This is a registry record, not a result. A trial being listed, recruiting or completed says nothing by itself about whether the compound works or is safe.
| Registry number | NCT02354781 |
|---|---|
| Status | Completed |
| Phase | Phase 1 / Phase 2 |
| Sponsor | Jerry R. Mendell |
| Enrollment | 3 participants |
| Design | Interventional, Na, Single Group, Masking: None |
| Conditions | Duchenne Muscular Dystrophy |
| Interventions | rAAV1.CMV.huFollistin344 |
| Start | Jan 2015 |
| Primary completion | Nov 2017 |
| Study completion | Nov 2017 |
| Results posted | Yes, in the registry |
| Registry last updated | Oct 11, 2023 |
| Collaborators | Duchenne Alliance; Milo Therapeutics |
Compounds on this site that cite this trial
What the registry says the trial is about
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.
Primary outcomes
These are what the trial was designed to measure first. They are registered in advance, which is what makes a later result checkable.
| Outcome measure | Time frame |
|---|---|
| Number of Dose Limiting Toxicity (DLT) Adverse Events as Assessed by 21 CFR 312.32. | DLT Adverse events will be recorded from the date of dosing and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of dosing and for up to 2 years after gene therapy administration. |
3 secondary outcomes also registered. See the registry record for the full list.
Source: ClinicalTrials.gov (U.S. National Library of Medicine). Last checked against the registry on Oct 3, 2026. See the trial changes feed for what has changed recently.
