A Randomized, Placebo-Controlled, Double-Blind, Proof-of-Concept Study of ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
Atavistik Bio, Inc
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.
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 rece…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- Yes
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 gluc…
Interventions
- DrugATV-1601
Administered orally, daily
- DrugPlacebo
Administered orally, daily
Location
- Massachusetts General HospitalBoston, Massachusetts