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Cell Therapy
Myoblast Transplantation for Duchenne Muscular Dystrophy
Phase 1 & 2
Waitlist Available
Led By Craig Campbell, MD MSc FRCPC
Research Sponsored by CHU de Quebec-Universite Laval
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 3 and 6 months after myoblast transplantation.
Awards & highlights
Study Summary
This trial is to see if it is safe to transplant normal myoblasts into patients muscles, and if it improves the strength of that muscle.
Eligible Conditions
- Duchenne Muscular Dystrophy
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at 3 and 6 months after myoblast transplantation.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 3 and 6 months after myoblast transplantation.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of Participants with Serious and Non-Serious Adverse Events as a measure of safety.
Secondary outcome measures
Percentage of dystrophin-positive fibers in a muscle biopsy 3 or 6 months after myoblast transplantation.
Presence of a cellular and humoral reaction against the donor antigens
Strength of the Extensor carpi radialis muscles.
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Myoblast transplantation & strengthExperimental Treatment1 Intervention
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.
Group II: Saline injection & strengthPlacebo Group1 Intervention
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.
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Who is running the clinical trial?
CHU de Quebec-Universite LavalLead Sponsor
167 Previous Clinical Trials
107,147 Total Patients Enrolled
Craig Campbell, MD MSc FRCPCPrincipal InvestigatorUniversity of Western Ontario, Canada
Jack Puymirat, MDPrincipal InvestigatorCentre de recherche du CHU de Quebec
1 Previous Clinical Trials
323 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- You have had a previous cancer.You had tuberculosis before or might have it without showing symptoms.You have had kidney problems in the past or your recent lab tests show that your kidneys may not be working properly.You have tested positive for HIV, hepatitis B, hepatitis C, HTLV-1, or anti-HTLV-2.A family member who is at least 18 years old and has the right genetic match should be available as a potential donor.You need to have received vaccinations for pneumococcus and Haemophilus influenzae.You have taken a medication that does not work well with tacrolimus in the past month.You have antibodies in your blood that could react against the donor's immune cells.You have had biopsies or injections in a specific muscle in your arm.You have changed your heart medications within the last 3 months before the first myoblast transplantation.There is no suitable donor available for a transplant.Your blood sugar levels are not within the normal range.You need to be able to move both wrists with a score of 2 or higher on a movement scale.If you need help breathing with a machine, you have been using the same settings for at least 3 months before the treatment starts, and you are expected to keep using these settings during the study.You have a long-lasting infection.You have been diagnosed with DMD and have a confirmed mutation in the dystrophin gene or less than 10% of muscle fibers test positive for dystrophin in a muscle biopsy.Your blood test shows abnormal levels of red or white blood cells, hemoglobin, or platelets.You have any major health conditions that are not related to DMD, like heart, hormone, blood, liver, immune system, metabolism, urinary, lung, nerve, skin, mental health, kidney, or other serious diseases.You have previously taken part in phase 1A of myoblast transplantation.You had a test on the muscles in your wrist within the last 6 months.You have made changes to any medication that could affect your muscle function within the last 3 months before the first myoblast transplantation.You have an abnormal feeling in your body that a doctor checks.You must be at least 16 years old to participate.You must have received vaccinations for pneumococcus and Haemophilus influenzae.A family member who is at least 18 years old and is a suitable match for organ donation should be accessible.You must be able to move both wrists with a certain level of strength.If you need help breathing, you have been using the same non-invasive ventilation settings for at least 3 months before starting the study and will keep using the same settings during the study.You must have a confirmed diagnosis of DMD, which can be shown by a specific mutation in the dystrophin gene or a muscle biopsy showing very low levels of dystrophin.
Research Study Groups:
This trial has the following groups:- Group 1: Saline injection & strength
- Group 2: Myoblast transplantation & strength
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many participants are actively engaging with this research initiative?
"Affirmative. Clinicaltrials.gov displays information confirming that this clinical trial, which was initially added on May 1st 2014, is still accepting enrolment requests. Recruiters are searching for 10 patients to be spread across 2 medical centres."
Answered by AI
Is this medical evaluation presently enrolling participants?
"Indeed, the information hosted on clinicaltrials.gov asserts that this medical trial is presently recruiting patients. It was first published in May 2014 while its most recent update occurred at the end of January 2021. This experiment requires 10 enrollees across 2 locations."
Answered by AI
Who else is applying?
What state do they live in?
California
What site did they apply to?
Children's Hospital London Health Sciences Centre
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
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