A Randomized, Placebo-Controlled, Double-Blind, Proof-of-Concept Study of ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 100
- 试验地点
- 4
研究概览
简要总结
This is a 2-part study evaluating ATV-1601 in participants with moderate to severe HHT. Part 1 is a randomized, double-blind, placebo-controlled study evaluating 3 dosing regimens of ATV-1601. Patients completing Part 1 may participate in the Part 2 open-label extension to receive ATV-1601.
详细描述
Part 1: This is a Phase 1/2 proof-of-concept, double-blind, multicenter, placebo-controlled study to evaluate the safety, pharmacokinetics and efficacy of 3 oral dosing regimens of ATV-1601. Participants who meet eligibility requirements will be randomized in a double-blind manner to one of 3 doses of ATV-1601 or placebo. Participants will receive double-blind study treatment for a 16-week period.
Part 2: Eligible participants who complete Part 1 may enroll in an open-label extension study to receive up to 2 years of additional treatment. All participants in the open-label extension will receive ATV-1601. Once the recommended Phase 2 dose (RP2D) is determined based on Part 1, all participants in Part 2 will have the option to switch to the RP2D.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Ability to provide informed consent prior to any study-specific procedures
- •Confirmed diagnosis of hereditary hemorrhagic telangiectasia (HHT) based on Curaçao criteria
- •Moderate to severe HHT with an ESS ≥ 4
- •Anemia at Screening and/or requirement for at least 1 red-cell unit (RUE) in the previous 6 months
- •Adequate hematologic, renal, and hepatic function per protocol-defined laboratory criteria
- •Use highly effective contraception during the study and for a protocol-defined period after last dose
排除标准
- •Clinically significant abnormalities of glucose metabolism including diagnosed Type 1 or uncontrolled Type 2 diabetes
- •Chronic cardiac disease, or cardiac rhythm abnormalities
- •History of significant cardiovascular, hepatic, renal, or hematologic disease not related to HHT that may confound study results
- •Use of prohibited concomitant medications within a protocol-defined washout period prior to first dose (including strong CYP modulators and certain herbal supplements)
- •Recent (within 6 weeks) major surgery or local ablative procedures, or procedures on nasal telangiectasias
- •Prior AKT inhibitor
- •Pregnant or breastfeeding women
- •Additional Criteria for Open-Label Extension:
- •Participants must complete the double-blind treatment period (Part 1)
研究组 & 干预措施
Part 1: 60 BID
Active drug, twice daily
干预措施: ATV-1601 (Drug)
Part 1: 60 mg QD
Active drug, once daily
干预措施: ATV-1601 (Drug)
Part 1: Placebo
Control Arm
干预措施: Placebo (Drug)
Part 1: 100 mg QD
Active drug, once daily
干预措施: ATV-1601 (Drug)