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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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 |
|
Recruiting Locations
Boston, Massachusetts 02114
Study Director
More Details
- Status
- Recruiting
- Sponsor
- Atavistik Bio, Inc
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.