drugset / Trial / NCT02208713

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

NCT02208713 ↗

Phase 1 Unknown 21 enrolled Royan Institute
NaSingle-groupOpen-labelTreatment

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.

Timeline

Start
2014-05
Primary completion
2017-11
Completion
2017-12

Drugs

EvaluationDrugModalityDoseRoute
Subject AdMSC Cell therapy — Intramuscular
Subject Muscle Derived Stem Cell Unknown — Intramuscular

Indications