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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Stem cell recipient | Experimental | The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Intramuscular injection | Biological | Intramuscular injection of stem cells in patients with FSHD. |
|
| 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 |
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Nasser Aghdami, MD,PhD | Contact | (+98)23562000 | 516 | nasser.aghdami@royaninstitute.org |
| Leila Arab, MD | Contact | (+98)23562000 | 414 | Leara91@gmail.com |
| Name | Affiliation | Role |
|---|---|---|
| Hamid Gourabi, PhD | Head of Royan Institute | Study Chair |
| Nasser Aghdami, MD,PhD | Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Royan Institute | Recruiting | Tehran | Iran |
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| Label | URL |
|---|---|
| Related Info | View source |
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| 6 months |
| CPK | Evaluation the decrease of CPK 1 month after cell transplantation. | 1month |
| Mahdi Vahid Dastjerdi, MD |
| Scientific Board of BouAli Hospital, Azad University |
| Study Director |
| Leila Arab, MD | Department of Regenerative Medicine & cell therapy of Royan Institute | Principal Investigator |
| ID | Term |
|---|---|
| D007273 | Injections, Intramuscular |
| ID | Term |
|---|---|
| D007267 | Injections |
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |
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