Status:
UNKNOWN
Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients
Lead Sponsor:
CHU de Quebec-Universite Laval
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
16+ years
Phase:
PHASE1
PHASE2
Brief 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...
Detailed Description
Duchenne Muscular Dystrophy (DMD) is a degenerative disease of genetic origin, due to a mutation in the gene coding for the protein dystrophin. This mutation leads to deficiency of dystrophin in the m...
Eligibility Criteria
Inclusion
- A clinical diagnosis of DMD must be confirmed (i.e., with supporting confirmation demonstrated by the identification of a mutation in the dystrophin gene compatible with DMD or presence of less than 10% dystrophin positive fibers in a muscle biopsy in a subject with DMD).
- The subject has to be older than 16 years of age.
- Male
- If on corticosteroids, a stable dose must be maintained for 6 months prior to myoblast transplantation and throughout the trial
- A potential haplotype compatible donor (the father, the mother, a brother or sister who is more than 18 years old) should be available.
- The subject must be able to move both wrists, with an MRC scale score of greater than or equal to 2.
- Subject must have been vaccinated for pneumococcus and Haemophilus influenzae.
- For subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception for the duration of the study.
- For subjects that need assisted ventilation, a stable regimen of non-invasive ventilation parameters for 3 months prior to the first myoblast transplantation and anticipation that they will be on a stable regimen throughout the study.
- Written informed consent of the subject and donor.
Exclusion
- An abnormal sensory examination
- Persisting abnormal values in a hemogram (red blood cells, white blood cells, hemoglobin or platelets out of laboratory normal range).
- A history of chronic infection.
- Abnormal glycosylated hemoglobin level and/or fasting blood glucose (values out of laboratory normal range)
- Previous neoplasia.
- Previous tuberculosis or potential carrier of latent tuberculosis.
- Any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease as determined by the Investigator that is not related to DMD
- Previous history of renal problems or laboratory analyses suggestive of a renal problem (cystatin C, blood urea nitrogen, electrolytes out of laboratory normal range).
- Previous biopsies or intramuscular injections in any of the extensor carpi radialis.
- Subject who participated to phase 1A of myoblast transplantation
- The subject uses a drug that is not compatible with tacrolimus (see section 6 "Concomitant medications" of protocol) within the last month. If the subject has previously used one of these drugs, the washout period before the onset of tacrolimus should be at least 1 month.
- Subject tests positive for HIV-1, HIV-2, antigen HIV-1, HBC (hepatitis B surface antigen (HBsAg) and hepatitis B core antigen) HCV, HTLV-1 and anti-HTLV-2.
- The subject was submitted to electromyography in the extensor carpi radialis, within the last 6 months.
- There are pre-existing antibodies in the subject serum against the donor lymphocytes.
- Any change (initiation, dose adjustment, interruption or discontinuation) in any medication that may affect muscle function (eg. Losartan, coenzyme Q10, green tea extract, idebenone, creatine, nutritional supplements, etc.) within 3 months of the first myoblast transplantation.
- Any change in cardiac medications (ACE inhibitor, beta-blocker, etc.) within 3 months of first myoblast transplantation.
- Any surgery or fracture of the upper extremity within 3 months prior to first myoblast transplantation or plans to have surgery during the course of the trial.
- No haplotype compatible donor is available.
- Unwillingness or inability of the subject to understand and comply with the requirements of this protocol in the opinion of the Investigator or sponsor.
- Previous tuberculosis or potential carrier of latent tuberculosis.
- Previous treatment with any other investigational product within 6 months of myoblast transplantation.
Key Trial Info
Start Date :
May 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 1 2024
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT02196467
Start Date
May 1 2014
End Date
January 1 2024
Last Update
January 29 2021
Active Locations (2)
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1
Children's Hospital London Health Sciences Centre
London, Ontario, Canada, N6A 4G5
2
Centre de recherche du CHU de Quebec - CHUL
Québec, Canada, G1V 4G2