Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy (MoveDMD)
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of CAT-1004 (now known as edasalonexent) in pediatric patients with a genetically confirmed diagnosis of DMD. Approximately 30 ambulatory, male patients from ≥4 to <8 years of age will be enrolled overall.
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.
Part A was initiated in April 2015 and is now complete. All three doses of CAT-1004 tested were generally well tolerated with no safety signals observed. The majority of adverse events were mild, and the most common adverse events were gastrointestinal (primarily diarrhea). There were no serious adverse events and no drug discontinuations.
Part B is 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 may also participate in Part B, along with newly enrolled patients. Following completion of Part B, all patients will receive CAT-1004 for 36 weeks in Part C, the open-label portion of the CAT-1004-201 study.
Muscular Dystrophy, Duchenne
|Study Design:||Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
|Official Title:||A Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular Dystrophy|
- Safety and tolerability (Adverse Events) [ Time Frame: 12 Weeks ]
- Muscle composition and inflammation as measured by MRI [ Time Frame: 12 Weeks ]
- Physical function, muscle strength, and parent/proxy reported physical functioning/quality of life [ Time Frame: 12 Weeks ]
- CAT-1004 PK and PD measures [ Time Frame: 12 Weeks ]
|Study Start Date:||April 2016|
|Estimated Study Completion Date:||September 2017|
|Estimated Primary Completion Date:||January 2017 (Final data collection date for primary outcome measure)|
Experimental: Cohort B1
Experimental: Cohort B2
Placebo Comparator: Cohort B3
Placebo Dose 1 or 2
Please refer to this study by its ClinicalTrials.gov identifier: NCT02439216
|United States, California|
|Los Angeles, California, United States, 90095|
|United States, Florida|
|Gainesville, Florida, United States, 32610|
|Orlando, Florida, United States, 32827|
|United States, Oregon|
|Portland, Oregon, United States, 97239|
|United States, Pennsylvania|
|Philadelphia, Pennsylvania, United States, 19104|