Recruiting
Phase 1
Phase 2

ATV-1601

Sponsor:

Atavistik Bio, Inc

Code:

NCT07601425

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)

Osler Weber Rendu Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Accepted

Interventions

ATV-1601

Placebo

Study Details

Brief 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.

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)

Osler Weber Rendu Disease

Study ID

NCT07601425

Start date

Jul 27, 2026

Status verified date

Aug, 2026

Completion date

Mar, 2030

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Accepted

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

Enrollment

100 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Part 1: 60 mg QD

Active drug, once daily

experimental: Part 1: 100 mg QD

Active drug, once daily

experimental: Part 1: 60 BID

Active drug, twice daily

experimental: Part 1: Placebo

Control Arm

Interventions

ATV-1601

Administered orally, daily

Placebo

Administered orally, daily

Primary outcome measure

  • Part 1: Safety and tolerability [ Time Frame: 16 weeks ]
  • Part 2: Safety and tolerability [ Time Frame: 24 months ]

Central Contacts and Locations

Central contacts

Study Director

857-285-5400Studydirector@atavistikbio.com

Locations

Arizona Pulmonary Specialists

Recruiting

Phoenix, Arizona, United States, 85013

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

More Information

Sponsor

Atavistik Bio, Inc

Last update posted

Sep 1, 2026

Last verified

Aug, 2026

Keywords

  • Arteriovenous malformations
  • ATV-1601
  • HarmonyHHT
  • Harmony-HHT
  • HarmonyHHTStudy
  • Vascular Diseases
  • Bleeding disorders
  • Hematologic disorders
  • Disorders
  • Telangiectasias
  • Hereditary hemorrhagic
  • Hemorrhagic disorders
  • Cardiovascular diseases
  • Vascular malformations
  • HHT

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Atavistik Bio, Inc on 2026-09-01.