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

April 26, 2017 updated by: Royan Institute

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

21

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Iran, Islamic Republic of
      • Tehran, Iran, Islamic Republic of
        • Recruiting
        • Royan Institute
        • Contact:
        • Contact:
        • Sub-Investigator:
          • Neda Jarooghi, MSc
        • Sub-Investigator:
          • Tina Bolourieh, BSc
        • Sub-Investigator:
          • Fatemeh Abbasi, MSc

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 50 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • Intramuscular cell transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Myalsia
Time Frame: 1month
Evaluation the presence of myalsia 1month after cell injection.
1month
Mass formation
Time Frame: 6 months
Evaluation the probability of mass formation 6 months after cell injection.
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hematoma
Time Frame: 1 month
Evaluation the presence of hematoma 1 month after injection.
1 month
Muscle bulk
Time Frame: 6 months
Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation.
6 months
CPK
Time Frame: 1month
Evaluation the decrease of CPK 1 month after cell transplantation.
1month

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Royan Institute

Investigators

  • 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 1, 2014

Primary Completion (Anticipated)

November 1, 2017

Study Completion (Anticipated)

December 1, 2017

Study Registration Dates

First Submitted

August 4, 2014

First Submitted That Met QC Criteria

August 4, 2014

First Posted (Estimate)

August 5, 2014

Study Record Updates

Last Update Posted (Actual)

April 27, 2017

Last Update Submitted That Met QC Criteria

April 26, 2017

Last Verified

April 1, 2017

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • Royan-Nerve-007

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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