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

Phase 1

• Conditions: Dystrophy
• Interventions: Biological: Intramuscular injection

• Registration Number: NCT02208713
• Lead Sponsor: Royan Institute

Brief Summary

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

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.

Study Timeline

• Study Start: 2014-05
• Study First Submitted: 2014-08-04
• Study First Submitted QC: 2014-08-04
• Study First Posted: 2014-08-05
• Status Verified: 2017-04
• Last Update Submitted: 2017-04-26
• Last Update Posted: 2017-04-27
• Primary Completion: 2017-11
• Study Completion: 2017-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

1. Age: 18-50
2. Both gender
3. Weakness of face muscle
4. FSHD phenotype positive
5. Genetic Test confirmation for FSHD

Exclusion Criteria

1. Co - morbidities: Heart & respiratory diseases,malignancy, rheumatologic disorders
2. Progressive form of disease
3. Not able to sign the consent

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Stem cell recipient	Intramuscular injection	The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection.

Primary Outcome Measures

Name	Time	Method
Mass formation	6 months	Evaluation the probability of mass formation 6 months after cell injection.
Myalsia	1month	Evaluation the presence of myalsia 1month after cell injection.

Secondary Outcome Measures

Name	Time	Method
Hematoma	1 month	Evaluation the presence of hematoma 1 month after injection.
CPK	1month	Evaluation the decrease of CPK 1 month after cell transplantation.
Muscle bulk	6 months	Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation.

Trial Locations

Locations (1)

Royan Institute; 🇮🇷 Tehran, Iran, Islamic Republic of