Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02196467 |
Recruitment Status :
Recruiting
First Posted : July 22, 2014
Last Update Posted : January 29, 2021
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Biological: Myoblast transplantation Procedure: Saline injection | Phase 1 Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 10 participants |
Allocation: | Randomized |
Intervention Model: | Single Group Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients |
Study Start Date : | May 2014 |
Estimated Primary Completion Date : | January 2024 |
Estimated Study Completion Date : | January 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: Myoblast transplantation & strength
30 million myoblasts will be transplanted per centimeter cube in the Extensor carpi radialis of one of the patient's forearms, resuspended in saline. The strength will be evaluated after 3 and 6 months and the presence of dystrophin after 3 or 6 months.
|
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). |
Sham Comparator: Saline injection & strength
The same saline solution used in the previous arm, but without cells, will be injected similarly per centimeter cube in the Extensor carpi radialis of the contralateral patient's forearm. The strength will be evaluated after 3 and 6 months and the presence of dystrophin after 3 or 6 months.
|
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). |
- Number of Participants with Serious and Non-Serious Adverse Events as a measure of safety. [ Time Frame: Up to 6 months ]The patients will be monitored for local and systemic potential adverse effects due to the transplantation and for adverse effects associated with immunosuppression with tacrolimus.
- Percentage of dystrophin-positive fibers in a muscle biopsy 3 or 6 months after myoblast transplantation. [ Time Frame: 6 months after the myoblast transplantation ]The presence of dystrophin positive fibers will be assessed in a muscle biopsy done 6 months after the myoblast transplantation.
- Strength of the Extensor carpi radialis muscles. [ Time Frame: At 3 and 6 months after myoblast transplantation. ]The strength of both Extensor carpi radialis will be evaluated 3 and 6 months after the myoblast transplantation to evaluate whether this transplantation improved the muscle strength, prevented or slowed down the progression of the muscle weakness.
- Presence of a cellular and humoral reaction against the donor antigens [ Time Frame: Every 4 weeks after transplantation for 6 months ]To assess antibody-mediated immune responses, a blood sample will be obtained at days D-14 and D15, at week 4 and every 4 weeks until the end of the treatment schedule according to the transplant pattern of the subject, and at the 3 and 6 month follow ups. These blood samples will be used to make cross-matches to determine whether the subject is producing antibodies reacting with the donor myoblasts. The antibodies against donor myoblasts will be detected by flow cytometry. Antibodies against donor HLA class I and II antigens will also be assessed by flow cytometry using single HLA antigen-coated beads (Flow PRA beads, One Lambda, Canoga Park, CA).

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 16 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02196467
Contact: Craig Campbell, MD MSc FRCPC | (519) 685-8332 | craig.campbell@lhsc.on.ca | |
Contact: Jacques Tremblay, PhD | (418)-525-4444 ext 47307 | Jacques-P.Tremblay@crchul.ulaval.ca |
Canada, Ontario | |
Children's Hospital London Health Sciences Centre | Recruiting |
London, Ontario, Canada, N6A 4G5 | |
Canada | |
Centre de recherche du CHU de Quebec - CHUL | Recruiting |
Quebec, Canada, G1V 4G2 |
Principal Investigator: | Craig Campbell, MD MSc FRCPC | University of Western Ontario, Canada | |
Principal Investigator: | Jack Puymirat, MD | Centre de recherche du CHU de Quebec |
Responsible Party: | CHU de Quebec-Universite Laval |
ClinicalTrials.gov Identifier: | NCT02196467 |
Other Study ID Numbers: |
SIRUL 104501 299825 ( Other Grant/Funding Number: CIHR/IRSC ) |
First Posted: | July 22, 2014 Key Record Dates |
Last Update Posted: | January 29, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | The clinical trial results that have a relation to its objectives will be published as scientific papers in scientific journals or presented at scientific meetings, but without revealing the identity of the participants who will always remain anonymous. |
Time Frame: | The data will begin to be available as of the fall of 2019, not being decided yet for how long. |
Access Criteria: | Information to be shared will be the expression of dystrophin, changes in strength, and evidence or not of rejection, in the muscles injected, as well as the side effects observed. |
myoblast transplantation strength tacrolimus immunosuppression |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |