Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age15+
SponsorRestem, LLC.
About this trial
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 criteria
Qualifiers
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.
Disqualifiers
Hypersensitivity to study product components including history of hypersensitivity to dimethyl sulfoxide (DMSO).
Active cancer or prior diagnosis of cancer within the past year (patients with basal and squamous cell cancer of skin will not be excluded).
Any other condition that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration, or follow-up.
Treatment with an investigational product within three months prior to randomization.
Trial design
Treatments tested in this trial
- ULSC
- Placebo
Treatment groups
16 Participants
are divided into 2 treatment groupsSponsors and collaborators
Restem, LLC.
Lead sponsor
Solve FSHD
Collaborator
Source ClinicalTrials.gov