NCT07601425PHASE1, PHASE2RecruitingAccepts Healthy Volunteers

Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)

Atavistik Bio, Inc

Start Date

6/1/2026

Completion Date

3/1/2030

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.

Detailed Description

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.

Eligibility Criteria

Age Range: 18 years to No maximum

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)

Interventions

DRUG

ATV-1601

DRUG

Placebo

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Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)Osler Weber Rendu Disease

Locations

Massachusetts General Hospital

Boston, Massachusetts 02114

United States