Evaluation of safety and efficacy of focal intramuscular injection of wharton’s gelly-derived mesenchymal stem cell in Duchenne muscular dystrophy pediatrics between 5 to 12 years old
- Conditions
- Duchenne muscular dystrophy.Muscular dystrophyG71.0
- Registration Number
- IRCT20150206020981N4
- Lead Sponsor
- Sinacell Company
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- 5
Age between 5 to 12
Genetic evidence of Duchenne disease as a cause of myopathy
The patient's ability to walk is not completely lost and the use of assistive devices such as canes and walkers is not permanent.
The patient's muscle strength is decreasing
Willingness and satisfaction to participate in the study by the patient's legal guardian in decision making and willingness to participate regularly in follow-up courses
Weight between 15 to 35
Advanced motor impairment leading to permanent use of crutches, walkers and wheelchairs
A history of known malignancy
History of associated genetic and non-genetic degenerative diseases
A history of any other acute or chronic illness that, at the discretion of the clinician responsible for implementing the plan, prevents the patient from entering the study.
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Respiratory Function. Timepoint: 1, 3, 6, 9 and 12 month after injection. Method of measurement: Spirometery.;Muscular Force. Timepoint: First every 14 days to 1 month, then Every 1 month to 1 year. Method of measurement: Physical Exam.;Cardiac Function. Timepoint: 1, 3, 6, 9 and 12 month after injection. Method of measurement: Echocardiography.
- Secondary Outcome Measures
Name Time Method Muscular Enzymes. Timepoint: 1, 3, 6, 9 and 12 month after injection. Method of measurement: Lab Data.