Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT) (NCT07601425) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
100 participantsStarted 2026-06
Plain-language summary
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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.