Actively Recruiting
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
Led by Restem, LLC. · Updated on 2025-12-08
16
Participants Needed
1
Research Sites
169 weeks
Total Duration
On this page
Sponsors
R
Restem, LLC.
Lead Sponsor
S
Solve FSHD
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 15 years or older
- Genetically confirmed diagnosis of FSHD type 1 or 2
- Ricci clinical severity score of 3 or more at screening
- Independently ambulatory at the time of the study
- Ability to comply with study requirements including MRI
- Use of two forms of highly effective contraception if of reproductive age during and for 3 months after the study
- Ability to understand and provide written informed consent
- Stable dose for at least 3 months prior to first dose of any drug(s) or supplements affecting muscle function, including immunomodulatory agents
- Reduced upper arm strength with Performance of Upper Limb score of 5 or less
- Current and up-to-date immunizations
- Reachable workspace (RSA) in dominant upper extremity between 0.2 and 0.7
- No contraindications to MRI
- Hematocrit 50% or less
- Prostate-specific antigen 4.0 ng/mL or less (3.0 ng/mL if first-degree relative with prostate cancer)
- Fasting blood glucose less than 126 mg/dL
You will not qualify if you...
- Hypersensitivity to study product components or dimethyl sulfoxide (DMSO)
- Active cancer or cancer diagnosis within the past year (except basal or squamous cell skin cancer)
- Any condition contraindicating enrollment, study product administration, or follow-up as judged by Investigator or Sponsor
- Treatment with investigational product within 3 months before randomization
- Known active opportunistic or life-threatening infections including HIV and hepatitis B or C
- Known active or inactive tuberculosis infection
- Use of products enhancing muscle growth chronically within 4 weeks before baseline
- Use of oral cytochrome P450 (CYP3A4), MATE, or OAT3 substrates as concomitant therapy
- Statin initiation or significant adjustment within 3 months before baseline (stable use allowed)
- Rapamycin treatment within 3 months before baseline
- Alternative diagnosis or coexisting myopathy or dystrophy confirmed by prior biopsy or investigations
- Muscle biopsy within 30 days before baseline
- Systolic blood pressure over 160 mmHg or diastolic over 100 mmHg
- Heavy alcohol use (more than 50g/day)
- Current testosterone or human growth hormone use
- Use of medications interfering with growth hormone or gonadal endocrine axis
- Pregnant or lactating participants
- Severe cardiac or pulmonary disease, active infection, or other conditions precluding safety and efficacy assessment
- Planned surgery during the trial period
- History of noncompliance with medical therapy
- Organ transplant recipient
- Neutropenia (absolute neutrophil count less than 1800/mm³ or less than 1000/mm³ in African-American participants)
- Severe renal impairment (eGFR less than 30 ml/kg*min)
- Recent or planned live attenuated virus vaccination
- Conditions impairing muscle strength assessment such as Parkinson's disease or severe musculoskeletal condition
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Stanford Neuroscience Health Center
Palo Alto, California, United States, 94304
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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