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Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
study id #: NCT02439216
condition: Muscular Dystrophy, Duchenne
status: active, not recruitingpurpose:
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.
intervention: Edasalonexent, Placebo
mechanism of action: NF-KB inhibitor to prevent muscle damage and promote muscle regeneration
start date: April 2016
estimated completion: January 12, 2017
phase of development: Phase 1/Phase 2
size / enrollment: 31
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 112 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.
- 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]
• 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
• 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 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
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