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

Purpose

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.

Conditions

  • Hereditary Hemorrhagic Telangiectasia (HHT)
  • Osler Weber Rendu Disease

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts 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 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)

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Participants will be randomized to one of 3 doses of ATV-1601 or placebo
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Part 1: 60 mg QD 		Active drug, once daily
  • Drug: ATV-1601
    Administered orally, daily
    Other names:
    • Active drug, capsule
Experimental
Part 1: 100 mg QD 		Active drug, once daily
  • Drug: ATV-1601
    Administered orally, daily
    Other names:
    • Active drug, capsule
Experimental
Part 1: 60 BID 		Active drug, twice daily
  • Drug: ATV-1601
    Administered orally, daily
    Other names:
    • Active drug, capsule
Experimental
Part 1: Placebo 		Control Arm
  • Drug: Placebo
    Administered orally, daily
    Other names:
    • Inactive, Placebo

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114
Contact:
Study Director

More Details

Status
Recruiting
Sponsor
Atavistik Bio, Inc

Study Contact

Study Director
857-285-5400
Studydirector@atavistikbio.com

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.