Restem

Quick Facts. Drug Restem-L. How Is It Given? Intravenous infusion. Phase 1. Participants 16. Placebo Yes, all participants receive 2 doses of placebo and 2 doses of drug. Genetic Testing Required. Rx Schedule doses over 9 months. Study Visits 11 in-person and 5 virtual. Notable Activities MRI, cardiopulmonary assessments. Open-Label Extension No. Eligibility. Age 15 and older, FSHD1 or FSHD2, Ricci score: ≥ 3 (using 0 - 10 scale), Able to walk without support, Must be able to do MRI, Reduced upper arm strength. Status. Currently enrolling. Locations: US (California). Trial ID: NCT07086521.

Ver en ClinicalTrials.gov

Study Overview

Lo básico

¿Quién lleva a cabo el ensayo clínico?
Restem is conducting the study.

¿Cuál es el objetivo del ensayo clínico?
Restem is a Phase 1 study utilizing umbilical cord lining-derived stem cell product (ULSC). The purpose of the study is to:

  • Check the safety of ULSC and monitor side effects, if any
  • Test whether ULSC can relieve symptoms of FSHD

¿Cómo actúa el medicamento?
ULSC works by supporting muscle repair and restoring immune system balance.

¿Se le dará a algunas personas un placebo en lugar del medicamento?
While this is a placebo-controlled study, all participants will receive two doses of ULSC and two doses of placebo in one of two sequences: ULSC first and placebo second, or placebo first and ULSC second.

¿Sabré si estoy recibiendo el medicamento o el placebo?
No, this is a double-blinded study.

¿Cómo se administrará el medicamento?
ULSC and the placebo will be given as an intravenous (IV) infusion. This means the medicine is given through a needle in a blood vein, usually in your arm.

¿Cuánto tiempo dura el ensayo clínico?
Participation in the trial is expected to last approximately 21 months. During the first 9 months, participants will receive two doses of study medication and two doses of placebo. This will be followed by an additional 12 months of follow-up assessments.

Requisitos de elegibilidad

¿Cuáles son los criterios para participar en el ensayo clínico?
Debes:

  • have genetic confirmation of FSHD1 or FSHD2
  • be 15 years old or older
  • be able to walk independently
  • be able to undergo an MRI

The study team will:

  • Assess your muscle strength
  • Evaluate the severity of your FSHD using the Ricci Clinical Severity Scale
  • Perform laboratory testing, including a complete blood count, metabolic panel, and fasting blood glucose levels

¿Qué criterios me excluirían del ensayo clínico?
No podrás participar si:

  • Have certain medical conditions, such as cancer, HIV, hepatitis B or C, tuberculosis, or Parkinson’s disease
  • Have high blood pressure (>160/100 mmHg) at screening
  • Are pregnant or plan to become pregnant · Other factors may exclude you. You can view more exclusion criteria on ensayos clínicos.gov.

¿Necesito hacerme una prueba genética para participar? Si es así, ¿me la proporcionarán?
Yes, you must have a genetic test confirming your diagnosis to be eligible for this study. Genetic testing can take many weeks to complete. Más información sobre las pruebas genéticas.

¿Cómo determinará el equipo del estudio si puedo participar en él?
The first step is usually a pre-screening discussion to talk through the study details, including the eligibility criteria. The study doctor or coordinator may have additional pre-screening steps for you to complete. If the team believes you may be a good fit, you will be scheduled for an in-person screening visit.

Your screening visit will take place at a study site. At this visit, you will learn more about the study and provide your consent to proceed. Then, the study team will perform all the tests needed to determine if you can take part in the study. This could take several hours, or you may need multiple visits. After all your test results come back, the study team will tell you whether you qualify for the study. This screening process can take up to 8 weeks.

Qué esperar si se inscribe

¿Qué actividades se me pedirá que realice?

  • Proporcionar muestras de sangre.
  • Someterse a resonancias magnéticas de cuerpo completo.
  • Realice una serie de pruebas para evaluar su fuerza y función muscular.
  • Responda cuestionarios sobre los síntomas relacionados con la FSHD y cómo se siente.
  • Utilice métodos anticonceptivos altamente eficaces. Si tiene la capacidad de quedar embarazada, se realizará pruebas de embarazo periódicas para confirmar que no lo está.

¿Cuántas visitas de estudio hay? ¿Son presenciales o virtuales?
You will be asked to attend 11 in-person visits and 5 virtual visits.

¿Tendré que pasar la noche en el hospital para alguna visita?
In-patient stays are not required; however, study visits can span several days.

¿Se ofrecerá el medicamento a los pacientes una vez finalizado el ensayo clínico?
No.

Costos y pagos

¿Tendré que pagar algún gasto médico? ¿Se le cobrarán los gastos a mi seguro?
All costs associated with the trial are paid for by Restem. Your insurance may be billed for standard of care costs that aren’t part of the study.

¿Me reembolsarán los gastos de viaje?
Travel may be reimbursed for eligible patients. Check with the study site for confirmation based on your needs.

Will I be paid a stipend for being in the clinical trial?
A stipend is paid to cover your time and effort in participating in this trial. The amount varies per visit.

¿Quién paga el ensayo clínico?
Restem is paying for the study.

Cómo registrarse

¿Cuántos voluntarios se necesitan?
The study needs 16 patients. However, many volunteers are needed because not everyone who volunteers will be able to participate.

¿Dónde se encuentran los centros de estudio que están llevando a cabo el ensayo clínico?
The study is conducted at Stanford University in Palo Alto, California in the US. To find the specific site, visit ensayos clínicos.gov and click on “Contacts and Locations” in the sidebar to open a list of locations. More locations may open as the study progresses.

¿Cómo puedo contactar con mi centro de estudio para obtener más información e inscribirme?
Para encontrar sitios específicos, visite ensayos clínicos.gov and click on “Contacts and Locations” in the sidebar to open a list of locations.

If your study site is marked as “Recruiting,” there should be an email and/or phone number listed for contact. Sometimes there is also contact information listed for sites that are “Not Yet Recruiting.” You should email and call these contacts and let them know that you are interested in the Restem study. If you are able and willing to travel to multiple different sites, you should contact each site individually.

¿Qué sucederá después de que me comunique con el centro de estudio?
It can take several weeks to hear back, so be patient. Make sure you provide your email and phone number to make it easier for the coordinators to contact you. You should also keep checking in on a monthly basis – this helps the study team know that you are committed to completing the clinical trial.

Más información