## Protocol Section
### Identification Module
**NCT ID:** NCT03105479

**Brief Title:** Comparison of Cadazolid Versus Vancomycin in Children With Clostridium Difficile-associated Diarrhea (CDAD)

**Official Title:** A Prospective, Multicenter Study to Investigate the Pharmacokinetics, Safety, and Efficacy of Cadazolid Versus Vancomycin in Pediatric Subjects With Clostridium Difficile-associated Diarrhea

#### Organization Study ID Info
**ID:** AC-061A303
#### Organization
**Class:** INDUSTRY
**Full Name:** Actelion
#### Secondary ID Infos
**ID:** 2015-004805-17
**Type:** EUDRACT_NUMBER
### Status Module
#### Completion Date
**Date:** 2018-04-17

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2019-04-03

**Type:** ACTUAL

**Last Update Submit Date:** 2019-03-14

**Overall Status:** TERMINATED

#### Primary Completion Date
**Date:** 2018-04-17

**Type:** ACTUAL

#### Results First Post Date
**Date:** 2019-04-03

**Type:** ACTUAL

**Results First Submit Date:** 2019-01-08

**Results First Submit QC Date:** 2019-03-14

#### Start Date
**Date:** 2017-04-14

**Type:** ACTUAL

**Status Verified Date:** 2019-03

#### Study First Post Date
**Date:** 2017-04-10
**Type:** ACTUAL
**Study First Submit Date:** 2017-03-23
**Study First Submit QC Date:** 2017-04-03
**Why Stopped:** Study suspended in October 2017 and terminated April 17, 2018 after decision to discontinue the study drug development
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Actelion

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** Cadazolid has demonstrated activity against a bacteria named Clostridium difficile in animal studies. The results of a first study conducted in adult patients have suggested efficacy of the new antibiotic, cadazolid, in the treatment of diarrhea caused by this bacteria. This is the first study of cadazolid in children. The overall purpose of this study is to provide reassurance on the safety and efficacy of cadazolid in children suffering from infection due to Clostridium difficile.

**Detailed Description:** This multicenter, study will be run into two parts. Both parts will be run in consecutive age cohorts, starting from the oldest age categories(12 to \< 18 years old) to the youngest (birth to \< 3 months).

* Part A is an open-label, dose finding part to be conducted in at least 24 subjects.
* Part B follows a randomized, assessor-blinded, parallel-group design with vancomycin used as an active comparator. Part B will be conducted in about 176 children.

In both parts, the treatment period will be 10 days and will be followed by a Follow-up period of 28-32 days.

### Conditions Module

**Conditions:**

- Clostridium Difficile Infection

**Keywords:**

- vancomycin

- children

- cadazolid

- clostridium difficile associated diarrhea

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** SINGLE

**Masking Description:** Assessor masking in Part B only (no masking in Part A)

**Who Masked:**

- OUTCOMES_ASSESSOR

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 1

**Type:** ACTUAL

**Phases:**

- PHASE2

- PHASE3

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Subjects from 12 years to 18 years old (exclusive) will receive cadazolid 500 mg per day for 10 days. The dose may be adjusted based on the pharmacokinetic (PK) and safety data reviewed for the first 3 subjects.

**Intervention Names:**

- Drug: Cadazolid

**Label:** Part A / Cohort A

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** Subjects from 6 years to 12 years old (exclusive) will receive cadazolid for 10 days. The dose will depend on the PK and safety data from cohort A reviewed by the Independent Data Monitoring Committee (IDMC).

**Intervention Names:**

- Drug: Cadazolid

**Label:** Part A / Cohort B

**Type:** EXPERIMENTAL

#### Arm Group 3

**Description:** Subjects from 2 years to 6 years old (exclusive) will receive cadazolid for 10 days. The dose will depend on the PK and safety data from cohort B reviewed by the IDMC.

**Intervention Names:**

- Drug: Cadazolid

**Label:** Part A / Cohort C

**Type:** EXPERIMENTAL

#### Arm Group 4

**Description:** Subjects from 3 months to 2 years old (exclusive) will receive cadazolid for 10 days. The dose will depend on the PK and safety data from cohort C reviewed by the IDMC.

**Intervention Names:**

- Drug: Cadazolid

**Label:** Part A/ Cohort D

**Type:** EXPERIMENTAL

#### Arm Group 5

**Description:** Subjects from birth to 3 months old (exclusive) will receive cadazolid for 10 days. The dose will depend on the PK and safety data from cohort D reviewed by the IDMC.

**Intervention Names:**

- Drug: Cadazolid

**Label:** Part A/ Cohort E

**Type:** EXPERIMENTAL

#### Arm Group 6

**Description:** Subjects from birth to 18 years old (exclusive) will receive cadazolid for 10 days, at the dose defined in the corresponding age cohort in Part A.

**Intervention Names:**

- Drug: Cadazolid

**Label:** Part B / Cadazolid

**Type:** EXPERIMENTAL

#### Arm Group 7

**Description:** Subjects from birth to 18 years old (exclusive) will receive vancomycin capsule (for subjects able to swallow) or vancomycin solution (for the others) during 10 days .

**Intervention Names:**

- Drug: Vancomycin capsule

- Drug: Vancomycin solution

**Label:** Part B / Vancomycin

**Type:** ACTIVE_COMPARATOR

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Part A / Cohort A

- Part A / Cohort B

- Part A / Cohort C

- Part A/ Cohort D

- Part A/ Cohort E

- Part B / Cadazolid

**Description:** Granules for oral suspension to be administered twice daily

**Name:** Cadazolid

**Other Names:**

- ACT-179811

**Type:** DRUG

#### Intervention 2

**Arm Group Labels:**

- Part B / Vancomycin

**Description:** Capsule containing 125 mg of vancomycin to be administered orally 4 times a day

**Name:** Vancomycin capsule

**Type:** DRUG

#### Intervention 3

**Arm Group Labels:**

- Part B / Vancomycin

**Description:** Vancomycin powder to be administered as oral solution at a dose of 40 mg/kg/day, 3 to 4 times a day

**Name:** Vancomycin solution

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** This is the percentage of participants in part B reported as with a clinical cure. Clinical Cure is defined as: • \<3 unformed bowel movement (UBM) per day (or no water diarrhea if subjects \< 2 years of age), for at least 2 consecutive days between first dose of study treatment up to end of treatment (EOT) (inclusive) AND • Subject remains well up to EOT + 2 days (inclusive) based on investigator judgment AND • No need for additional antimicrobial treatment active against Clostridium difficile-associated diarrhea (CDAD) between first dose of study treatment up to EOT + 2 days (inclusive). percentage of subjects with a clinical cure

**Measure:** Clinical Cure Rate During Part B

**Time Frame:** Day 10 (End of Treatment) + 2 days

**Description:** Blood samples are collected at different timepoints on Day 10 for the determination of cadazolid Cmax after 10 days of treatment.

**Measure:** Maximal Plasma Concentration (Cmax) of Cadazolid During Part A

**Time Frame:** Day 10 (End of Treatment)

**Description:** Blood samples are collected at different timepoints to determine the time when the maximal plasma concentration of cadazolid is reached.

**Measure:** Time to Reach Cmax (Tmax) of Cadazolid During Part A

**Time Frame:** Day 10 (End of Treatment)

**Description:** Blood samples are collected at different timepoints for the determination of the cadazolid AUC over one dosing interval (0-12h) on Day 10.

**Measure:** Area Under the Plasma Concentration Time Curve (AUC) of Cadazolid During Part A

**Time Frame:** Day 10 (End of Treatment)

**Description:** A fecal sample is collected as the end-of-treatment visit in all participants in Part A.

**Measure:** Fecal Concentrations of Cadazolid During Part A

**Time Frame:** Day 10 (End of Treatment)

#### Secondary Outcomes

**Description:** This is the percentage of participants in Part A reported as with a clinical cure. Clinical Cure is defined as: • \<3 unformed bowel movement (UBM) per day (or no water diarrhea if subjects \< 2 years of age), for at least 2 consecutive days between first dose of study treatment up to end of treatment (EOT) (inclusive) AND • Subject remains well up to EOT + 2 days (inclusive) based on investigator judgment AND • No need for additional antimicrobial treatment active against Clostridium difficile-associated diarrhea (CDAD) between first dose of study treatment up to EOT + 2 days (inclusive).

**Measure:** Clinical Cure Rate During Part A

**Time Frame:** Day 10 (End of Treatment) + 2 days

**Description:** This is the percentage of participants in Parts A and B reported as with sustained clinical cure. Sustained clinical cure is defined as • Clinical Cure and no Recurrence until 30 days after the last study drug intake (end of study).

**Measure:** Sustained Clinical Cure Rate During Part A and Part B

**Time Frame:** Day 40 (on average)

**Description:** This is the percentage of participants in Parts A and B assessed as having a recurrence out of subjects meeting the criteria for Clinical Cure. Recurrence is defined as: • Clinical Cure AND New episode of diarrhea with ≥ 3 UBMs (or watery diarrhea if subject \< 2 years) on any day between EOT + 3 days and end of study AND • Stool test showing positive C. difficile (as defined in Inclusion Criterion 4), AND •Antimicrobial treatment active against CDAD started between EOT + 3 days and end of study.

**Measure:** Recurrence Rate During Part A and Part B

**Time Frame:** Day 40 (on average)

**Description:** This it the time (in days) elapsed between the last dose of study drug and the onset day of new episode of diarrhea reported as Kaplan-Meier estimates (KM estimates)

**Measure:** Time to Recurrence in Part B

**Time Frame:** Day 40 (on average)

**Description:** This is the time (in days) elapsed between the first dose of study treatment and the resolution of diarrhea and reported as Kaplan-Meier estimates (KM estimates). The date of resolution of Diarrhea (ROD) is defined as the date of the first day of the 2 consecutive days on treatment with \< 3 UBM (or no watery diarrhea for subjects \< 2 years of age). Time to ROD is the time (in days) elapsed between the first dose of study treatment and the ROD

**Measure:** Time to Resolution of Diarrhea in Part B

**Time Frame:** Day 10

**Description:** Number of participants who prematurely discontinued the study treatment due to an adverse event

**Measure:** Adverse Events Leading to Premature Discontinuation of Study Treatment

**Time Frame:** Up to Day 10

**Description:** Number of participants with any marked abnormalities in laboratory parameters up to 7 days after end of treatment

**Measure:** Marked Abnormalities in Clinical Laboratory Parameters

**Time Frame:** Day 17 (on average)

**Description:** Number of subjects with any treatment-emergent abnormalities in vital signs (up to 7 days after end of treatment)

**Measure:** Marked Abnormalities in Vital Signs

**Time Frame:** Day 17 (on average)

**Description:** Number of subjects with any TEAEs. A TEAE is any adverse event temporally associated with the use of study treatment (from study treatment initiation until 7 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.

**Measure:** Treatment-emergent Adverse Events (TEAES)

**Time Frame:** Day 17 (on average)

### Eligibility Module

**Eligibility Criteria:** Key Inclusion Criteria:

* Signed informed consent by parents or legally authorized representatives (LAR) and assent by the child according to local requirements prior to initiation of any study-mandated procedure.
* Male or female from birth to \< 18 years of age, diagnosed with Clostridium Difficile-associated diarrhea (CDAD).
* Females of childbearing potential must have a negative pregnancy test at screening and must agree to use an adequate and reliable method of contraception.

Key Exclusion Criteria:

* Positive Rotavirus test for subjects \< 5 years.
* Fulminant or life-threatening CDAD.
* More than one previous episode of CDAD in the 3 month period prior to enrollment / randomization.
* Antimicrobial treatment active against CDAD administered within 24 h prior to screening except for metronidazole treatment failures (MTF).
* Subjects with body weight \< 3 kg.
* Inflammatory bowel disease, chronic abdominal pain, or chronic diarrhea of any etiology.
* Fecal microbiota transplant (FMT), immunoglobulin therapy, or any investigational drug to prevent or treat CDAD within 1 month period (or 5 half-lives in case of investigational drug, whichever is longer) prior to enrollment / randomization.
* Monoclonal antibodies against C. difficile within 6 months prior to enrollment / randomization.
* Previous vaccination against C. difficile.
* Known mental disorders.
* Any circumstances or conditions, which, in the opinion of the investigator, may affect the subject's full participation in the study, or compliance with the protocol.

**Maximum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Idaho Falls

**Country:** United States

**Facility:** Snake River Research, PLLC

**State:** Idaho

**Zip:** 83404

**Location 2:**

**City:** Chicago

**Country:** United States

**Facility:** University of Chicago, Dept. Of Medicine

**State:** Illinois

**Zip:** 60637

**Location 3:**

**City:** Shreveport

**Country:** United States

**Facility:** Louisiana State University Health Sciences Center - Shreveport

**State:** Louisiana

**Zip:** 71103

**Location 4:**

**City:** Syracuse

**Country:** United States

**Facility:** SUNY Upstate Medical University - Upstate Golisano Children's Hospital (GCH) - Pediatric Designated AIDS Center

**State:** Ohio

**Zip:** 13210

**Location 5:**

**City:** Houston

**Country:** United States

**Facility:** Texas Children's Hospital Feigin Cente

**State:** Texas

**Zip:** 77030

**Location 6:**

**City:** Jette

**Country:** Belgium

**Facility:** Universitair Ziekenhuis Brussel - Kinderziekenhuis

**Zip:** 1090

**Location 7:**

**City:** Calgary

**Country:** Canada

**Facility:** Infection Prevention & Control, AGW5 Foothills Medical Center 1403 29th Street N.W.

**Zip:** T2N 2T9

**Location 8:**

**City:** Brno

**Country:** Czechia

**Facility:** FN Brno

**Zip:** 62500

**Location 9:**

**City:** Budapest

**Country:** Hungary

**Facility:** Egyesített Szent István és Szent László Kórház - Rendelőintézet / Gyermekinfektológiai Osztály

**Zip:** 1097

**Location 10:**

**City:** Gyula

**Country:** Hungary

**Facility:** Pándy Kálmán Megyei Kórház

**Zip:** 5700

**Location 11:**

**City:** Milano

**Country:** Italy

**Facility:** Ospedale Buzzi

**Zip:** 20154

**Location 12:**

**City:** Roma

**Country:** Italy

**Facility:** Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale Pediatrico Bambino Gesu

**Zip:** 00165

**Location 13:**

**City:** Bydgoszcz

**Country:** Poland

**Facility:** Wojewodzki Szpital Obserwacyjno-Zakazny im. Tadeusza Browicza

**Zip:** 85-030

**Location 14:**

**City:** Poznan

**Country:** Poland

**Facility:** Specjalistyczny Zespół Opieki Zdrowotnej nad Matką i Dzieckiem

**Zip:** 61-734

**Location 15:**

**City:** Rzeszow

**Country:** Poland

**Facility:** Gabinet Lekarski Bartosz Korczowski

**Zip:** 35-302

**Location 16:**

**City:** Warsaw

**Country:** Poland

**Facility:** Klinika Gastroenterologii, Hepatologii, Zaburzeń Odżywiania i Pediatrii, Instytut "Pomnik - Centrum Zdrowia Dziecka"

**Zip:** 04-730

**Location 17:**

**City:** Bucharest

**Country:** Romania

**Facility:** Institutul National De Boli Infectioase "Prof. Dr. Matei Bals", sectia IX pediatrie

**Zip:** 21105

**Location 18:**

**City:** Iasi

**Country:** Romania

**Facility:** Spitalul Clinic de Boli Infectioase "Sfanta Parascheva" Iasi, Clinica de Boli Infectioase I,

**Zip:** 700116

**Location 19:**

**City:** Barcelona

**Country:** Spain

**Facility:** Hospital Sant Joan de Déu, Esplugues

**Zip:** 8950

**Location 20:**

**City:** Madrid

**Country:** Spain

**Facility:** Hospital Universitario Infantil LA PAZ

**Zip:** 28046

## Document Section

### Large Document Module

#### Large Docs

- Date: 2017-03-01

- Filename: Prot_000.pdf

- Has ICF: False

- Has Protocol: True

- Has SAP: False

- Label: Study Protocol

- Size: 2397107

- Type Abbrev: Prot

- Upload Date: 2019-03-14T09:15

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000012817

- Term: Signs and Symptoms, Digestive

- ID: D000016908

- Term: Gram-Positive Bacterial Infections

- ID: D000001424

- Term: Bacterial Infections

- ID: D000001423

- Term: Bacterial Infections and Mycoses

- ID: D000007239

- Term: Infections

### Condition Browse Module - Browse Branches

- Abbrev: BC01

- Name: Infections

- Abbrev: All

- Name: All Conditions

- Abbrev: BC23

- Name: Symptoms and General Pathology

### Condition Browse Module - Browse Leaves

- ID: M10283

- Name: Infections

- Relevance: LOW

- As Found: Unknown

- ID: M6368

- Name: Communicable Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M7159

- Name: Diarrhea

- Relevance: HIGH

- As Found: Diarrhea

- ID: M6247

- Name: Clostridium Infections

- Relevance: HIGH

- As Found: Clostridium Difficile Infection

- ID: M15622

- Name: Signs and Symptoms, Digestive

- Relevance: LOW

- As Found: Unknown

- ID: M4722

- Name: Bacterial Infections

- Relevance: LOW

- As Found: Unknown

- ID: M19252

- Name: Gram-Positive Bacterial Infections

- Relevance: LOW

- As Found: Unknown

- ID: M12136

- Name: Mycoses

- Relevance: LOW

- As Found: Unknown

- ID: M4721

- Name: Bacterial Infections and Mycoses

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000003015

- Term: Clostridium Infections

- ID: D000003967

- Term: Diarrhea

### Intervention Browse Module - Ancestors

- ID: D000000900

- Term: Anti-Bacterial Agents

- ID: D000000890

- Term: Anti-Infective Agents

### Intervention Browse Module - Browse Branches

- Abbrev: Infe

- Name: Anti-Infective Agents

- Abbrev: All

- Name: All Drugs and Chemicals

- Abbrev: PhSol

- Name: Pharmaceutical Solutions

### Intervention Browse Module - Browse Leaves

- ID: M17388

- Name: Vancomycin

- Relevance: HIGH

- As Found: Interaction

- ID: M21860

- Name: Pharmaceutical Solutions

- Relevance: LOW

- As Found: Unknown

- ID: M4222

- Name: Anti-Bacterial Agents

- Relevance: LOW

- As Found: Unknown

- ID: M4214

- Name: Anti-Infective Agents

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: D000014640

- Term: Vancomycin

### Misc Info Module

#### Removed Countries

- Country: Czech Republic

- Version Holder: 2024-05-24

## Results Section

### Adverse Events Module

**Description:** All adverse events (AE) which occurred at any time during the treatment period (10 days with cadazolid) and during the follow-up period (about 30 days) are reported. All AEs reported below occurred during the follow-up period.

#### Event Groups

**Group ID:** EG000

**Title:** Overall Study (Part A and Part B)

**Deaths Num At Risk:** 1

**Description:** Only one subject was included in cohort A; no subject was enrolled in the other cohorts of Part A or Part B due to early study termination.

**ID:** EG000

**Other Num Affected:** 1

**Other Num at Risk:** 1

**Serious Number At Risk:** 1

**Title:** Overall Study (Part A and Part B)

**Frequency Threshold:** 0

#### Other Events

**Term:** Sore throat

**Assessment Type:** SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA 20.0

**Term:** Headache

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 20.0

**Term:** Vomiting

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 20.0

**Time Frame:** From study treatment initiation up to Day 37 (i.e., 27 days after the end of treatment)

## Results Section - Baseline Characteristics Module

### Denomination Counts

**Group ID:** BG000

**Value:** 1

**Units:** Participants

### Group

**ID:** BG000

**Title:** Part A / Cohort A

**Description:** Subjects from 12 years to 18 years old (exclusive) will receive cadazolid 500 mg per day for 10 days. The dose may be adjusted based on the pharmacokinetic (PK) and safety data reviewed for the first 3 subjects.

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 1

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 1

**Class Title:** Adolescents (12 years to < 18 years)

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 1

**Class Title:** Children (2 years to < 12 years

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 1

**Class Title:** infants and toddlers (3 months to < 2 years

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 1

**Category Title:** Female

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** Male

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 1

**Class Title:** 

### Measure

#### Denomination

**Units:** Participants

##### Denomination Counts

**Group ID:** BG000

**Value:** 0

**Class Title:** 

**Denomination Units Selected:** 

## Results Section - Baseline Characteristics Module

### Measure 1
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Age, Customized
**Unit of Measure:** Participants
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Race and Ethnicity were not collected from any participant.
**Title:** Race and Ethnicity Not Collected
**Unit of Measure:** Participants
**Population Description:** Only one subject was enrolled in Part A. Part B was not conducted due to early study termination. Consequently the baseline characteristics are from one subject only.
## Results Section - More Information Module
### Certain Agreement
**Other Details:** Any study-related publication written independently by investigators must be submitted to Actelion for review at least 30 days prior to submission for publication or presentation. Upon review, Actelion may provide comments, and may also request alterations and/or deletions for the sole purpose of protecting its confidential information and/or patent rights.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Limitations and Caveats
**Description:** Only one subject was enrolled due to early study termination. Consequently results are not meaningful and no statistical analyses could be performed.
### Point of Contact
**Email:** clinical-trials-disclosure@its.jnj.com
**Organization:** Actelion Pharmaceuticals Ltd
**Phone:** 0041615656565
**Title:** clinical trial disclosure desk
## 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
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
#### Outcome Measure 2
#### Outcome Measure 3
#### Outcome Measure 4
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 4520
**Title:** 
#### Outcome Measure 6
#### Outcome Measure 7
#### Outcome Measure 8
#### Outcome Measure 9
#### Outcome Measure 10
#### Outcome Measure 11
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
#### Outcome Measure 12
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
#### Outcome Measure 13
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
#### Outcome Measure 14
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** This is the percentage of participants in part B reported as with a clinical cure. Clinical Cure is defined as: • \<3 unformed bowel movement (UBM) per day (or no water diarrhea if subjects \< 2 years of age), for at least 2 consecutive days between first dose of study treatment up to end of treatment (EOT) (inclusive) AND • Subject remains well up to EOT + 2 days (inclusive) based on investigator judgment AND • No need for additional antimicrobial treatment active against Clostridium difficile-associated diarrhea (CDAD) between first dose of study treatment up to EOT + 2 days (inclusive). percentage of subjects with a clinical cure
**Population Description:** Due to the premature study termination, no subject was enrolled into Part B and consequently the percentage of subjects with a clinical cure could not be reported.
**Reporting Status:** POSTED
**Time Frame:** Day 10 (End of Treatment) + 2 days
**Title:** Clinical Cure Rate During Part B
**Type:** PRIMARY
##### Group
**Description:** Due to early termination of the study, no subject was enrolled into Part B.
**ID:** OG000
**Title:** Part B
#### Outcome Measure 2
**Description:** Blood samples are collected at different timepoints on Day 10 for the determination of cadazolid Cmax after 10 days of treatment.
**Population Description:** Due to the premature study termination, cadazolid concentrations were obtained from only one subject and pharmacokineitc plasma profile was not analyzed because of lack of meaningful data.
**Reporting Status:** POSTED
**Time Frame:** Day 10 (End of Treatment)
**Title:** Maximal Plasma Concentration (Cmax) of Cadazolid During Part A
**Type:** PRIMARY
##### Group
**Description:** One Subject aged between 12 and 18 years old received cadazolid 500 mg per day for 10 days.
**ID:** OG000
**Title:** Part A / Cohort A
#### Outcome Measure 3
**Description:** Blood samples are collected at different timepoints to determine the time when the maximal plasma concentration of cadazolid is reached.
**Population Description:** Due to the premature study termination, cadazolid concentrations were obtained from only one subject and pharmacokineitc plasma profile was not analyzed because of lack of meaningful data.
**Reporting Status:** POSTED
**Time Frame:** Day 10 (End of Treatment)
**Title:** Time to Reach Cmax (Tmax) of Cadazolid During Part A
**Type:** PRIMARY
##### Group
**Description:** One Subject aged between 12 and 18 years old received cadazolid 500 mg per day for 10 days.
**ID:** OG000
**Title:** Part A / Cohort A
#### Outcome Measure 4
**Description:** Blood samples are collected at different timepoints for the determination of the cadazolid AUC over one dosing interval (0-12h) on Day 10.
**Population Description:** Due to the premature study termination, cadazolid concentrations were obtained from only one subject and pharmacokineitc plasma profile was not analyzed because of lack of meaningful data.
**Reporting Status:** POSTED
**Time Frame:** Day 10 (End of Treatment)
**Title:** Area Under the Plasma Concentration Time Curve (AUC) of Cadazolid During Part A
**Type:** PRIMARY
##### Group
**Description:** One Subject aged between 12 and 18 years old received cadazolid 500 mg per day for 10 days.
**ID:** OG000
**Title:** Part A / Cohort A
#### Outcome Measure 5
**Description:** A fecal sample is collected as the end-of-treatment visit in all participants in Part A.
**Parameter Type:** NUMBER
**Population Description:** Due to premature termination, fecal sample was collected from only one subject, consequently mean values cannot be provided and no statistical analyses can be performed.
**Reporting Status:** POSTED
**Time Frame:** Day 10 (End of Treatment)
**Title:** Fecal Concentrations of Cadazolid During Part A
**Type:** PRIMARY
**Unit of Measure:** mcg/g
##### Group
**Description:** One Subject aged between 12 and 18 years old received cadazolid 500 mg per day for 10 days.
**ID:** OG000
**Title:** Part A / Cohort A
#### Outcome Measure 6
**Description:** This is the percentage of participants in Part A reported as with a clinical cure. Clinical Cure is defined as: • \<3 unformed bowel movement (UBM) per day (or no water diarrhea if subjects \< 2 years of age), for at least 2 consecutive days between first dose of study treatment up to end of treatment (EOT) (inclusive) AND • Subject remains well up to EOT + 2 days (inclusive) based on investigator judgment AND • No need for additional antimicrobial treatment active against Clostridium difficile-associated diarrhea (CDAD) between first dose of study treatment up to EOT + 2 days (inclusive).
**Population Description:** Due to premature termination, data were collected from only one subject, consequently percentage of participants with clinical cure cannot be calculated and no statistical analyses can be performed
**Reporting Status:** POSTED
**Time Frame:** Day 10 (End of Treatment) + 2 days
**Title:** Clinical Cure Rate During Part A
**Type:** SECONDARY
##### Group
**Description:** One Subject aged between 12 and 18 years old received cadazolid 500 mg per day for 10 days.
**ID:** OG000
**Title:** Part A / Cohort A
#### Outcome Measure 7
**Description:** This is the percentage of participants in Parts A and B reported as with sustained clinical cure. Sustained clinical cure is defined as • Clinical Cure and no Recurrence until 30 days after the last study drug intake (end of study).
**Population Description:** Due to the premature termination of the study and consequent lack of meaningful data, no analyses were performed.
**Reporting Status:** POSTED
**Time Frame:** Day 40 (on average)
**Title:** Sustained Clinical Cure Rate During Part A and Part B
**Type:** SECONDARY
##### Group
**Description:** Only one subject was included in cohort A; no subject was enrolled in the other cohorts due to early study termination.
**ID:** OG000
**Title:** Part A / Cohorts A to E
##### Group
**Description:** No subject was enrolled in cohort B due to early study termination.
**ID:** OG001
**Title:** Part B / Cadazolid
#### Outcome Measure 8
**Description:** This is the percentage of participants in Parts A and B assessed as having a recurrence out of subjects meeting the criteria for Clinical Cure. Recurrence is defined as: • Clinical Cure AND New episode of diarrhea with ≥ 3 UBMs (or watery diarrhea if subject \< 2 years) on any day between EOT + 3 days and end of study AND • Stool test showing positive C. difficile (as defined in Inclusion Criterion 4), AND •Antimicrobial treatment active against CDAD started between EOT + 3 days and end of study.
**Population Description:** Due to the premature termination of the study and consequent lack of meaningful data, no analyses were performed.
**Reporting Status:** POSTED
**Time Frame:** Day 40 (on average)
**Title:** Recurrence Rate During Part A and Part B
**Type:** SECONDARY
##### Group
**Description:** Only one subject was included in cohort A; no subject was enrolled in the other cohorts due to early study termination.
**ID:** OG000
**Title:** Part A / Cohorts A to E
##### Group
**Description:** No subject was enrolled in cohort B due to early study termination.
**ID:** OG001
**Title:** Part B / Cadazolid
#### Outcome Measure 9
**Description:** This it the time (in days) elapsed between the last dose of study drug and the onset day of new episode of diarrhea reported as Kaplan-Meier estimates (KM estimates)
**Population Description:** Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B
**Reporting Status:** POSTED
**Time Frame:** Day 40 (on average)
**Title:** Time to Recurrence in Part B
**Type:** SECONDARY
##### Group
**Description:** Due to early termination of the study, no subject was enrolled into Part B.
**ID:** OG000
**Title:** Part B
#### Outcome Measure 10
**Description:** This is the time (in days) elapsed between the first dose of study treatment and the resolution of diarrhea and reported as Kaplan-Meier estimates (KM estimates). The date of resolution of Diarrhea (ROD) is defined as the date of the first day of the 2 consecutive days on treatment with \< 3 UBM (or no watery diarrhea for subjects \< 2 years of age). Time to ROD is the time (in days) elapsed between the first dose of study treatment and the ROD
**Population Description:** Due to premature termination, data were collected from only one subject, consequently KM estimates cannot be calculated and no statistical analyses can be performed
**Reporting Status:** POSTED
**Time Frame:** Day 10
**Title:** Time to Resolution of Diarrhea in Part B
**Type:** SECONDARY
##### Group
**Description:** Due to early termination of the study, no subject was enrolled into Part B.
**ID:** OG000
**Title:** Part B
#### Outcome Measure 11
**Description:** Number of participants who prematurely discontinued the study treatment due to an adverse event
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B
**Reporting Status:** POSTED
**Time Frame:** Up to Day 10
**Title:** Adverse Events Leading to Premature Discontinuation of Study Treatment
**Type:** SECONDARY
**Unit of Measure:** Participants
##### Group
**Description:** Only one subject was included in cohort A; no subject was enrolled in the other cohorts due to early study termination.
**ID:** OG000
**Title:** Part A / Cohorts A to E
##### Group
**Description:** No subject was enrolled in cohort B due to early study termination.
**ID:** OG001
**Title:** Part B / Cadazolid
#### Outcome Measure 12
**Description:** Number of participants with any marked abnormalities in laboratory parameters up to 7 days after end of treatment
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B
**Reporting Status:** POSTED
**Time Frame:** Day 17 (on average)
**Title:** Marked Abnormalities in Clinical Laboratory Parameters
**Type:** SECONDARY
**Unit of Measure:** Participants
##### Group
**Description:** Only one subject was included in cohort A; no subject was enrolled in the other cohorts due to early study termination.
**ID:** OG000
**Title:** Part A / Cohorts A to E
##### Group
**Description:** No subject was enrolled in cohort B due to early study termination.
**ID:** OG001
**Title:** Part B / Cadazolid
#### Outcome Measure 13
**Description:** Number of subjects with any treatment-emergent abnormalities in vital signs (up to 7 days after end of treatment)
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B
**Reporting Status:** POSTED
**Time Frame:** Day 17 (on average)
**Title:** Marked Abnormalities in Vital Signs
**Type:** SECONDARY
**Unit of Measure:** Participants
##### Group
**Description:** Only one subject was included in cohort A; no subject was enrolled in the other cohorts due to early study termination.
**ID:** OG000
**Title:** Part A / Cohorts A to E
##### Group
**Description:** No subject was enrolled in cohort B due to early study termination.
**ID:** OG001
**Title:** Part B / Cadazolid
#### Outcome Measure 14
**Description:** Number of subjects with any TEAEs. A TEAE is any adverse event temporally associated with the use of study treatment (from study treatment initiation until 7 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B
**Reporting Status:** POSTED
**Time Frame:** Day 17 (on average)
**Title:** Treatment-emergent Adverse Events (TEAES)
**Type:** SECONDARY
**Unit of Measure:** Participants
##### Group
**Description:** Only one subject was included in cohort A; no subject was enrolled in the other cohorts due to early study termination.
**ID:** OG000
**Title:** Part A / Cohorts A to E
##### Group
**Description:** No subject was enrolled in cohort B due to early study termination.
**ID:** OG001
**Title:** Part B / Cadazolid
### Participant Flow Module
#### Group
**Description:** Subjects from 12 years to 18 years old (exclusive) are to be treated with cadazolid 250 mg twice daily for 10 days. The dose may be adjusted based on the pharmacokinetic (PK) and safety data reviewed for the first 3 subjects.
**ID:** FG000
**Title:** Part A / Cohort A
#### Group
**Description:** Subjects from 6 years to 12 years old (exclusive) are to be treated with cadazolid for 10 days (dose determined based on the PK and safety data from cohort A reviewed by the Independent Data Monitoring Committee (IDMC)).
**ID:** FG001
**Title:** Part A / Cohort B
#### Group
**Description:** Subjects from 2 years to 6 years old (exclusive) are to be treated with cadazolid for 10 days. (dose determined based on the PK and safety data from cohort B reviewed by the IDMC).
**ID:** FG002
**Title:** Part A / Cohort C
#### Group
**Description:** Subjects from 3 months to 2 years old (exclusive) are to be treated with cadazolid for 10 days (dose determined based on the PK and safety data from cohort B reviewed by the IDMC).
**ID:** FG003
**Title:** Part A/ Cohort D
#### Group
**Description:** Subjects from birth to 3 months old (exclusive) are to be treated with cadazolid for 10 days (dose determined based on the PK and safety data from cohort B reviewed by the IDMC). .
**ID:** FG004
**Title:** Part A/ Cohort E
#### Group
**Description:** Subjects from birth to 18 years old (exclusive) are to be treated with cadazolid for 10 days, at the dose defined in the corresponding age cohort in Part A.
**ID:** FG005
**Title:** Part B / Cadazolid
#### Group
**Description:** Subjects from birth to 18 years old (exclusive)are to be treated with vancomycin for 10 days either as capsules (for subjects able to swallow) or oral solution (for the others) .
**ID:** FG006
**Title:** Part B / Vancomycin
#### Period
**Title:** Overall Study
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
**Recruitment Details:** Due to early termination of the study after sponsor's decision to discontinue the development of cadazolid, only one patient was enrolled at one site in the US.
**Has Results:** True