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Tests treatment safety and results for FSHD - Facioscapulohumeral Muscular Dystrophy
Official title Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
ClinicalTrials.gov ID: NCT07086521
What this study is testing
What is ULSC?
ULSC is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fshd - facioscapulohumeral muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 15 and older
You may be able to join if
- 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...
- Participant will have the ability to comply with the requirements of the study, including MRI.
- All participants of reproductive age/capacity will be required to use adequate contraception, defined as two forms of highly effective...
You likely can't join if
- 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...
- Treatment with an investigational product within three months prior to randomization.
- Known active opportunistic or life-threatening infections including Human Immunodeficiency virus (HIV) and hepatitis B or C.
- Known active or inactive tuberculosis infection.
See the full eligibility criteria
- 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.
- All participants of reproductive age/capacity will be required to use adequate contraception, defined as two forms of highly effective contraceptives, with any partners during the study period and for at least three...
- Participant will have the ability to understand and provide written informed consent.
- For those participants who are on drug(s) or supplements that may affect muscle function, as determined by the Investigator, participants must be on a stable dose of that drug(s) or supplement for at least 3 months...
- Reduced upper arm strength as measured by the Performance of Upper Limb score of ≤5.
- Current and up-to-date immunizations.
- Total relative reachable surface area (RSA) (Q1-Q4) without weight in the dominant upper extremity assessed by reachable workspace (RWS) ≥ 0.2 and ≤ 0.7.
- No contraindications to MRI.
- Hematocrit of ≤ 50%
- Prostate-specific antigen ≤ 4.0 ng/mL (or ≤ 3.0 ng/mL if the participant has a first-degree relative with prostate cancer)
- Fasting blood glucose \<126 mg/dL
- 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.
- Known active opportunistic or life-threatening infections including Human Immunodeficiency virus (HIV) and hepatitis B or C.
- Known active or inactive tuberculosis infection.
- Use of a product that putatively enhances muscle growth or activity on a chronic basis within 4 weeks before baseline
- Orally administered cytochrome P450 (CYP3A4) substrates and multidrug and toxin extrusion (MATE) and organic anion transporter (OAT)3 substrates are not permitted as concomitant therapy.
- Statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline (stable, chronic statin use is permissible).
- Rapamycin treatment within 3 months before baseline.
- Evidence of an alternative diagnosis other than FSHD or a coexisting myopathy or dystrophy, based on prior muscle biopsy or other available investigations.
- Muscle biopsy within 30 days before baseline.
- A systolic blood pressure over 160 or a diastolic pressure over 100
- Heavy alcohol use (greater than 50g/day)
- Current testosterone or HGH use
- Current use of medications that interfere with the growth hormone or gonadal endocrine axis.
- Pregnant of lactating participants.
- Concomitant severe cardiac, pulmonary disease, active infection, or other conditions that preclude assessment of safety and how well it works of the study product.
- Anticipated need for surgery during the trial period.
- A history of prevalent noncompliance with medical therapy.
- Recipient of an organ transplant.
- Neutropenia (absolute neutrophil count \<1,800/mm\^3 [or \<1,000/mm\^3 in African-American participants]).
- Severe impairment in renal function (estimated glomerular filtration rate \<30 ml/kg\ min).
- Recent of planned use of vaccination with live attenuated viruses.
- Condition that would impair an assessment of muscle strength, including neurological disorders such as Parkinson's disease or severe musculoskeletal condition.
The study team makes the final eligibility decision.
Where it's taking place
- Palo Alto, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 15 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Palo Alto, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.