Target FSHD

Quick Facts. Drug Clenbuterol. How Is It Given? Orally. Phase 1. Participants 30. Placebo No. Genetic Testing Required (for participant or first-degree relative). Rx Schedule Twice daily for 6 months. Study Visits 5 in-person and 1 follow-up call. Notable Activities MRI, leg muscle biopsy. Open-Label Extension No. Eligibility. Age 18-75, FSHD1 or FSHD2, Clinical severity score 1 to 4, Must be able to walk 30ft unassisted, Must be able to do MRI. Status. Currently enrolling. Locations: US (Kansas, New York, Washington). Data expected in 2028 Trial ID: NCT06721299在 ClinicalTrials.gov 上查看

Study Overview

基础知识

谁在进行临床试验？
The University of Kansas Medical Center is conducting the study.

临床试验的目的是什么？
Target FSHD is a Phase 1 study of a medicine called Clenbuterol. The purpose of the study is to:

  • Evaluate how safe Clenbuterol is and what the side effects of taking it might be at different doses
  • Explore outcome measures that could be used to measure efficacy in a future Phase 2 study
  • Explore if Clenbuterol can be repurposed to both stop disease progression and restore motor function

药物如何发挥作用？
In FSHD, the DUX4 gene becomes active in muscles when it normally should be silent. Clenbuterol may help by turning down the harmful signals caused by DUX4 inside muscle cells. It works through beta‑2 receptors in muscle and is part of a group of medications called beta‑2 agonists.

是否会给一些人服用安慰剂而不是药物？
No, all patients will receive the medicine.

如何给药？
Clenbuterol will be taken orally twice daily.

临床试验需要多长时间？
The trial will take 8-9 months with patients taking the medicine for 6 months.

资格

参加临床试验的标准是什么？
你必须

  • 患有 FSHD1 或 FSHD2
  • have a genetic test confirming your FSHD1 or FSHD2 or a first-degree relative’s FSHD1
  • be between 18 and 75 years old
  • show some signs of muscle weakness due to FSHD
  • be able walk 30 meters unassisted
  • be able to undergo an MRI and have an MRI eligible muscle for needle biopsy

研究小组将对您进行以下评估

  • the severity of your FSHD, using the Ricci Clinical Severity Scale
  • an area of leg muscle that can undergo a biopsy.

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

  • Take certain medications, such as beta-blockers, anabolic agents, potassium-wasting diuretics, blood thinners, or GLP-1 agonists
  • 怀孕或计划怀孕
  • 其他因素可能会将您排除在外。您可以在以下网站查看更多排除标准 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 or you must have a first degree relative with a genetic report showing confirmation of FSHD1. If you don’t have one, the study team may be able to help you get one. However, the study will not cover the cost of your genetic test.

We recommend you work with your doctor to start the process of getting a genetic test before trying to join this clinical trial. Genetic testing can take many weeks to complete. Because of this, the study team may give priority to patients who are already tested, or who have already started the process. 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 to provide medical records to the site for review. Next a pre-screening phone call to talk through the eligibility criteria will be completed bya coordinator at your study site. After this initial call, your coordinator may have additional pre-screening steps for you to complete. If you are a good fit, your coordinator will schedule an in-person screening visit.

您的筛查访问将在研究地点进行。在这次就诊中，您将了解更多关于这项研究的信息，并同意继续进行研究。然后，研究小组将为您进行所有必要的检查，以确定您是否可以参加研究。这可能需要几个小时，或者您可能需要多次就诊。所有检查结果出来后，研究小组会告诉您是否可以参加研究。这一筛选过程可能需要长达 6 周的时间。.

报名后的注意事项

我需要参加哪些活动？

  • 提供血液和尿液样本
  • 提供 2 份肌肉活检样本。这需要使用针头抽取一小块肌肉，大小约为一块长粒大米。手术使用局部麻醉剂
  • 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 5 in-person visits and complete 1 safety follow-up call.

我是否需要在探访时过夜？
An overnight stay is not required for the study.

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?
No, all costs are paid for by University of Kansas Medical Center.

我能报销差旅费吗？
Yes, reimbursement will be available for travel after approval of receipts.

Will I be paid a stipend for being in the clinical trial?
Yes, subjects will receive a $50 stipend for every on-site visit completed.

谁来支付临床试验的费用？
The study is funded by the National Institute of Arthritis and Musculoskeletal and Skin Disease through a research grant awarded at the University of Kansas Medical Center.

如何注册

需要多少志愿者？
The study needs 30 participants. However, many more volunteers are needed because not everyone who volunteers will be eligible and able to take part.

开展临床试验的研究机构在哪里？
The study is conducted across multiple locations in the United States. To find specific sites, visit 临床试验 and click on “Contacts and Locations” in the sidebar to open up 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 up 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 Target FSHD 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？
可能需要数周或数月才能收到回复，请耐心等待。研究机构可能仍在为研究做准备，可能还无法与任何人取得联系。有时，研究机构会因为有更多感兴趣的患者而招收不过来。请确保提供您的电子邮件和电话号码，以便协调员更容易与您联系。您还应坚持每月报到一次，这有助于研究小组了解您对完成临床试验的承诺。.

更多信息