Clinical trial
Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular Dystrophy
Name
AAV9 Dup2 U7
Description
Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
Trial arms
Trial start
2020-01-15
Estimated PCD
2023-11-19
Trial end
2025-11-19
Status
Active (not recruiting)
Phase
Early phase I
Treatment
scAAV9.U7.ACCA
A single dose of scAAV9.U7.ACCA will be systemically delivered via a peripheral vein injection.
Arms:
Cohort 1 (Minimal Efficacious Dose)
Size
3
Primary endpoint
Monitoring for the development of unacceptable toxicity.
2 years
Eligibility criteria
Inclusion Criteria:
* Age greater than 6 months and less than 14 years
* Confirmed duplication of exon 2 in the DMD gene using a clinically accepted technique that completely defines the mutation
* Pre-ambulant (not yet walking) or ambulant (as defined by the ability to walk 10 meters without assistance)
* Males of any ethnic group will be eligible
* Ability to cooperate with muscle testing
* In subjects age 4 and above, stable dose and regimen of corticosteroid therapy (prednisone, deflazacort, or their generic forms) for at least 12 weeks prior to gene transfer.
Exclusion Criteria:
* Active viral infection based on clinical observations
* Symptoms or signs of cardiomyopathy, including:
1. Dyspnea on exertion, pedal edema, shortness of breath upon lying flat, or rales at the base of the lungs
2. 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 SI creates unnecessary risks for gene transfer
* AAV9 binding antibody titers ≥ 1:400 as determined by ELISA immunoassay
* Abnormal laboratory values in the clinically significant range as listed in Table 7, based upon normal values in the Nationwide Children's Hospital Laboratory.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'This trial will deliver the minimal efficacious dose as determined by preclinical studies and approved by the FDA to determine safety and target engagement.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 3, 'type': 'ACTUAL'}}
Updated at
2023-02-09
1 organization
1 product
1 indication
Organization
Megan WaldropProduct
scAAV9.U7.ACCAIndication
Duchenne muscular dystrophy