Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-03-31

Study Completion Date

2010-07-31

Brief Summary

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The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).

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Detailed Description

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This phase I randomized double blind dose escalation study investigates the safety and efficacy of the mini-dystrophin gene transferred to the biceps muscle for Duchenne muscular dystrophy patients, ages 5 to 12 years of age, using a recombinant adeno-associated virus. Eligible participants must have a known dystrophin gene mutation and may be concurrently treated with corticoid steroids. The mini-dystrophin gene or a placebo agent (normal saline or empty viral capsids) are injected directly into both biceps muscles while under conscious sedation. Following the gene transfer, patients are admitted to the hospital for 48 hours of observation followed by weekly outpatient visits at the Columbus Children's Hospital Neuromuscular Clinic. A bilateral muscle biopsy is preformed following 6 weeks with long term follow up will consisting of bi-annual visits for the next 2 years.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Low Dose

Low dose cohort - 2.0E10 vg/kg

Group Type EXPERIMENTAL

rAAV2.5-CMV-minidystrophin (d3990)

Intervention Type BIOLOGICAL

Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.

High Dose

High Dose - 1.0E11 vg/kg

Group Type EXPERIMENTAL

rAAV2.5-CMV-minidystrophin (d3990)

Intervention Type BIOLOGICAL

Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.

Interventions

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rAAV2.5-CMV-minidystrophin (d3990)

Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Known null mutation of the Dystrophin gene
* Male age of 5 years or older
* If taking corticosteroids, must have dose unchanged for the past 3 months
* Serum creatine kinase elevation greater than 10x normal value (established by Children's Hospital)
* Progressive, symmetrical proximal muscle weakness of arms and legs

Exclusion Criteria

* Unable to cooperate for muscle strength testing
* Joint contractures that prohibit muscle strength testing
* Concomitant illness
* Individuals predisposed to excessive vagal responses (bradyarrhythmia or hypotension)
* Controlled substance abuse

Minimum Eligible Age

5 Years

Maximum Eligible Age

15 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No