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

Dystrophy Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02208713
• Other study ID #: Royan-Nerve-007
• Status: Recruiting
• Phase: Phase 1
• First received: August 4, 2014
• Last updated: April 26, 2017
• Start date: May 2014
• Est. completion date: December 2017

Study information

• Verified date: April 2017
• Source: Royan Institute
• Contact: Nasser Aghdami, MD,PhD
• Phone: (+98)23562000
• Email: nasser.aghdami[@]royaninstitute.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 21
• Est. completion date: December 2017
• Est. primary completion date: November 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

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

Related Conditions & MeSH terms

• Dystrophy

Intervention

Biological:
• Intramuscular injection
Intramuscular injection of stem cells in patients with FSHD.

Locations

Country	Name	City	State
Iran, Islamic Republic of	Royan Institute	Tehran

Sponsors (1)

Lead Sponsor	Collaborator
Royan Institute

Country where clinical trial is conducted

Iran, Islamic Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Myalsia	Evaluation the presence of myalsia 1month after cell injection.	1month
Primary	Mass formation	Evaluation the probability of mass formation 6 months after cell injection.	6 months
Secondary	Hematoma	Evaluation the presence of hematoma 1 month after injection.	1 month
Secondary	Muscle bulk	Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation.	6 months
Secondary	CPK	Evaluation the decrease of CPK 1 month after cell transplantation.	1month

External Links

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