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.

在 ClinicalTrials.gov 上查看

Study Overview

基础知识

谁在进行临床试验？
Restem is conducting the study.

临床试验的目的是什么？
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

药物如何发挥作用？
ULSC works by supporting muscle repair and restoring immune system balance.

是否会给一些人服用安慰剂而不是药物？
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.

我是否知道自己服用的是药物还是安慰剂？
No, this is a double-blinded study.

如何给药？
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.

临床试验需要多长时间？
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.

资格

参加临床试验的标准是什么？
你必须

  • 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

What criteria would exclude me from the clinical trial?
如果您有以下情况，将无法参加比赛

  • 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 临床试验.

我是否需要进行基因测试才能加入？如果需要，是否会提供？
Yes, you must have a genetic test confirming your diagnosis to be eligible for this study. Genetic testing can take many weeks to complete. Learn more about genetic testing.

研究小组如何确定我能否参加研究？
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.

报名后的注意事项

我需要参加哪些活动？

  • Provide blood samples
  • 接受全身核磁共振成像扫描
  • 进行一系列测试，评估您的肌肉力量和功能
  • 回答有关前列腺增生症相关症状和感觉的调查问卷
  • 采取高效的避孕措施。如果您有怀孕的能力，您将定期进行妊娠测试，以确认您没有怀孕。.

研究访问有多少次？是面对面的还是虚拟的？
You will be asked to attend 11 in-person visits and 5 virtual visits.

我是否需要在探访时过夜？
In-patient stays are not required; however, study visits can span several days.

临床试验结束后，会向患者提供这种药物吗？
No.

成本和付款

Will I have to pay for any medical expenses? Will my insurance be billed for any costs?
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.

我能报销差旅费吗？
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.

谁来支付临床试验的费用？
Restem is paying for the study.

如何注册

需要多少志愿者？
The study needs 16 patients. However, many volunteers are needed because not everyone who volunteers will be able to participate.

开展临床试验的研究机构在哪里？
The study is conducted at Stanford University in Palo Alto, California in the US. To find the specific site, visit 临床试验 and click on “Contacts and Locations” in the sidebar to open a list of locations. More locations may open as the study progresses.

如何联系我的学习地点以了解更多信息并注册？
要查找具体地点，请访问 临床试验 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.

与研究机构联系后会发生什么？
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.

更多信息