NCT07601425

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.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P75+ for phase_1

Timeline
46mo left

Started Jun 2026

Typical duration for phase_1

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 12, 2026

Completed
10 days until next milestone

First Posted

Study publicly available on registry

May 22, 2026

Completed
10 days until next milestone

Study Start

First participant enrolled

June 1, 2026

Expected
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

2.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2030

Last Updated

May 22, 2026

Status Verified

May 1, 2026

Enrollment Period

1.5 years

First QC Date

May 12, 2026

Last Update Submit

May 15, 2026

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)Osler Weber Rendu Disease

Keywords

Arteriovenous malformationsATV-1601HarmonyHHTHarmony-HHTHarmonyHHTStudyVascular DiseasesBleeding disordersHematologic disordersDisordersTelangiectasiasHereditary hemorrhagicHemorrhagic disordersCardiovascular diseasesVascular malformationsHHT

Outcome Measures

Primary Outcomes (2)

  • Part 1: Safety and tolerability

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

    16 weeks

  • Part 2: Safety and tolerability

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

    24 months

Secondary Outcomes (21)

  • Part 1: Change in Epistaxis duration

    16 weeks

  • Part 1: Epistaxis frequency

    16 weeks

  • Part 1: Epistaxis intensity

    16 weeks

  • Part 1: Intensity-weighted epistaxis duration

    16 weeks

  • Part 1: Epistaxis Severity Score (ESS)

    16 weeks

  • +16 more secondary outcomes

Study Arms (4)

Part 1: 60 mg QD

EXPERIMENTAL

Active drug, once daily

Drug: ATV-1601

Part 1: 100 mg QD

EXPERIMENTAL

Active drug, once daily

Drug: ATV-1601

Part 1: 60 BID

EXPERIMENTAL

Active drug, twice daily

Drug: ATV-1601

Part 1: Placebo

EXPERIMENTAL

Control Arm

Drug: Placebo

Interventions

ATV-1601DRUG

Administered orally, daily

Also known as: Active drug, capsule
Part 1: 100 mg QDPart 1: 60 BIDPart 1: 60 mg QD
PlaceboDRUG

Administered orally, daily

Also known as: Inactive, Placebo
Part 1: Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Telangiectasia, Hereditary HemorrhagicArteriovenous MalformationsVascular DiseasesHemostatic DisordersHematologic DiseasesDiseaseTelangiectasisHemorrhagic DisordersCardiovascular DiseasesVascular Malformations

Interventions

Capsules

Condition Hierarchy (Ancestors)

Hemic and Lymphatic DiseasesCardiovascular AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Dosage FormsPharmaceutical Preparations

Central Study Contacts

Patrick McNamara

CONTACT

857-285-5400patrickmcnamara@atavistikbio.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Participants will be randomized to one of 3 doses of ATV-1601 or placebo
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 12, 2026

First Posted

May 22, 2026

Study Start (Estimated)

June 1, 2026

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

March 1, 2030

Last Updated

May 22, 2026

Record last verified: 2026-05

Data Sharing

IPD Sharing
Will not share