Study of Eteplirsen in DMD Patients (PROMOVI)
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| ClinicalTrials.gov Identifier: NCT02255552 |
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Recruitment Status :
Active, not recruiting
First Posted : October 2, 2014
Last Update Posted : July 6, 2018
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Sponsor:
Sarepta Therapeutics
Information provided by (Responsible Party):
Sarepta Therapeutics
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Brief Summary:
The main objective of this study is to provide 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).
| 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 |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| 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 |
| Study Start Date : | September 2014 |
| Estimated Primary Completion Date : | May 2019 |
| Estimated Study Completion Date : | February 2020 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Duchenne and Becker muscular dystrophy
MedlinePlus related topics:
Muscular Dystrophy
Drug Information available for:
Eteplirsen
| Arm | Intervention/treatment |
|---|---|
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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).
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Drug: eteplirsen
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).
Other Names:
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No Intervention: Untreated Group
Approximately 30 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.
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Primary Outcome Measures :
- Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Change from Baseline to Week 96 ]
Secondary Outcome Measures :
- 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 ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 7 Years to 16 Years (Child) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Male 7-16 years old
- Diagnosed with DMD, genotypically confirmed
- Stable dose of corticosteroids for at least 24 weeks
- Have intact right and left alternative upper muscle groups
- Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
- Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%
Exclusion Criteria:
- Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months
- Participation in any other DMD interventional clinical study within 12 weeks
- Major surgery within 3 months
- Presence of other clinically significant illness
- Major change in the physical therapy regime within 3 months
Other inclusion/exclusion criteria apply.
Information from the National Library of Medicine
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
Hide Study Locations
Locations
| United States, Arizona | |
| Neuromuscular Research Center | |
| Phoenix, Arizona, United States, 85028 | |
| United States, California | |
| David Geffen School of Medicine at UCLA | |
| Los Angeles, California, United States, 90095 | |
| University of California, Davis Medical Center | |
| Sacramento, California, United States, 95817 | |
| Rady Children's Hospital, U.C. San Diego | |
| San Diego, California, United States, 92130 | |
| Stanford University School of Medicine/Medical Center | |
| Stanford, California, United States, 94305 | |
| United States, Colorado | |
| Children's Hospital Colorado | |
| Aurora, Colorado, United States, 80045 | |
| United States, Connecticut | |
| Connecticut Children's Medical Center | |
| Hartford, Connecticut, United States, 06106 | |
| United States, District of Columbia | |
| Children's National Health System | |
| Washington, District of Columbia, United States, 20010 | |
| United States, Florida | |
| The University of Florida, Powell Gene Therapy Center | |
| Gainesville, Florida, United States, 32610 | |
| NW FL Clinical Research Group, LLC | |
| Gulf Breeze, Florida, United States, 32561 | |
| Nemours Children's Hospital | |
| Orlando, Florida, United States, 32827 | |
| United States, Georgia | |
| Rare Disease Research Center | |
| Atlanta, Georgia, United States, 30318 | |
| Emory University | |
| Atlanta, Georgia, United States, 30324 | |
| United States, Illinois | |
| Ann and Robert H. Lurie Children's Hospital of Chicago | |
| Chicago, Illinois, United States, 60611 | |
| United States, Iowa | |
| University of Iowa Children's Hospital | |
| Iowa City, Iowa, United States, 52242 | |
| United States, Kansas | |
| University of Kansas Medical Center | |
| Kansas City, Kansas, United States, 66160 | |
| United States, Maryland | |
| Kennedy Krieger Institute | |
| Baltimore, Maryland, United States, 21205 | |
| United States, Massachusetts | |
| Massachusetts General Hospital | |
| Boston, Massachusetts, United States, 02114 | |
| Children's Hospital Boston | |
| Boston, Massachusetts, United States, 02115 | |
| United States, Michigan | |
| Children's Hospital of Michigan | |
| Detroit, Michigan, United States, 48201 | |
| United States, Minnesota | |
| University of Minnesota | |
| Minneapolis, Minnesota, United States, 55455 | |
| United States, Missouri | |
| St. Louis Children's Hospital | |
| Saint Louis, Missouri, United States, 63110 | |
| United States, New Hampshire | |
| Dartmouth-Hitchcock Medical Center | |
| Lebanon, New Hampshire, United States, 03756 | |
| United States, New York | |
| Columbia University Medical Center | |
| New York, New York, United States, 10032 | |
| University of Rochester Clinical Research Center | |
| Rochester, New York, United States, 14642 | |
| United States, North Carolina | |
| Levine Childrens Hospital, Carolinas Medical Center | |
| Charlotte, North Carolina, United States, 28207 | |
| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center (CCHMC) | |
| Cincinnati, Ohio, United States, 45229 | |
| Nationwide Children's Hospital | |
| Columbus, Ohio, United States, 43205 | |
| United States, Oregon | |
| Shriners Hospital for Children | |
| Portland, Oregon, United States, 97239 | |
| United States, Pennsylvania | |
| Penn State Hershey Medical Center | |
| Hershey, Pennsylvania, United States, 17033 | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Children's Hospital of Pittsburgh of UPMC | |
| Pittsburgh, Pennsylvania, United States, 15224 | |
| United States, Tennessee | |
| Vanderbilt University Medical Center | |
| Nashville, Tennessee, United States, 37232 | |
| United States, Texas | |
| The University of Texas Southwestern Medical Center | |
| Dallas, Texas, United States, 75390 | |
| Texas Children's Hospital | |
| Houston, Texas, United States, 77030 | |
| United States, Utah | |
| University of Utah | |
| Salt Lake City, Utah, United States, 84132 | |
| United States, Washington | |
| Seattle Children's Hospital | |
| Seattle, Washington, United States, 98105 | |
Sponsors and Collaborators
Sarepta Therapeutics
Investigators
| Study Chair: | Paul Korner, MD, MBA | Sarepta Therapeutics | |
| Study Chair: | Catherine Stehman-Breen, MD, MS | Sarepta Therapeutics |
| Responsible Party: | Sarepta Therapeutics |
| ClinicalTrials.gov Identifier: | NCT02255552 History of Changes |
| Other Study ID Numbers: |
4658-301 |
| First Posted: | October 2, 2014 Key Record Dates |
| Last Update Posted: | July 6, 2018 |
| Last Verified: | July 2018 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
Keywords provided by Sarepta Therapeutics:
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DMD, Duchenne, Eteplirsen, dystrophy, dystrophin, exon 51 |
Additional relevant MeSH terms:
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Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |