Clinical trial: Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients (NCT02196467)

Eligibility criteria:
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.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 16 Years old.


Patient description (medical language): An 8-year-old boy is brought to the clinic by his parents because of weakness and difficulty of standing up from a sitting position. The mother is healthy but had a brother who died in his 20th after being disabled and using wheelchairs in the last few years of his life. Physical examination shows 3/5 lower extremity muscle strength and enlarged calf muscles. The other physical examination and vital signs are unremarkable. Muscle biopsy showed absence of dystrophin protein. The patient is diagnosed with DMD.

Patient description (patient's own words): I brought my  8-year-old son to the doctor's clinic. He was struggling to stand up from a chair for the past few days. My wife is healthy but she was pretty worried since his brother died in his 20s after being disabled and using a wheelchair for the last years of his life. The doctor did an exam which highlighted weakness in the leg muscles and unusually large calf muscles. The rest of the exam was fine, and his vitals were normal. My son did a muscle biopsy, and it showed that the protein important for muscle strength and function is missing. The doctor diagnosed him with Duchenne muscular dystrophy.

Does the patient match the trial criteria? 0