Sirolimus for HHT-Related Nosebleeds

Completed Phase 2 NCT05269849

Study Description

This pilot study investigates the effectiveness and safety of sirolimus for treating moderate or severe epistaxis (nosebleeds) in patients with hereditary hemorrhagic telangiectasia (HHT). HHT is characterized by vascular malformations that negatively affect an individual's quality of life. Topical therapeutics trials have been negative and surgical therapies offer only temporary relief, leaving a considerable need for effective systemic therapies. Sirolimus has been identified as a potential pathway-based therapy for HHT and has shown promising results as an mTOR inhibitor. In this pilot study, subjects will be administered 2mg of sirolimus once daily to obtain a trough level of 6-10ng/ml for three months. This study will provide safety and efficacy data to support a future randomized clinical trial of sirolimus for epistaxis in HHT patients.

Study Details

Registry ID:
NCT05269849
Conditions:
Hereditary Hemorrhagic Telangiectasia, Nosebleeds, Epistaxis
Phase:
Phase 2
Target Enrollment:
10 participants
Start Date:
Age Range:
18–999 years
Healthy Volunteers:
No

Study Locations (1 site)

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