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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02255552 |
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Date of registration:
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25/09/2014 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Study of Eteplirsen in DMD Patients
PROMOVI |
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Scientific title:
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An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy |
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Date of first enrolment:
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November 17, 2014 |
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Target sample size:
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109 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02255552 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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United States
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Contacts
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Name:
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Medical Director |
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Address:
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Telephone:
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Email:
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Affiliation:
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Sarepta Therapeutics, Inc. |
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Key inclusion & exclusion criteria
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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.
Age minimum:
7 Years
Age maximum:
16 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy (DMD)
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Intervention(s)
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Drug: eteplirsen
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Primary Outcome(s)
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Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96
[Time Frame: Baseline, Week 96]
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Secondary Outcome(s)
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Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96
[Time Frame: Baseline, Week 96]
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Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96
[Time Frame: Baseline, Week 96]
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Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96
[Time Frame: Baseline, Week 96]
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Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96
[Time Frame: Baseline, Week 96]
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Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96
[Time Frame: Week 96]
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Number of Participants Who Lost Ambulation (LOA) by Week 96
[Time Frame: Up to Week 96]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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