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Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy

NCT02376816 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2017-11-24

No results posted yet for this study

Summary

The proposed phase I clinical trial is a pilot study to evaluate safety and biological activity of the rAAVrh74.MCK.micro-Dystrophin vector administered by an intramuscular route. This study will evaluated the micro-Dystrophin vector as a potential dystrophin replacement mechanism for Duchenne Muscular Dystrophy. Two cohorts will undergo gene transfer in a standard three-six dose escalation scheme to establish maximum tolerated dose (MTD) using toxicity. A minimum of three subjects will be enrolled into each cohort. The first cohort will receive a total dose of 3E11 vg. The second cohort will receive 1E12 vg total dose.

Conditions

Interventions

BIOLOGICAL

rAAVrh74.MCK.micro-Dystrophin

Recombinant adeno-associated virus carrying a truncated "micro" dystrophin transgene under control of a muscle specific MCK promoter.

Sponsors & Collaborators

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

    collaborator NIH

  • Jerry R. Mendell

    lead OTHER

Principal Investigators

  • Jerry R Mendell, MD · Nationwide Children's Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SINGLE_GROUP

Eligibility

Min Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-03-31
Primary Completion
2017-09-30
Completion
2017-09-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

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 NCT02376816 on ClinicalTrials.gov