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Sirolimus for Nosebleeds in HHT
Sponsor: Unity Health Toronto
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.
Official title: Sirolimus for Nosebleeds in HHT: A Phase II Pilot Study
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2022-03-16
Completion Date
2024-12-02
Last Updated
2026-06-04
Healthy Volunteers
No
Interventions
Sirolimus
Oral sirolimus provided with starting dose of 2mg once daily, adjusted to maintain drug blood levels of 6-10 ng/ml (3-month course)
Locations (1)
St. Michael's Hospital
Toronto, Ontario, Canada