Trials / Completed
CompletedNCT02354781
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
Phase I/II Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 3 (actual)
- Sponsor
- Jerry R. Mendell · Academic / Other
- Sex
- Male
- Age
- 7 Years
- Healthy volunteers
- Not accepted
Summary
The proposed clinical trial is an outgrowth of the safety record and functional improvement seen in the BMD follistatin gene therapy trial. In this study the investigators propose to inject AAV1.CMV.huFS344 at a total dose of 2.4E12 vg/kg to six DMD patients. This dose will be divided between gluteal muscles, quadriceps and tibialis anterior. This is a wider distribution of vector than given to BMD patients, who overall improved the distance walked on the 6MWT without adverse events related to viral transduction into a single muscle.
Detailed description
The primary objective of this study is safety and endpoints will include hematology, serum chemistry, urinalysis, immunologic response to rAAV1 and follistatin, and reported history and observations of symptoms. Efficacy measures will be used as secondary outcomes and include the distance walked on the 6MWT, functional tests by PT, life quality questionnaire, MRI, EIM, and muscle biopsy. Subject will have follow up visits on days 7, 14, 30, 45, 60, 90, 180 and 9,12, 18 and 24 months post-gene transfer.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | rAAV1.CMV.huFollistin344 | Six DMD patients will receive rAAV1.CMV.huFollistatin344 to both limbs by multiple injections to gluteal muscles, quadriceps and tibialis anterior muscles. |
Timeline
- Start date
- 2015-01-01
- Primary completion
- 2017-11-01
- Completion
- 2017-11-01
- First posted
- 2015-02-03
- Last updated
- 2023-10-11
- Results posted
- 2020-04-15
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT02354781. Inclusion in this directory is not an endorsement.