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rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies

NCT02710500 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2021-05-13

No results posted yet for this study

Summary

The proposed clinical trial is a double-blind, randomized controlled study with direct intramuscular injection of rAAVrh.74.MHCK7.DYSF.DV gene vector to the extensor digitorum brevis muscle (EDB). Two cohorts of subjects with dysferlin deficiency, each with proven mutations will undergo gene transfer. A minimum of three subjects will be enrolled into each cohort.

Conditions

  • Dysferlinopathy

Interventions

DRUG

rAAVrh74.MHCK7.DYSF.DV

Biological/Vaccine: rAAVrh74.MHCK7.DYSF.DV Recombinant adeno-associated virus carrying a dysferlin transgene under control of a muscle specific MHCK7 promoter.

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Jerry R Mendell, MD · Director, Center for Gene Therapy

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-03-31
Primary Completion
2019-07-31
Completion
2019-07-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02710500 on ClinicalTrials.gov