Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2019-08-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Catabasis Pharmaceuticals

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