Clinical trial NCT02439216: Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
Official title: A Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Muscular Dystrophy, Duchenne
Keywords: Muscular Dystrophies
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases
Duchenne muscular dystrophy
DMD
dystrophin
dystrophy
Duchenne
Lead sponsor: Catabasis Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2015-05-08
Enrollment: 31
Sex: MALE
Minimum age (years): 4
Maximum age (years): 7
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.\*\*

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

Primary outcomes:
measure: Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B
timeFrame: Baseline to Week 12
Secondary outcomes:
measure: Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C
timeFrame: Baseline to Week 12
measure: Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C
timeFrame: Baseline to Week 12
measure: Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C
timeFrame: Baseline to Week 12
measure: Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
timeFrame: Screening to Week 152
Other outcomes:
Locations:
city: Los Angeles
country: United States
state: California
city: Gainesville
country: United States
state: Florida
city: Orlando
country: United States
state: Florida
city: Portland
country: United States
state: Oregon
city: Philadelphia
country: United States
state: Pennsylvania
Study officials:
References: