A Phase 1, Double-blinded, Randomized, Dose-repeating, Placebo-controlled, Cross-over Study to Assess the Safety and Preliminary Efficacy of Allogeneic ULSC on Disease Severity in Facioscapulohumeral Muscular Dystrophy (FSHD)
Restem, LLC.
Summary
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 answer are: * Is ULSC as safe as placebo (a look-alike saline without cells) in repeated IV infusion? * Does ULSC improve symptoms of FSHD after each dose? Researchers will compare ULSC to placebo. Participants will: * Have been diagnosed with FSHD of a Ricci clinical severity score 3 or more. * Participate in this study for total duration of 21 months with 11 in-person visits and 5 virtual visits. * Visit the clinic for a total of 4 IV infusions (250 mL) 3 months apart. * Receive 2 doses of ULSC and 2 doses placebo in either of two sequences, as assigned: ULSC first (Day 0 and Month 3) and placebo second (Month 6 and Month 9), or placebo first (Day 0 and Month 3) and ULSC second (Month 6 and Month 9). * Return for follow-up visits after each dose and up to 12 months after final dose.
Eligibility
- Age range
- 15+ years
- Sex
- All
- Healthy volunteers
- No
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 have the ability to comply with the requirements of the study, including MRI. * All participants of reproductive age/capacity will be required to use adequate contraception, defined as two forms of highly effective contraceptives, with any partners during the study period and for at least three month…
Interventions
- BiologicalULSC
Allogeneic umbilical-cord lining stem cells (ULSC) are cryopreserved and supplied in vials to be thawed and prepared for infusion at point of use. Each dose of 1 x 10\^8 ULSC will be added to into 250 sterile saline for infusion (total volume of 260 mL volume).
- BiologicalPlacebo
The Placebo will be 250 ml of sterile saline for IV administration.
Location
- Stanford Neuroscience Health CenterPalo Alto, California