NCT07601425 · Atavistik Bio, Inc
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
What this study is about
This is a 2-part study evaluating ATV-1601 in participants with moderate to severe HHT. Part 1 is a randomly assigned, where neither patients nor doctors know which treatment is given, compared against an inactive treatment study evaluating 3 dosing regimens of ATV-1601.
View original scientific description
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.
Interventions
DRUG
ATV-1601
Administered orally, daily
DRUG
Placebo
Administered orally, daily
Primary outcome measures
Part 1: Safety and tolerability
Time frame: 16 weeks
Number and severity of treatment-emergent adverse events (TEAEs) and study drug-related TEAEs
Part 2: Safety and tolerability
Time frame: 24 months
Type, incidence, severity, timing, seriousness and relatedness of AEs and laboratory abnormalities
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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)
Where
- Phoenix, Arizona
- Augusta, Georgia
- Indianapolis, Indiana
- Boston, Massachusetts
- Rochester, Minnesota
- Salt Lake City, Utah
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 1, 2026 · Source of record for eligibility and locations