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

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

Status

UNKNOWN

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02208713

Enrollment

Registered

2014-08-05

Start date

2014-05-31

Completion date

2017-12-31

Last updated

2017-04-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Dystrophy

Keywords

FSHD muscle derived stem cell adipose derived mesenchymal stem cell intramuscular injection

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.

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.

Interventions

BIOLOGICALIntramuscular injection

Intramuscular injection of stem cells in patients with FSHD.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 50 Years

Healthy volunteers

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

Design outcomes

Primary

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

Secondary

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

Countries

Iran

Contacts

Primary ContactNasser Aghdami, MD,PhD

nasser.aghdami@royaninstitute.org(+98)23562000

Backup ContactLeila Arab, MD

Leara91@gmail.com(+98)23562000

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026