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