Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
Recruiting now · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
In brief
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.
Key facts
- Study ID
- NCT07601425
- Run by
- Atavistik Bio, Inc
- People needed
- 100
- Starts
- 2026-06-01
- Expected to finish
- 2030-03-01
- Last updated by the study team
- 2026-07-30
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- 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
You may not qualify if…
- 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)
Where it is running
- Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)
Full record on ClinicalTrials.gov
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