Intramuscular Transplantation of Muscle Derived Stem Cell and Adipose Derived Mesenchymal Stem Cells in Patients With Facioscapulohumeral Dystrophy (FSHD)

Study Overview
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Brief Summary
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Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and myotonic dystrophy. The symptoms including: Pain, facial weakness, scapular fixator, humeral, truncal, pelvic girdle and lower-extremity weakness, High frequency hearing loss, Retinal telangiectasia . The existing treatments are not effective so, cell therapy is a new hope to improve patients' quality of life. Therefore, We design this clinical trial to evaluate the safety and feasibility of stem cell transplantation.

Detailed Description
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In this study, we select 15 patients with FSHD based on eligibility criteria. All the patients underwent physical examination, laboratory evaluations, EMG-NCV, muscle sonography and muscle MRI. Then, a sample of patient's muscle is taken from Biceps Femoralis to isolate and culture of MDSC. The AD-MSC is prepared from Royan Adipose Tissue Bank. The patient is admitted in general hospital, and the cell suspensions are injected into biceps, triceps and trapezoids muscles by neurologists. After transplantation, the patients will be under observation for 5 hours and will be discharged if no side effect happen. All the patients will be followed at 1,2,4,6 and 12 months after cell injection.

Official Title
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Intramuscular Transplantation of Autologous Muscle Derived Stem Cell(MDSC) and Adipose Derived Mesenchymal Stem Cells (AD-MSC) in Patients With Facioscapulohumeral Dystrophy (FSHD), Phase I Clinical Trial

Conditions
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Dystrophy

Intervention / Treatment
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* Biological: Intramuscular injection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age: 18-50 Both gender Weakness of face muscle FSHD phenotype positive Genetic Test confirmation for FSHD Exclusion Criteria: Co - morbidities: Heart & respiratory diseases,malignancy, rheumatologic disorders Progressive form of disease Not able to sign the consent

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Stem cell recipient<br>The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection. | Biological: Intramuscular injection<br>* Intramuscular injection of stem cells in patients with FSHD.<br>* Other names: Intramuscular cell transplantation;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Myalsia | Evaluation the presence of myalsia 1month after cell injection. | 1month | 
| Mass formation | Evaluation the probability of mass formation 6 months after cell injection. | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematoma | Evaluation the presence of hematoma 1 month after injection. | 1 month | 
| Muscle bulk | Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation. | 6 months | 
| CPK | Evaluation the decrease of CPK 1 month after cell transplantation. | 1month | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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FSHD muscle derived stem cell adipose derived mesenchymal stem cell intramuscular injection