A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
报名条件（概要）
- 年龄：12 Years – 16 Years
- 性别：不限
- 健康志愿者：不接受
完整入排标准请以官方页面为准。
开展国家/地区
India、Nigeria、Oman
药品与企业信息
Participants will receive a dose of 400 mg (two 200 mg oral tablets) etavopivat once daily.
Participants will receive a stable dose of HU.
研发企业
试验官方登记的主要申办方；研究机构申办时会显示相应机构名称。
生产企业
公开药品批准资料或正式说明书暂未提供明确的生产企业信息。
研发企业与生产企业可能不是同一家。研究阶段药品可能尚未公布生产企业；生产企业仅取自美国上市原研药说明书所列企业（不含仿制药），不作推测；仅在其他国家上市的药品暂无法显示。
研究简介（原文）
The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke. Participants will be divided into two cohorts depending on their transcranial doppler (TCD) ultrasound results and whether or not they receive hydroxyurea (medication that they may already be taking). In one cohort, participants with conditional transcranial doppler (TCD) or participants with abnormal TCD who are not able to receive hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. In another cohort, participants with conditional TCD or participants with abnormal TCD who are receiving a stable dose of hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. The participant will start a 52-week (1 year) treatment period. The participant will take 400 milligrams (mg) of etavopivat once a day for the 52 weeks. The dose of 400 mg will be taken as 2 tablets by mouth, each containing 200 mg of etavopivat. Etavopivat may be taken with or without food. Each dose should be taken with a glass of water. As part of the study, the participants will be asked to visit the clinic frequently. The participant will have the opportunity to participate in a 48-week optional extension treatment period. The optional extension treatment period will allow continued as-sessment of safety of etavopivat in paediatric patients. At the end of the study, if deemed appro-priate the participant, the caregiver, and the study doctor, the participant may be offered the op-portunity to participate in a separate study to continue receiving etavopivat. If/when this separate study becomes available, the participant may only transfer to the new study after completion of the 52-week primary treatment period and at any time during the 48-week optional extension treatment period.
适应症
主动联系研究团队
这项试验目前在中国大陆没有研究中心
这种情况下，直接发"入组咨询"往往会被回复"您所在地区没有中心"。更有效的做法是申请同情用药／扩展准入（由主治医生一同提出），或询问能否到最近的境外中心参加。下面已为您切换到对应的信件版本。
查看完整申请路径与成功率说明 →总联系人（申办方／研究总部）
- Novo Nordiskclinicaltrials@novonordisk.com(+1) 866-867-7178
问自己是否符合条件、如何预筛
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英文版（发给研究团队）
Dear Study Team, I am a patient in China and I am interested in participating in your clinical trial: Study Title: A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke ClinicalTrials.gov Identifier: NCT05953584 Sponsor: Forma Therapeutics, Inc. Investigational product: Etavopivat, Hydroxyurea About me: - Age: (please fill in) - Sex: (please fill in) - Confirmed diagnosis and stage: (please fill in) - Biomarkers / genetic testing: not available - Prior and current treatments: (please fill in) - Performance status (ECOG): not assessed - Current location: China - Travel ability: (please fill in) Could you please let me know: 1. Whether I might be eligible for this study; 2. Which site would be closest and most practical for me, and whether remote pre-screening is possible; 3. What documents (medical records, pathology or imaging reports, recent labs) I should prepare for pre-screening. I can provide English translations of my medical records. Thank you very much for your time and help. Kind regards, (your name) (your email / phone with country code)
中文对照（供您核对）
尊敬的研究团队： 我是一位来自中国的患者，希望咨询参加以下临床试验的可能： 研究名称：A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke 试验编号：NCT05953584 申办方：Forma Therapeutics, Inc. 研究药物：Etavopivat、Hydroxyurea 我的基本情况： - 年龄：（请填写） - 性别：（请填写） - 确诊疾病与分期：（请填写） - 基因／标志物检测：暂无 - 既往及当前治疗：（请填写） - 体能状态（ECOG）：未评估 - 目前所在城市：中国 - 可前往范围：（请填写） 想请教三个问题： 1. 我是否可能符合本研究的入组标准？ 2. 哪个中心对我最方便可行？能否远程预筛？ 3. 预筛需要准备哪些资料（病历、病理、影像、近期化验）？ 我可以提供英文翻译版病历。感谢您的时间与帮助。 顺祝安康 （您的姓名） （您的邮箱／带国际区号的电话）
提示：请与主治医生一起发信——由医生署名的申请，回复率远高于患者单独发信。首封邮件不要附身份证号等敏感信息。研究团队通常在 3–10 个工作日内回复，两周无回音可礼貌追问一次。
下一步怎么做？
- 把本页信息带给您的主治医生，评估是否适合参加。
- 点击下方按钮打开官方注册页，查看研究中心联系方式。
- 直接联系研究团队咨询报名事宜（通常有中文同声翻译服务可协助沟通）。