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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02208713
Recruitment Status : Unknown
Verified April 2017 by Royan Institute.
Recruitment status was:  Recruiting
First Posted : August 5, 2014
Last Update Posted : April 27, 2017
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Dystrophy Biological: Intramuscular injection Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 2014
Estimated Primary Completion Date : November 2017
Estimated Study Completion Date : December 2017

Arm Intervention/treatment
Experimental: Stem cell recipient
The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection.
Biological: Intramuscular injection
Intramuscular injection of stem cells in patients with FSHD.
Other Name: Intramuscular cell transplantation

Primary Outcome Measures :
  1. Myalsia [ Time Frame: 1month ]
    Evaluation the presence of myalsia 1month after cell injection.

  2. Mass formation [ Time Frame: 6 months ]
    Evaluation the probability of mass formation 6 months after cell injection.

Secondary Outcome Measures :
  1. Hematoma [ Time Frame: 1 month ]
    Evaluation the presence of hematoma 1 month after injection.

  2. Muscle bulk [ Time Frame: 6 months ]
    Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation.

  3. CPK [ Time Frame: 1month ]
    Evaluation the decrease of CPK 1 month after cell transplantation.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02208713

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Contact: Nasser Aghdami, MD,PhD (+98)23562000 ext 516
Contact: Leila Arab, MD (+98)23562000 ext 414

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Iran, Islamic Republic of
Royan Institute Recruiting
Tehran, Iran, Islamic Republic of
Contact: Nasser Aghdami, MD,PhD    (+98)212356000 ext 516   
Contact: Leila Arab, MD    (+98)23562000 ext 414   
Sub-Investigator: Neda Jarooghi, MSc         
Sub-Investigator: Tina Bolourieh, BSc         
Sub-Investigator: Fatemeh Abbasi, MSc         
Sponsors and Collaborators
Royan Institute
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Study Chair: Hamid Gourabi, PhD Head of Royan Institute
Study Director: Nasser Aghdami, MD,PhD Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute
Study Director: Mahdi Vahid Dastjerdi, MD Scientific Board of BouAli Hospital, Azad University
Principal Investigator: Leila Arab, MD Department of Regenerative Medicine & cell therapy of Royan Institute
Additional Information:
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Responsible Party: Royan Institute Identifier: NCT02208713    
Other Study ID Numbers: Royan-Nerve-007
First Posted: August 5, 2014    Key Record Dates
Last Update Posted: April 27, 2017
Last Verified: April 2017
Keywords provided by Royan Institute:
FSHD muscle derived stem cell adipose derived mesenchymal stem cell intramuscular injection