Arrowhead and Sarepta’s SRP-1001 Trial

Quick facts. Drug: SRP (formerly ARO-DUX4). How Is It Given? Intravenous injection. Phase: 1/2a. Participants: 60. Placebo: Yes, 3:1. Genetic Testing: Required, provided if needed. Rx Schedule: Part 1: duration 3 months. Part 2: 4 doses over 1 year. Study Visits: Approximately 20. Notable Activities: MRI, leg muscle biopsy. Open-Label Extension: Yes, if warranted by study results. Eligibility. Age 16-70. FSHD1. Clinical Severity Scale 3-8. Must have leg muscle suitable for biopsy & be able to do MRI. Status. Currently enrolling. Locations: New Zealand, Australia, Canada, Europe, Thailand. Data expected: TBD. Trial ID: NCT06131983.

在 ClinicalTrials.gov 上查看

Study Overview

基础知识

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

临床试验的目的是什么？
This is a Phase 1/2a study of a medicine called ARO-DUX4. The purpose of the study is to:

  • Understand how ARO-DUX4 acts in the human body
  • Evaluate how safe ARO-DUX4 is and what the side effects of taking it might be

药物如何发挥作用？
FSHD is caused by expression of the DUX4 gene in muscles. ARO-DUX4 reduces the expression of DUX4. It belongs to a class of medicines called RNA therapeutics.

是否会给一些人服用安慰剂而不是药物？

是的，这是一项安慰剂对照研究。部分患者将接受安慰剂而非药物。.

我是否知道自己服用的是药物还是安慰剂？
不，你不会知道。这是一项双盲研究。患者和研究小组不会知道谁服用了药物，谁服用了安慰剂。.

如何给药？
ARO-DUX4 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. Each infusion could take 1 to 4 hours. In Part 1 of the study, participants will receive a single dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 2 or 4 doses of ARO-DUX4 or placebo.

临床试验需要多长时间？ 
In Part 1, participants will be monitored for adverse events for 90 days. In Part 2, participants will be monitored for adverse events for 360 days.

Please see “What to Expect if You Enroll” for more information about the extension study.

资格

参加临床试验的标准是什么？ 
你必须

  • 16至70岁
  • be genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record
  • have clinical severity score between 3 and 8 (scale, 0 to 10)
  • have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader
  • a 12-lead electrocardiogram (ECG) at screening with no abnormalities that may compromise participant’s safety in the study

研究小组将对您进行以下评估

  • 上下肢肌肉无力
  • an area of 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

  • Were diagnosed with infantile FSHD
  • Have certain medical conditions, such as obesity, bleeding disorders, or other skin or muscle conditions
  • 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. If you don’t have one, the study will be able to provide the test at no cost to you.

研究小组如何确定我能否参加研究？ 
第一步通常是通过电话进行预选，了解申请资格标准。研究机构的协调员会给您打电话。初次通话后，协调员可能会要求您完成其他预选步骤。如果您符合条件，您的协调员将为您安排一次面对面的筛查访问。.

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 ~20 study visits. Some of these can be done virtually.

我是否需要在探访时过夜？
有些患者可能需要留院过夜。其他患者可能需要在输液 24 小时后返回研究地点进行随访。.

成本和付款

我是否需要支付任何医疗费用？我的保险会收到账单吗？ 
No, all costs are paid for by Arrowhead Pharmaceuticals.

我能报销差旅费吗？
Yes. Arrowhead Pharmaceuticals will arrange and pay for airfare, lodging, and ground transportation for you and one caregiver. You may also be reimbursed for reasonable expenses related to the study, such as meals and parking.

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

如何注册

需要多少志愿者？ 
The study seeks 52 volunteers with FSHD who meet the eligibility criteria.

开展临床试验的研究机构在哪里？ 
The study will be conducted in Canada, New Zealand, Australia, Thailand, Germany, Italy. Netherlands, and Spain. Additional global sites are being considered. Please check ClinicalTrials.gov for additional sites in the future.

如何联系我的学习地点以了解更多信息并注册？
To find specific site contact information, visit 临床试验.

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 ARO-DUX4 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