Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients

Inclusion Criteria:

• 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 Criteria:

• 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.