Overview

Sirolimus for Nosebleeds in HHT

Status:
Recruiting
Trial end date:
2027-02-10
Target enrollment:
Participant gender:
Summary
This pilot study is to determine the safety and efficacy of oral sirolimus (blood trough level 6-10ng/ml) in patients with HHT that are experiencing moderate or severe epistaxis. The effect of oral sirolimus on epistaxis will be compared to baseline using the Patient-Reported Outcome of cumulative weekly nose Bleeding Duration (PRO-CB). The PRO-CB association with biomarker variability over the duration of the study will be investigated. In the pilot study subjects will be treated with 2mg of sirolimus once daily to obtain a trough level of 6-10ng/ml for 3 months.
Phase:
Phase 2
Details
Lead Sponsor:
Unity Health Toronto
Collaborator:
National Institutes of Health (NIH)
Treatments:
Sirolimus