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.

Voir sur ClinicalTrials.gov

Study Overview

Les bases

Qui mène l'essai clinique ?
Restem is conducting the study.

Quel est l'objectif de l'essai clinique ?
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

Comment le médicament agit-il ?
ULSC works by supporting muscle repair and restoring immune system balance.

Certaines personnes recevront-elles un placebo au lieu du médicament ?
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.

Saurai-je si je reçois le médicament ou le placebo ?
No, this is a double-blinded study.

Comment le médicament sera-t-il administré ?
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.

Quelle est la durée de l'essai clinique ?
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.

Éligibilité

Quels sont les critères d'inclusion dans l'essai clinique ?
Vous devez :

  • 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

Quels sont les critères qui m'excluraient de l'essai clinique ?
Vous ne pourrez pas participer si vous.. :

  • 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 clinicaltrials.gov.

Dois-je subir un test génétique pour être inclus ? Dans l'affirmative, ce test sera-t-il fourni ?
Yes, you must have a genetic test confirming your diagnosis to be eligible for this study. Genetic testing can take many weeks to complete. En savoir plus sur les tests génétiques.

Comment l'équipe de l'étude déterminera-t-elle si je peux participer à l'étude ?
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.

Ce à quoi vous pouvez vous attendre si vous vous inscrivez

Quelles sont les activités qui me seront demandées ?

  • Fournir des échantillons de sang
  • Passer des examens IRM du corps entier
  • Effectuer une série de tests pour évaluer la force et la fonction musculaires.
  • Répondre à des questionnaires sur les symptômes liés à la FSHD et sur la façon dont vous vous sentez.
  • Utilisez une méthode de contraception très efficace. Si vous avez la capacité de tomber enceinte, vous ferez régulièrement des tests de grossesse pour confirmer que vous n'êtes pas enceinte.

Combien de visites sont prévues dans le cadre de l'étude ? Sont-elles en personne ou virtuelles ?
You will be asked to attend 11 in-person visits and 5 virtual visits.

Devrai-je passer la nuit sur place lors des visites ?
In-patient stays are not required; however, study visits can span several days.

Le médicament sera-t-il proposé aux patients une fois l'essai clinique terminé ?
No.

Coûts et paiements

Devrai-je payer les frais médicaux ? Les frais seront-ils facturés à mon assurance ?
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.

Les frais de voyage me seront-ils remboursés ?
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.

Qui finance l'essai clinique ?
Restem is paying for the study.

Comment s'inscrire

Combien de volontaires sont nécessaires ?
The study needs 16 patients. However, many volunteers are needed because not everyone who volunteers will be able to participate.

Où se trouvent les sites d'étude qui mènent l'essai clinique ?
The study is conducted at Stanford University in Palo Alto, California in the US. To find the specific site, visit clinicaltrials.gov and click on “Contacts and Locations” in the sidebar to open a list of locations. More locations may open as the study progresses.

Comment puis-je contacter mon site d'étude pour en savoir plus et m'inscrire ?
Pour trouver des sites spécifiques, visitez clinicaltrials.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.

Que se passera-t-il une fois que j'aurai contacté mon site d'étude ?
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.

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