Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2

Withdrawn before enrolling · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients that will involve direct intramuscular injection to the extensor digitorum brevis muscle (EDB).

Key facts

Study ID

NCT02704325

Run by

Kevin Flanigan

People needed

0

Starts

2016-04-01

Expected to finish

2020-02-01

Last updated by the study team

2018-02-06

Who can join

Age: 9 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Nonambulant subjects, age 9 or older
• Confirmed mutation in the DMD gene using a clinically accepted technique that completely defines the mutation
• A magnetic resonance image of the EDB showing preservation of sufficient muscle mass to permit transfection
• Males of any ethnic group will be eligible
• Ability to cooperate with all study procedures
• Willingness of sexually active subjects with reproductive capacity to practice reliable method of contraception (If appropriate).
• Stable dose of corticosteroid therapy (including either prednisone or deflazacort and their generic forms) for 12 weeks prior to gene transfer

You may not qualify if…

• Active viral infection based on clinical observations.
• The presence of a DMD mutation without weakness or loss of function
• 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
• Persistent leukopenia or leukocytosis (WBC ≤ 3.5 K/µL or ≥ 20.0 K/µL) or an absolute neutrophil count < 1.5K/µL
• Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
• Subjects with rAAVrh74 binding antibody titers ≥ 1:400 as determined by ELISA immunoassay
• Presence of circulating anti-Sda antibodies as determined by study approved laboratory.
• Abnormal laboratory values in the clinically significant range, 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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