- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02208713
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
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 Contact
- Name: Leila Arab, MD
- Phone Number: 414 (+98)23562000
- Email: Leara91@gmail.com
Study Contact Backup
- Name: Nasser Aghdami, MD,PhD
- Phone Number: 516 (+98)23562000
- Email: nasser.aghdami@royaninstitute.org
Study Locations
-
-
-
Tehran, Iran, Islamic Republic of
- Recruiting
- Royan Institute
-
Contact:
- Leila Arab, MD
- Phone Number: 414 (+98)23562000
- Email: Leara91@gmail.com
-
Contact:
- Nasser Aghdami, MD,PhD
- Phone Number: 516 (+98)212356000
- Email: nasser.aghdami@royaninstitute.org
-
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:
- Age: 18-50
- Both gender
- Weakness of face muscle
- FSHD phenotype positive
- Genetic Test confirmation for FSHD
Exclusion Criteria:
- Co - morbidities: Heart & respiratory diseases,malignancy, rheumatologic disorders
- Progressive form of disease
- 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.
|
Intramuscular injection of stem cells in patients with FSHD.
Other Names:
|
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
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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