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

Phase 2Not Yet RecruitingNCT07601425

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)

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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