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

Conditions

• FSHD - Facioscapulohumeral Muscular Dystrophy

Eligibility Criteria

Sex

ALL

Age

15 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• ULSC — BIOLOGICAL
  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).
• Placebo — BIOLOGICAL
  The Placebo will be 250 ml of sterile saline for IV administration.

Study Details

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.

Key Dates

Start date

Dec 1, 2025

Status verified

Dec 2025

Primary completion

Sep 30, 2028

Completion

Mar 31, 2029

Study Design

Enrollment

16 participants (estimated)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Experimental: Group 1: ULSC first; Placebo second
  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.
• Experimental: Group 2: Placebo first; ULSC second
  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).

Primary Outcome Measure

Adverse Events (AE) and Serious Adverse Events (SAEs) that begin during or following treatment infusion. [ Time Frame: 7 days and 30 days after each infusion, as well as 6 months after the final dose for each period (Month 9 and Month 15). ]

Locations (1)

Find similar trials in Palo Alto, CA

Related Studies

• A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
  PHASE3 · Recruiting · Avidity Biosciences, Inc. · Orange, California
• BetterLife FSHD: A Patient-driven Health and Research Platform
  Recruiting · FSHD Society · Randolph, Massachusetts