Intramuscular Transplantation of Muscle Derived Stem Cell and Adipose Derived Mesenchymal Stem Cells in Patients With Facioscapulohumeral Dystrophy (FSHD)
1 other identifier
interventional
21
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started May 2014
Typical duration for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2014
CompletedFirst Submitted
Initial submission to the registry
August 4, 2014
CompletedFirst Posted
Study publicly available on registry
August 5, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2017
CompletedApril 27, 2017
April 1, 2017
3.5 years
August 4, 2014
April 26, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Myalsia
Evaluation the presence of myalsia 1month after cell injection.
1month
Mass formation
Evaluation the probability of mass formation 6 months after cell injection.
6 months
Secondary Outcomes (3)
Hematoma
1 month
Muscle bulk
6 months
CPK
1month
Study Arms (1)
Stem cell recipient
EXPERIMENTALThe patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection.
Interventions
Intramuscular injection of stem cells in patients with FSHD.
Eligibility Criteria
You may qualify if:
- Age: 18-50
- Both gender
- Weakness of face muscle
- FSHD phenotype positive
- Genetic Test confirmation for FSHD
You may not qualify if:
- Co - morbidities: Heart \& respiratory diseases,malignancy, rheumatologic disorders
- Progressive form of disease
- Not able to sign the consent
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Royan Institutelead
Study Sites (1)
Royan Institute
Tehran, Iran
Related Links
MeSH Terms
Interventions
Intervention Hierarchy (Ancestors)
Study Officials
- 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
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER GOV
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 4, 2014
First Posted
August 5, 2014
Study Start
May 1, 2014
Primary Completion
November 1, 2017
Study Completion
December 1, 2017
Last Updated
April 27, 2017
Record last verified: 2017-04