At a glance
ClinicalIndex Comparison RecordStandardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Sirolimus for Nosebleeds in HHT: A Phase II Pilot Study
In Brief
A Phase 2 clinical trial evaluating Sirolimus for Hereditary Hemorrhagic Telangiectasia and 2 related conditions. Completed, enrolled 10 participants across 1 site.
Signals
Detailed 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.
Study Details
Timeline
Arms & Interventions
All participants received Sirolimus and were followed over the 9-month study period (3-month baseline, 3-month treatment period, 3-month follow up period). During 3-month treatment period, oral sirolimus was provided with a target blood trough of 6-10 ng/ml
Interventions
Oral sirolimus provided with starting dose of 2mg once daily, adjusted to maintain drug blood levels of 6-10 ng/ml (3-month course)