Stem Cell Therapy for Facioscapulohumeral Muscular Dystrophy

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Restem, LLC.

Must be taking: Immunomodulatory agents

Must not be taking: CYP3A4 substrates, Statins, Rapamycin, others

Disqualifiers: Cancer, HIV, Tuberculosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 11

Duration: 12 months

16 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a stem cell treatment to determine its safety and effectiveness for people with Facioscapulohumeral Muscular Dystrophy (FSHD), a condition that weakens muscles. Researchers aim to discover if the stem cell product, ULSC, administered through an IV, is safe and can improve symptoms compared to a placebo (a look-alike without active cells). Individuals with FSHD who can still walk independently might be suitable for this study. Participants will receive both the stem cell treatment and placebo at different times to compare effects. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

If you're taking medications or supplements that affect muscle function, you need to be on a stable dose for at least 3 months before starting the trial and continue that dose throughout the study. Some medications, like certain oral drugs and statins, have specific restrictions, so it's best to discuss your current medications with the study team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

A previous study showed that patients receiving stem cells from umbilical cords, such as ULSC, improved in muscle strength without major safety concerns. Early results suggest these stem cells are generally safe for individuals with muscular dystrophies. No significant reports of serious side effects have emerged from these studies, indicating that any side effects were neither severe nor common. As this trial is in its early stages, it focuses on safety, with researchers closely monitoring for any side effects. Overall, the treatment appears promising in terms of safety, but ongoing research will offer more detailed information. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for FSHD?

Researchers are excited about ULSC stem cell therapy for Facioscapulohumeral Muscular Dystrophy (FSHD) because it offers a novel approach compared to standard treatments like physical therapy and corticosteroids, which primarily manage symptoms. ULSC therapy involves using umbilical cord-derived stem cells that have the potential to regenerate damaged muscle tissue, addressing the root cause of the disease rather than just alleviating symptoms. This treatment is administered through an IV, providing a targeted and potentially more effective delivery method. By focusing on muscle regeneration, ULSC therapy could offer patients improved muscle function and quality of life, making it a promising alternative to existing options.

What evidence suggests that ULSC might be an effective treatment for FSHD?

This trial will compare the effects of ULSC, a stem cell therapy derived from the umbilical cord, with a placebo in participants with Facioscapulohumeral Muscular Dystrophy (FSHD). Research has shown that stem cells from the umbilical cord, like those in ULSC, might help improve muscle strength in people with muscular dystrophies. In previous studies, these stem cells, taken from Wharton's jelly, showed promising results in boosting average muscle strength. They could potentially help with conditions like FSHD by easing some symptoms and improving muscle function. While more research is needed to confirm these findings, early results offer encouragement for those affected by FSHD. ^{1 2 3 4 5}

Who Is on the Research Team?

John W Day, MD, PhD

Principal Investigator

Stanford University, School of Medicine, Neuromuscular Research Division

Are You a Good Fit for This Trial?

This trial is for individuals with genetically confirmed Facioscapulohumeral Muscular Dystrophy (FSHD) who have a Ricci score of ≥3, indicating moderate to severe symptoms. They must be at least 15 years old, able to give consent, follow study requirements including MRI scans, and use two forms of contraception if applicable. Participants should also have specific upper arm strength and reachable workspace measurements.

Inclusion Criteria

I can understand and sign the consent form.

My vaccinations are current and up-to-date.

I have a genetic diagnosis of FSHD 1 or FSHD 2.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 4 IV infusions of ULSC or placebo, 3 months apart, in a cross-over design

12 months

4 in-person visits for infusions

Follow-up

Participants are monitored for safety and effectiveness after treatment, with follow-up visits after each dose and up to 12 months after the final dose

12 months

7 in-person visits, 5 virtual visits

What Are the Treatments Tested in This Trial?

Interventions

• ULSC

Trial Overview The trial tests the safety and preliminary effectiveness of ULSC treatment compared to placebo in FSHD patients. It involves four IV infusions over nine months: two doses each of ULSC and placebo in random sequences. The study spans 21 months with multiple clinic visits and virtual check-ins.

How Is the Trial Designed?

Group I: Group 2: Placebo first; ULSC second Experimental Treatment 2 Interventions

Group 2 will receive Placebo IV administrations on Day 0 and Month 3. They will then cross-over to receive 1 x 10\^8 ULSC IV administrations on Month 6 and Month 9 (2 x 10\^8 ULSC in total).

Group II: Group 1: ULSC first; Placebo second Experimental Treatment 2 Interventions

Group 1 will receive a dose of 1 x 10\^8 ULSC through IV administration on Day 0 and Month 3 (two doses, 2 x 10\^8 ULSC in total from both doses). They will then cross-over to Placebo IV administrations on Month 6 and Month 9.

Who Is Running the Clinical Trial?

Restem, LLC.

Lead Sponsor

Trials

2

Recruited

30+

Solve FSHD

Collaborator

Trials

1

Recruited

100+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07086521

NCT07086521 | Safety and Preliminary Efficacy of ULSC in ... The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral ...

2. trialx.com trialx.com/clinical-trials/listings/307832/safety-and-preliminary-efficacy-of-ulsc-in-facioscapulohumeral-muscular-dystrophy-fshd/?&radius=50

Safety and Preliminary Efficacy of ULSC in ... 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.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8459640/

The use of umbilical cord‐derived mesenchymal stem cells ... The administration of Wharton's jelly‐derived mesenchymal stem cells in patients with muscular dystrophies improves average muscle strength measured with a ...

4. withpower.com withpower.com/trial/phase-1-muscular-dystrophy-facioscapulohumeral-7-2025-3d12f

Stem Cell Therapy for Facioscapulohumeral Muscular ... 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.

5. mdaquest.org mdaquest.org/simply-stated-updates-in-facioscapulohumeral-muscular-dystrophy-fshd/

Simply Stated: Updates in Facioscapulohumeral Muscular ... Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder that may affect all muscles across the lifespan of an individual.