Texas Children's Cancer and Hematology Center Harmony-HHT: a Proof-of-Concept Study of ATV-1601 in Participants with Hereditary Hemorrhagic Telangiectasia (HHT)
Conditions
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
Description
This is a 2-part study evaluating ATV-1601, an investigational oral precision therapy that selectively inhibits the AKT1 protein, 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.
Eligibility
- Age 18 years and older
- Confirmed hereditary hemorrhagic telangiectasia (HHT)
- Moderate-to-severe disease with Epistaxis Severity Score (ESS) ≥4
- Anemia and/or at least one red-cell transfusion unit within previous 6 months
- Adequate hematologic, renal, and hepatic function
- Able to complete double-blind treatment period for open-label extension eligibility
Detailed inclusion and exclusion criteria are listed at clinicaltrials.gov.
