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Recruiting NCT07601425

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

Phase I / Phase II Interventional Hereditary Hemorrhagic Telangiectasia (HHT) Osler Weber Rendu Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ATV-1601, Placebo.
Who it may be relevant to
Registry conditions: Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Randomized, Placebo-Controlled, Double-Blind, Proof-of-Concept Study of ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)

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.

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.

Interventions

  • Drug ATV-1601
    Administered orally, daily
  • Drug Placebo
    Administered orally, daily

Primary outcome measures

  • Part 1: Safety and tolerability [Time frame: 16 weeks]
  • Part 2: Safety and tolerability [Time frame: 24 months]
Secondary outcome measures (12)
  • Part 1: Change in Epistaxis duration [Time frame: 16 weeks]
  • Part 1: Epistaxis frequency [Time frame: 16 weeks]
  • Part 1: Epistaxis intensity [Time frame: 16 weeks]
  • Part 1: Intensity-weighted epistaxis duration [Time frame: 16 weeks]
  • Part 1: Epistaxis Severity Score (ESS) [Time frame: 16 weeks]
  • Part 1: Change in Hemoglobin [Time frame: 16 weeks]
  • Part 1: Change in Parenteral iron use [Time frame: 16 weeks]
  • Part 1: Change in Blood transfusion requirements [Time frame: 16 Weeks]
  • Part 1: Pharmacokinetics - Maximum observed concentration (Cmax) [Time frame: 16 Weeks]
  • Part 1: Pharmacokinetics - Area under the concentration-time curve over the dosing interval (AUCtau) [Time frame: 16 Weeks]
  • Part 1: Pharmacokinetics - Area under the concentration-time curve extrapolated to infinity (AUCinf) [Time frame: 16 Weeks]
  • Part 1: Pharmacokinetics - Time to maximum concentration (Tmax) [Time frame: 16 Weeks]

Eligibility criteria

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)

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 1 center
  • Massachusetts General Hospital — Boston

Identifiers

NCT: NCT07601425 · ATV-1601-102 · Harmony-HHT

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗