Palo Alto, CANCT07086521Now EnrollingIRB Ready

FSHD - Facioscapulohumeral Muscular Dystrophy Clinical Trial in Palo Alto, CA

Access cutting-edge fshd - facioscapulohumeral muscular dystrophy treatment through this clinical trial at a research site in Palo Alto. Study-provided care at no cost to qualified participants.

Sponsored by Restem, LLC.

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This study follows strict safety protocols and ethical guidelines

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All study-related fshd - facioscapulohumeral muscular dystrophy treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to Palo Alto

    Convenient for CA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Palo Alto site if eligible
  4. 4Begin participation

About This FSHD - Facioscapulohumeral Muscular Dystrophy Study in Palo Alto

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.

Sponsor: Restem, LLC.

Who Can Participate

Inclusion 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 months beyond the study period for safety.
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 prior to the first dose of study drug and remain on that stable dose for the duration of the study. This includes the following drug category: o Immunomodulatory agents, including targeted biological therapies.
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

Exclusion Criteria

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 efficacy 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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Palo Alto?

Yes, this clinical trial (NCT07086521) has an active research site in Palo Alto, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

FSHD - Facioscapulohumeral Muscular Dystrophy Treatment Options in Palo Alto, CA

If you're searching for fshd - facioscapulohumeral muscular dystrophy treatment options in Palo Alto, CA, this clinical trial (NCT07086521) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Palo Alto research site is actively enrolling participants for this clinical trial. You'll receive care from experienced fshd - facioscapulohumeral muscular dystrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all fshd - facioscapulohumeral muscular dystrophy clinical trials near you to find additional studies recruiting in your area.

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