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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: |
NCT01540409 |
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Date of registration:
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23/02/2012 |
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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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Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
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Scientific title:
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Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201 |
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Date of first enrolment:
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February 27, 2012 |
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Target sample size:
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12 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01540409 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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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:
A subject must meet all of the following criteria to be eligible for this study.
1. The subject and/or their parent/legal guardian are willing and able to provide signed
informed consent.
2. The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
3. The subject has a parent(s) or legal guardian(s) who is able to understand and comply
with all of the study procedure requirements.
Exclusion Criteria:
A subject who meets any of the following criteria will be excluded from this study.
1. The subject has a prior or ongoing medical condition that, in the Investigator's
opinion, could adversely affect the safety of the subject or make it unlikely that the
course of treatment or follow-up would be completed or impair the assessment of study
results.
Age minimum:
7 Years
Age maximum:
13 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: AVI-4658 (Eteplirsen)
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Primary Outcome(s)
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Change From Baseline in the Percentage of Dystrophin Positive Fibers (PDPF) at Week 48
[Time Frame: Parent Baseline and Week 48]
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Change From Baseline in the 6 Minute Walk Test (6MWT) at Week 240
[Time Frame: Parent Baseline and Week 240]
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Secondary ID(s)
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4658-us-202
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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