Clinical trial NCT02208713: Intramuscular Transplantation of Muscle Derived Stem Cell and Adipose Derived Mesenchymal Stem Cells in Patients With Facioscapulohumeral Dystrophy (FSHD)
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Dystrophy
Keywords: FSHD muscle derived stem cell adipose derived mesenchymal stem cell intramuscular injection
Lead sponsor: Royan Institute
Sponsor class: OTHER_GOV
First posted: 2014-08-05
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Myalsia
timeFrame: 1month
measure: Mass formation
timeFrame: 6 months
Secondary outcomes:
measure: Hematoma
timeFrame: 1 month
measure: Muscle bulk
timeFrame: 6 months
measure: CPK
timeFrame: 1month
Other outcomes:
Locations:
city: Tehran
country: Iran, Islamic Republic of
facility: Royan Institute
Study officials:
affiliation: Head of Royan Institute
name: Hamid Gourabi, PhD
role: STUDY_CHAIR
affiliation: Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute
name: Nasser Aghdami, MD,PhD
role: STUDY_DIRECTOR
affiliation: Scientific Board of BouAli Hospital, Azad University
name: Mahdi Vahid Dastjerdi, MD
role: STUDY_DIRECTOR
affiliation: Department of Regenerative Medicine & cell therapy of Royan Institute
name: Leila Arab, MD
role: PRINCIPAL_INVESTIGATOR
References: