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Gene Therapy Trial Report

Summary

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.


NCTID NCT04240314 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name AT702
Compound Description scAAV9.U7.ACCA
Sponsor Megan Waldrop
Funder Type Other
Recruitment Status
Completed
Enrollment Count 3 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant DMD (exon 2)
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Exon skipping/splice editor
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Dose 1 3E13 vg/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-01-22
Completion Date 2025-07-01
Last Update 2025-09-11

Participation Criteria


Eligible Age 6 Months - 13 Years
Standard Ages Child
Sexes Eligible for Study MALE
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Product was licensed to Audentes/Astellas Therapeutics in 2019, discontinuation was announced in April 2022

Resources/Links