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Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients

NCT02196467 · Status: UNKNOWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2021-01-29

No results posted yet for this study

Summary

This Phase I/II of the clinical trial is to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis) of the patients is safe and will improve the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of a donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm cube in a progressively higher surface of the radialis (i.e., 3, 6 and 9 cm2). The contralateral muscle will be injected with saline to serve as a control. The strength of both muscles will be measured at 3 months post transplantation to verify whether the myoblast transplantation improved the strength of the muscle. If there is no significant strength improvement, the protocol will be terminated immediately for that patient. If there is a significant strength improvement, the patient will be maintained under immunosuppression until 6 months post transplant and his strength will be re-evaluated.

Conditions

Interventions

BIOLOGICAL

Myoblast transplantation

30 million myoblasts will be transplanted per centimeter cube in the Extensor carpi radialis of one of the patient's forearm, resuspended in saline (a total of 0.5 ml of suspension per centimetre cube of muscle).

PROCEDURE

Saline injection

A saline solution (the same used to resuspend de myoblasts in the first intervention) will be injected similarly in the Extensor carpi radialis of the contralateral patient's forearm (a total of 0.5 ml of saline per centimetre cube of muscle).

Sponsors & Collaborators

  • CHU de Quebec-Universite Laval

    lead OTHER

Principal Investigators

  • Craig Campbell, MD MSc FRCPC · University of Western Ontario, Canada

  • Jack Puymirat, MD · Centre de recherche du CHU de Quebec

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SINGLE_GROUP

Eligibility

Min Age
16 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-05-31
Primary Completion
2024-01-31
Completion
2024-01-31

Countries

  • Canada

Study Locations

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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 NCT02196467 on ClinicalTrials.gov