MedTrace Logo

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

NCT07086521 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2025-12-08

No results posted yet for this study

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.

Conditions

  • FSHD - Facioscapulohumeral Muscular Dystrophy

Interventions

BIOLOGICAL

ULSC

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).

BIOLOGICAL

Placebo

The Placebo will be 250 ml of sterile saline for IV administration.

Sponsors & Collaborators

  • Solve FSHD

    collaborator UNKNOWN

  • Restem, LLC.

    lead INDUSTRY

Principal Investigators

  • John W Day, MD, PhD · Stanford University, School of Medicine, Neuromuscular Research Division

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-12-01
Primary Completion
2028-09-30
Completion
2029-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07086521 on ClinicalTrials.gov