Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)

Recruiting Phase 1 Interventional Study
FSHD - Facioscapulohumeral Muscular Dystrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
15 and older
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
16 (estimated)
Sponsor
Restem, LLC. · Industry
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About This Trial
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2. It will assess safety and preliminary efficacy in relieving symptoms of FSHD with ULSC administered in two intravenous (IV) doses of 100 million cells per dose. The main questions that this study plans to answe…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Participants will be ≥15 years old. * Diagnosis of genetically confirmed FHSD 1 or FSHD 2. * Participants should have a Ricci clinical severity score of ≥3 (range is out of 0-10), at screening, and must be independently ambulatory at the time of the study. * Participant will h…
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