Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy (MoveDMD®)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02439216|
Recruitment Status : Active, not recruiting
First Posted : May 8, 2015
Last Update Posted : October 23, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Drug: Edasalonexent Drug: Placebo||Phase 1 Phase 2|
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 will continue through a new open-label extension study. Families currently enrolled in MoveDMD Part C are encouraged to speak with their trial site for additional information.**
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||31 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||A Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular Dystrophy|
|Study Start Date :||April 2016|
|Actual Primary Completion Date :||January 12, 2017|
|Estimated Study Completion Date :||August 2019|
Experimental: Dose 1
Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day
Experimental: Dose 2
Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day
Placebo Comparator: Placebo
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|