High importance
Aug 21, 2026
To evaluate the safety and efficacy of the topical application of TOR-582 (sirolimus) for managing epistaxis in patients with hereditary hemorrhagic telangiectasia (HHT).
Adults with HHT experiencing frequent nosebleeds underwent a week-long observation period followed by up to 12 weeks of applying varying strengths of TOR-582 ointment twice daily in each nostril. Participants completed questionnaires, maintained a nosebleed diary, underwent nasal examinations, and had blood tests for safety monitoring.
The study aims to establish the safety profile of TOR-582 and determine the optimal dosing regimen through participant responses and clinical assessments over the treatment duration.
The primary limitation may include the short observational period of 12 weeks, which may not provide long-term data on efficacy or potential side effects. Variability in participant response to treatment could also affect results.
Effective and safe management of epistaxis in HHT can significantly improve patients' quality of life, reduce the need for invasive procedures, and potentially decrease anemia-related complications, thus addressing a critical unmet medical need in this patient population.
People with hereditary hemorrhagic telangiectasia (HHT) often experience frequent and severe nosebleeds that can disrupt daily life and lead to anemia, medical procedures, and reduced quality of life. This study is testing a new nasal ointment called TOR-582, which contains sirolimus, to determine whether it can be used safely when applied inside the nose. Adults with HHT and frequent nosebleeds will be invited to participate. Participants will first complete one week of observation without treatment, followed by up to 12 weeks of applying the study ointment inside each nostril twice daily. Different participants will receive different strengths of the ointment so researchers can identify the safest dose. During the study, participants will attend study visits, complete questionnaires about their nosebleeds and quality of life, keep a daily nosebleed diary, undergo nasal examinations, and have blood tests to monitor safety and medication levels. The information gained from this study will help determine whether this topical treatment can be safely studied further and will support the development of a new, less invasive option for managing nosebleeds in people with HHT.