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Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy (MoveDMD®)

Primary Purpose

Muscular Dystrophy, Duchenne

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Edasalonexent
Placebo
Sponsored by
Catabasis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Muscular Dystrophies, Musculoskeletal Diseases, Nervous System Diseases, Neuromuscular Diseases, Duchenne muscular dystrophy, DMD, dystrophin, dystrophy, Duchenne

Eligibility Criteria

4 Years - 7 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Written informed consent from parent or legal guardian prior to participation and, for patients who are 7 years of age, written assent from patient
  • Diagnosis of DMD based on a clinical phenotype with increased serum CK and the presence of a mutation in the dystrophin gene known to be associated with a DMD phenotype
  • Ability to walk independently (assistive devices are permitted)
  • Adequate immunization for influenza and varicella

Exclusion Criteria:

  • Use of corticosteroids within prior 6 months of treatment initiation or planning to initiate steroid therapy within the next 6 months
  • Other prior or ongoing significant medical conditions

  • Exposure to another investigational drug (such as eteplirsen or idebenone) within 28 days prior to start of study treatment or ongoing participation in any other therapeutic clinical trial

    • Note: There are separate criteria for patients who participated in Part A versus newly enrolling patients. New patients must meet all of the Part A entry criteria to participate in Part B.

Patients who participated in Part A must meet the following criteria to participate in Part B:

  • Completed Part A
  • Continue to meet all of the Part A entry criteria, including an absence of safety concerns (however, patients may be ≥8 years of age)

There are no entry criteria for Part C; all patients who complete Part B will automatically continue in Part C

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Dose 1

Dose 2

Placebo

Arm Description

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Matching placebo

Outcomes

Primary Outcome Measures

Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B
The LLC5-T2 calculated from the unweighted average of the T2 relaxation times of all 5 lower leg muscles for each patient at each evaluation (the medial gastrocnemius, peroneals, soleus, tibialis anterior, and tibialis posterior muscles). Increases in LLC5-T2 relaxation time indicate muscle damage, inflammation, edema, and fat infiltration and are highly correlated with muscle fat

Secondary Outcome Measures

Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C
The 10MWT determines the speed to walk a distance of 10 meters. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C
The 4-stair climb test determines the speed to climb 4 standard steps. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C
The stand from supine test determines the speed to stand from a supine position. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
A TEAE was any adverse event (AE) that started during or after the first dose of IP through the end of the safety follow-up period. Part B TEAEs were those that started on or after the first dose date in Part B through the date of Week 12 clinic dose. TEAEs for the Part C (Active B or C) analysis were those that started on or after the first dose date of active treatment in Part B or Part C. Drug related AEs included those marked as "Related" or "Possibly Related" to the study treatment.

Full Information

First Posted
April 29, 2015
Last Updated
September 20, 2022
Sponsor
Catabasis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02439216
Brief Title
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
Acronym
MoveDMD®
Official Title
A Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
September 2022
Overall Recruitment Status
Completed
Study Start Date
April 2016 (undefined)
Primary Completion Date
January 12, 2017 (Actual)
Study Completion Date
August 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Catabasis Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from ≥4 to <8 years of age will be enrolled. Edasalonexent is an orally administered small molecule targeted to inhibit activated NF-κB, a molecule that is activated from infancy in DMD and which is central to causing muscle damage and preventing muscle regeneration. Data on magnetic resonance imaging of the lower and upper leg muscles, physical function (including timed function tests) and muscle strength will be studied.
Detailed Description
Part A was a 1-week, open-label study to assess safety, tolerability, pharmacokinetics and biomarkers for three dose levels of edasalonexent and is now complete. Part B was a randomized, double-blind, placebo-controlled, multiple dose study to evaluate the safety, efficacy, PK, and PD of edasalonexent over 12 weeks. Patients who participated in Part A also participated in Part B, along with newly enrolled patients. Patients received either edasalonexent 67 mg/kg/day, edasalonexent 100 mg/kg/day, or placebo in Part B. Part B is now complete. Following completion of Part B, patients receive edasalonexent for 138 weeks in Part C, the open-label portion of the MoveDMD study. Patients on the 67 mg/kg/day treatment moved to the 100 mg/kg/day treatment. Patients on the 100 mg/kg/day treatment remained on the 100 mg/kg/day treatment. If clinically indicated, concomitant treatment with eteplirsen (Exondys 51™) may be acceptable in patients with amenable gene mutations during Part C after the patient has been exposed to edasalonexent for 6 months. **Following completion of MoveDMD Part C, access to edasalonexent for trial participants will continue through the open-label extension study, GalaxyDMD.**

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Duchenne
Keywords
Muscular Dystrophies, Musculoskeletal Diseases, Nervous System Diseases, Neuromuscular Diseases, Duchenne muscular dystrophy, DMD, dystrophin, dystrophy, Duchenne

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
31 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Dose 1
Arm Type
Experimental
Arm Description
Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day
Arm Title
Dose 2
Arm Type
Experimental
Arm Description
Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Matching placebo
Intervention Type
Drug
Intervention Name(s)
Edasalonexent
Other Intervention Name(s)
CAT-1004, Edasa
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B
Description
The LLC5-T2 calculated from the unweighted average of the T2 relaxation times of all 5 lower leg muscles for each patient at each evaluation (the medial gastrocnemius, peroneals, soleus, tibialis anterior, and tibialis posterior muscles). Increases in LLC5-T2 relaxation time indicate muscle damage, inflammation, edema, and fat infiltration and are highly correlated with muscle fat
Time Frame
Baseline to Week 12
Secondary Outcome Measure Information:
Title
Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C
Description
The 10MWT determines the speed to walk a distance of 10 meters. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Time Frame
Baseline to Week 12
Title
Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C
Description
The 4-stair climb test determines the speed to climb 4 standard steps. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Time Frame
Baseline to Week 12
Title
Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C
Description
The stand from supine test determines the speed to stand from a supine position. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Time Frame
Baseline to Week 12
Title
Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
Description
A TEAE was any adverse event (AE) that started during or after the first dose of IP through the end of the safety follow-up period. Part B TEAEs were those that started on or after the first dose date in Part B through the date of Week 12 clinic dose. TEAEs for the Part C (Active B or C) analysis were those that started on or after the first dose date of active treatment in Part B or Part C. Drug related AEs included those marked as "Related" or "Possibly Related" to the study treatment.
Time Frame
Screening to Week 152

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Written informed consent from parent or legal guardian prior to participation and, for patients who are 7 years of age, written assent from patient Diagnosis of DMD based on a clinical phenotype with increased serum CK and the presence of a mutation in the dystrophin gene known to be associated with a DMD phenotype Ability to walk independently (assistive devices are permitted) Adequate immunization for influenza and varicella Exclusion Criteria: Use of corticosteroids within prior 6 months of treatment initiation or planning to initiate steroid therapy within the next 6 months Other prior or ongoing significant medical conditions Exposure to another investigational drug (such as eteplirsen or idebenone) within 28 days prior to start of study treatment or ongoing participation in any other therapeutic clinical trial Note: There are separate criteria for patients who participated in Part A versus newly enrolling patients. New patients must meet all of the Part A entry criteria to participate in Part B. Patients who participated in Part A must meet the following criteria to participate in Part B: Completed Part A Continue to meet all of the Part A entry criteria, including an absence of safety concerns (however, patients may be ≥8 years of age) There are no entry criteria for Part C; all patients who complete Part B will automatically continue in Part C
Facility Information:
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
City
Orlando
State/Province
Florida
ZIP/Postal Code
32827
Country
United States
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

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