High importance
Sep 1, 2026
To evaluate the efficacy and safety of ATV-1601 in patients with moderate to severe Hereditary Hemorrhagic Telangiectasia (HHT).
A two-part study design, where Part 1 is a randomized, double-blind, placebo-controlled trial assessing three different dosing regimens of ATV-1601. Participants who complete Part 1 can enroll in Part 2, an open-label extension where all will receive ATV-1601.
Outcomes from Part 1 will provide preliminary safety and efficacy data on ATV-1601 in reducing the severity and frequency of arteriovenous malformations (AVMs) and associated symptoms in HHT patients.
The study may be limited by sample size, potential placebo effects in Part 1, and the lack of long-term follow-up data post-treatment in Part 2.
HHT is a significant vascular disorder leading to life-threatening complications. This study could lead to new treatment options that specifically target the underlying pathophysiology of HHT, potentially improving patient care and quality of life for affected individuals.
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.