Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)
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|ClinicalTrials.gov Identifier: NCT02255552|
Recruitment Status : Active, not recruiting
First Posted : October 2, 2014
Last Update Posted : May 18, 2018
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy (DMD)||Drug: eteplirsen||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||110 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||September 2014|
|Estimated Primary Completion Date :||May 2019|
|Estimated Study Completion Date :||February 2020|
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 30 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.
- Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Change from Baseline to Week 96 ]
- The percentage of dystrophin-positive fibers [ Time Frame: Change from Baseline ]
- Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Change from Baseline ]
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): NCT02255552
Show 37 Study Locations
|Study Chair:||Chris Mix, MD||Sarepta Therapeutics|
|Study Chair:||Catherine Stehman-Breen, MD, MS||Sarepta Therapeutics|