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

Royan Institute

Status and phase: Unknown; Phase 1

Conditions: Dystrophy

Treatments: Biological: Intramuscular injection

Study type: Interventional

Funder types: Other

Identifiers

NCT02208713

Royan-Nerve-007

Details and patient eligibility

About

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.

Full 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.

Enrollment: 21 estimated patients

Sex: All

Ages: 18 to 50 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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21 participants in 1 patient group

Stem cell recipient

Experimental group

Description:

The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection.

Treatment:

Biological: Intramuscular injection