Status:
COMPLETED
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
Lead Sponsor:
Kevin Flanigan
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
4+ years
Phase:
PHASE1
PHASE2
Brief Summary
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients. There will be a modified intravascular limb infusion (ILI) procedure that will be used to seque...
Detailed Description
This is an open-label, dose escalation trial where the vector will be delivered via the femoral artery to the muscles of both legs of DMD subjects. The primary objective of this study is the assessme...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- Ambulant patients age 4 years or older
- Confirmed mutations in the DMD gene using a clinical accepted technique that completely defines the mutation 1,2
- • Measurably impaired muscle function (defined as less than 80% of the predicted value for 100 MWT), but with sufficient muscle preservation to ensure assessment of muscle transfection based on clinical evaluation by the PI and expert colleagues. This degree of preservation will include:
- Ability to extend the knee fully against gravity
- Preserved ambulation with ability to walk ≥ 350 meters during the 6MWT
- A magnetic resonance image of the quadriceps showing preservation of sufficient muscle mass to permit transfection
- Males of any ethnic group will be eligible
- Ability to cooperate with muscle testing
- Stable daily dose of corticosteroid therapy (including either prednisone, prednisolone, deflazacort or their generic forms) for 12 weeks prior to gene transfer
- Exclusion Criteria
- Active viral infection based on clinical observations
- The presence of a DMD mutation without weakness or loss of function
- Subject is amenable to or is currently being treated with eteplirsen
- 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 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 ≥ 1:50 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
Exclusion
Key Trial Info
Start Date :
November 6 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 31 2023
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT03333590
Start Date
November 6 2017
End Date
December 31 2023
Last Update
March 13 2025
Active Locations (1)
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1
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205