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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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26 September 2023 |
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Main ID: |
NCT03333590 |
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Date of registration:
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01/11/2017 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
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Scientific title:
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Phase I/IIa Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 |
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Date of first enrolment:
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November 6, 2017 |
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Target sample size:
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2 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT03333590 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Kevin Flanigan, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Nationwide Children's Hospital |
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Key inclusion & exclusion criteria
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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
Age minimum:
4 Years
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Biological: rAAVrh74.MCK.GALGT2
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Primary Outcome(s)
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Number of Unanticipated Grade III or Higher Treatment-Related Toxicities
[Time Frame: 2 years]
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Secondary Outcome(s)
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Expression of GALGT2 as Demonstrated by Immunofluorescent Staining With Anti-CT Epitope Antibodies or WFA Lectin in Muscle Biopsy Sections at 120 Days Post Injection (Cohort 1) and 90 Days Post-injection (Cohort 2).
[Time Frame: Day 90 (Cohort 2) and Day 120 (Cohort 1)]
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GALGT2 Protein Expression Quantified by Western Blot and Assessed by Densitometry in Muscle Biopsy Tissue at 120 Days Post-injection (Cohort 1) and 90 Days Post-injection (Cohort 2)
[Time Frame: Day 90 (Cohort 2) and Day 120 (Cohort 1)]
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Secondary ID(s)
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GALGT2 Gene Therapy for DMD
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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