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NCT01519349 · ClinicalTrials.gov registry record · Phase 1
Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
A Phase 1 study, sponsored by Nationwide Children's Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
NCT01519349: Completed Phase 1 study, sponsored by Nationwide Children's Hospital.
NCT01519349 is a Phase 1 study that has completed, run by Nationwide Children's Hospital. The registered enrollment target is 15 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01519349, a Phase 1 study, has completed, sponsored by Nationwide Children's Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
Study Summary
The investigators are performing a gene therapy clinical trial in Becker muscular dystrophy (BMD) and sporadic inclusion body myositis (sIBM) patients. Both of these conditions have an important common feature: loss of ability to walk because of weakness of the thigh muscles. The investigators plan to do a gene therapy trial to deliver a gene to muscle called follistatin (FS344) that can build muscle size and strength. If successful, the investigators can increase the size of the thigh muscle and potentially prolong a patient's ability to walk. The gene will be carried into the muscle by a virus called adeno-associated virus (AAV). This virus occurs naturally in muscle and does not cause any human disease, setting the stage for its safe use in a clinical trial. Presently there is no treatment that can reverse Becker muscular dystrophy or sporadic inclusion body myositis. Only supportive care is currently possible. In this study, subjects with either of these diseases will have shots of the follistatin gene injected directly into thigh muscle on one (first cohort) or both legs (2nd and 3rd cohort). One hundred and eighty days following the gene delivery, the muscle will undergo biopsy to look closely at the muscle to see if the muscle fibers are bigger. Between the time of the gene transfer and the muscle biopsy, patients will be carefully monitored for any side effects of the treatment. This will include an MRI of the thigh muscle before treatment and at day 180 following treatment. Blood and urine tests, as well as physical examination will be done on the subjects during the screening visit and on days 0, 1, 2, 7, 14, 30, 60, 90, and 180 to make sure that there are no side effects from the gene injections. Sutures will be removed 2 weeks post-biopsy. Additional blood samples will be collected at 9, 12, 18, and 24 months. Patients will be seen at the end of 1st and 2nd years for a physical exam, assessment of muscle strength and appropriate blood tests.
Primary Outcome
Safety trial based on development of unacceptable toxicity defined as the occurrence of any Grade III or higher treatment-related toxicities.
Interventions
- BIOLOGICAL rAAV1.CMV.huFollistatin344
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2012-01 |
| Est. Completion | 2017-10 |
| Phase | Phase 1 |
What the finished NCT01519349 record still lists
NCT01519349 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower).
The record links to 0 conditions, and to 1 intervention - of which rAAV1.CMV.huFollistatin344 is the first listed.
NCT01519349 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01519349 about?
NCT01519349 is a clinical study titled "Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis". The investigators are performing a gene therapy clinical trial in Becker muscular dystrophy (BMD) and sporadic inclusion body myositis (sIBM) patients. Both of these conditions have an important common feature: loss of ability to walk because of weakness of the thigh muscles. The investigators plan ...
What is the current status of trial NCT01519349?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2012-01. Estimated completion is 2017-10.
What interventions are being tested in trial NCT01519349?
The interventions under investigation include: rAAV1.CMV.huFollistatin344 (BIOLOGICAL).
Who is sponsoring clinical trial NCT01519349?
This trial is sponsored by Nationwide Children's Hospital, which has 188 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01519349's enrollment target sits among peer trials
15 1798th of 2000 higher than 157 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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