Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy
Completed · Phase 1
Conditions studied: Duchenne Muscular Dystrophy
In brief
The proposed phase I clinical trial is a pilot study to evaluate safety and biological activity of the rAAVrh74.MCK.micro-Dystrophin vector administered by an intramuscular route. This study will evaluated the micro-Dystrophin vector as a potential dystrophin replacement mechanism for Duchenne Muscular Dystrophy. Two cohorts will undergo gene transfer in a standard three-six dose escalation scheme to establish maximum tolerated dose (MTD) using toxicity. A minimum of three subjects will be enrolled into each cohort. The first cohort will receive a total dose of 3E11 vg. The second cohort will receive 1E12 vg total dose.
Key facts
- Study ID
- NCT02376816
- Run by
- Jerry R. Mendell
- People needed
- 2
- Starts
- 2015-03-01
- Expected to finish
- 2017-09-01
- Last updated by the study team
- 2017-11-24
Who can join
Age: 7 and older. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- Age 7 or older; must be wheelchair-dependent
- Confirmed Dystrophin mutations based on mutation compatibility with micro-dys cDNA based on previously published methods.
- Males of any ethnic group will be eligible.
- Ability to cooperate with muscle testing.
- Willingness of sexually active subjects with reproductive capacity to practice reliable method of contraception (If appropriate).
You may not qualify if…
- Active viral infection based on clinical observations.
- Symptoms or signs of cardiomyopathy, including:
- Dyspnea on exertion, pedal edema, shortness of breath upon lying flat, or rales at the base of the lungs
- Echocardiogram with ejection fraction below 40%
- Serological evidence of HIV infection, or Hepatitis A, B or C infection
- Diagnosis of (or ongoing treatment for) an autoimmune disease
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer.
- Subjects with AAVrh74 binding antibody titers ≥ 1:50 as determined by ELISA immunoassay.
- Abnormal laboratory values in the clinically significant range as defined in protocol or based upon normal values in the Nationwide Children's Hospital Laboratory.
Where it is running
- Nationwide Children's Hospital — Columbus, Ohio, United States
Full record on ClinicalTrials.gov
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