Participant must be able to swallow tablets
* Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
* Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
* Participant is capable of and has given signed informed consent

Exclusion Criteria:

* Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study
* Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
* Participants that received treatment with an investigational medical product within 30 days (or 5 half-lives of the medication, whichever is longer) prior to Day 1
* Participants with history of poor compliance with relevant MG therapy
* Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding

**Maximum Age:** 75 Years

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** contact@nmdpharma.com

**Name:** NMD Pharma A/S

**Phone:** contact@nmdpharma.com

**Role:** CONTACT

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000009135

- Term: Muscular Diseases

- ID: D000009140

- Term: Musculoskeletal Diseases

- ID: D000020879

- Term: Neuromuscular Manifestations

- ID: D000009461

- Term: Neurologic Manifestations

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000010335

- Term: Pathologic Processes

- ID: D000020361

- Term: Paraneoplastic Syndromes, Nervous System

- ID: D000009423

- Term: Nervous System Neoplasms

- ID: D000009371

- Term: Neoplasms by Site

- ID: D000009369

- Term: Neoplasms

- ID: D000010257

- Term: Paraneoplastic Syndromes

- ID: D000020274

- Term: Autoimmune Diseases of the Nervous System

- ID: D000019636

- Term: Neurodegenerative Diseases

- ID: D000020511

- Term: Neuromuscular Junction Diseases

- ID: D000009468

- Term: Neuromuscular Diseases

- ID: D000001327

- Term: Autoimmune Diseases

- ID: D000007154

- Term: Immune System Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC04

- Name: Neoplasms

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC20

- Name: Immune System Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC05

- Name: Musculoskeletal Diseases

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M12112

- Name: Myasthenia Gravis

- Relevance: HIGH

- As Found: Myasthenia Gravis

- ID: M20944

- Name: Muscle Weakness

- Relevance: HIGH

- As Found: Myasthenia

- ID: M13204

- Name: Paresis

- Relevance: LOW

- As Found: Unknown

- ID: M4554

- Name: Asthenia

- Relevance: LOW

- As Found: Unknown

- ID: M12092

- Name: Muscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12097

- Name: Musculoskeletal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22619

- Name: Neuromuscular Manifestations

- Relevance: LOW

- As Found: Unknown

- ID: M12404

- Name: Neurologic Manifestations

- Relevance: LOW

- As Found: Unknown

- ID: M16355

- Name: Syndrome

- Relevance: LOW

- As Found: Unknown

- ID: M13170

- Name: Paraneoplastic Syndromes

- Relevance: LOW

- As Found: Unknown

- ID: M22160

- Name: Paraneoplastic Syndromes, Nervous System

- Relevance: LOW

- As Found: Unknown

- ID: M12367

- Name: Nervous System Neoplasms

- Relevance: LOW

- As Found: Unknown

- ID: M4629

- Name: Autoimmune Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22094

- Name: Autoimmune Diseases of the Nervous System

- Relevance: LOW

- As Found: Unknown

- ID: M21558

- Name: Neurodegenerative Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22297

- Name: Neuromuscular Junction Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12411

- Name: Neuromuscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M10200

- Name: Immune System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T3973

- Name: Myasthenia Gravis

- Relevance: HIGH

- As Found: Myasthenia Gravis

### Condition Browse Module - Meshes

- ID: D000009157

- Term: Myasthenia Gravis

- ID: D000018908

- Term: Muscle Weakness

### Misc Info Module

- Version Holder: 2024-05-31
