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 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.
|Muscular Dystrophy, Duchenne||Drug: Edasalonexent Drug: Placebo||Phase 1 Phase 2|
|Study Design:||Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Participant, Care Provider, 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 ]
- Edasalonexent PK and PD measures [ Time Frame: 12 Weeks ]
|Study Start Date:||April 2016|
|Estimated Study Completion Date:||March 2018|
|Primary Completion Date:||January 2017 (Final data collection date for primary outcome measure)|
Experimental: Cohort B1
Edasalonexent 67 mg/kg/day
Other Name: CAT-1004
Experimental: Cohort B2
Edasalonexent 100 mg/kg/day
Other Name: CAT-1004
|Placebo Comparator: Cohort B3||Drug: Placebo|
Part A is now complete. All three doses of edasalonexent 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 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 and safety signals were not observed in either treatment group.
Following completion of Part B, patients receive edasalonexent for 60 weeks in Part C, the open-label portion of the MoveDMD study. Based on the results from Part B of the study, patients on the 67 mg/kg/day treatment will be switched to the 100 mg/kg/day treatment for the remainder of Part C. Patients on the 100 mg/kg/day treatment will remain on the 100 mg/kg/day treatment for the remainder of Part C.
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|