Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)
The main objective of this study is to provide confirmatory evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Sites are currently being initiated into the study. Initiation of approximately 39 planned sites in the United States is expected to be completed by June 2016. The initiated sites can be found in the "Contacts and Locations" section of this posting in addition to a listing of the city and states of sites the investigators are working to initiate. This information will be updated on a rolling basis as additional sites are initiated.
|Study Design:||Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: No masking
Primary Purpose: Treatment
|Official Title:||An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy|
- Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ]
- The percentage of dystrophin-positive fibers [ Time Frame: Baseline, Weeks 24, 48, 72, 96 ]
- Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Baseline,Weeks 12, 24, 36, 48 ]
- Evaluate the long-term effects of eteplirsen up to 96 weeks [ Time Frame: Week 1-96 ]
|Study Start Date:||September 2014|
|Estimated Study Completion Date:||May 2019|
|Estimated Primary Completion Date:||January 2019 (Final data collection date for primary outcome measure)|
Experimental: Treated Group
Approximately 80 patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping will receive 30 mg/kg of eteplirsen weekly for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
No Intervention: Untreated Group
Approximately 80 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.
This is an open-label, multi-center study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group). Following primary efficacy endpoints, dosing will continue to week 144 to evaluate the long term effects of eteplirsen.
Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg Eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks). Patients in the untreated group will not receive treatment.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.
Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.
The sponsor is working to initiate approximately 39 sites across the United States. Sites will vary in the following functions:
- Local Site (N=39) - Enrolls patients and is the primary contact point for their patients. Sites will perform all protocol activities (including dosing and laboratory assessments), except for functional assessments and biopsies.
- Hub Site (N=14) - Performs functional (physical) assessments at specified times per protocol.
- Surgical Site (N=2) - Performs muscle biopsies for the Treated group.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02255552
Show 36 Study Locations
|Study Chair:||Catherine Stehman-Breen, MD||Sarepta Therapeutics|