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UnknownNCT02208713

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

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

Status
Unknown
Phase
Phase 1
Study type
Interventional
Enrollment
21 (estimated)
Sponsor
Royan Institute · Other Government
Sex
All
Age
18 Years – 50 Years
Healthy volunteers
Not accepted

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.

Conditions

Interventions

TypeNameDescription
BIOLOGICALIntramuscular injectionIntramuscular injection of stem cells in patients with FSHD.

Timeline

Start date
2014-05-01
Primary completion
2017-11-01
Completion
2017-12-01
First posted
2014-08-05
Last updated
2017-04-27

Locations

1 site across 1 country: Iran

Source: ClinicalTrials.gov record NCT02208713. Inclusion in this directory is not an endorsement.