Summary
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.
Official Title
A Randomized, Placebo-Controlled, Double-Blind, Proof-of-Concept Study of ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
Details
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.
Keywords
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease, Arteriovenous malformations, ATV-1601, HarmonyHHT, Harmony-HHT, HarmonyHHTStudy, Vascular Diseases, Bleeding disorders, Hematologic disorders, Disorders, Telangiectasias, Hereditary hemorrhagic, Hemorrhagic disorders, Cardiovascular diseases, Vascular malformations, HHT, Hereditary Hemorrhagic Telangiectasia, Hemostatic Disorders, Hematologic Diseases, Disease, Telangiectasis, Capsules