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

Phase 2RecruitingNCT07601425

Atavistik Bio, Inc100 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

100

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)Osler Weber Rendu Disease

Interventions

ATV-1601DRUG

Administered orally, daily

PlaceboDRUG

Administered orally, daily

Eligibility

Sex: ALLMin age: 18 YearsHealthy volunteers:

Medical Language ↔ Plain English

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)

Locations (3)

Arizona Pulmonary Specialists

Phoenix, Arizona, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

RECRUITING

Outcomes

Primary Outcomes

Part 1: Safety and tolerability

Number and severity of treatment-emergent adverse events (TEAEs) and study drug-related TEAEs

Time frame: 16 weeks

Part 2: Safety and tolerability

Type, incidence, severity, timing, seriousness and relatedness of AEs and laboratory abnormalities

Time frame: 24 months

Secondary Outcomes

Part 1: Change in Epistaxis duration

28-day total duration compared to baseline

Time frame: 16 weeks

Part 1: Epistaxis frequency

28-day frequency of nosebleeds compared to baseline

Time frame: 16 weeks

Part 1: Epistaxis intensity

28-day average epistaxis intensity (6-point scale) of nosebleeds compared to baseline

Time frame: 16 weeks

Part 1: Intensity-weighted epistaxis duration

28-day intensity-weighted duration of nosebleeds

Time frame: 16 weeks

Part 1: Epistaxis Severity Score (ESS)

The Epistaxis Severity Score (ESS) is a validated 6-question instrument with scores ranging from 0 to 10, where higher scores indicate more severe epistaxis symptoms.

Time frame: 16 weeks

Part 1: Change in Hemoglobin

Hemoglobin levels compared to baseline

Time frame: 16 weeks

Central Contacts

Study Director

CONTACT

857-285-5400 Studydirector@atavistikbio.com

Data from ClinicalTrials.gov

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