Tacrolimus Trial for Hereditary Hemorrhagic Telangiectasia (HHT)
Status:
Recruiting
Trial end date:
2023-09-01
Target enrollment:
Participant gender:
Summary
This study will investigate the effectiveness of oral low-dose tacrolimus for the treatment
of recurrent nasal hemorrhage in HHT subjects. The primary outcome for the trials will be the
reduction of epistaxis severity (minutes of bleeding per week). The biological outcomes of
interest are the regression of vascular malformations as well as tissue and circulation
biomarkers of the relevant mechanistic pathways. In this Phase II open label trial, we
estimate a sample size of 30 subjects with HHT, with moderate-severe recurrent epistaxis will
be required. Subject will be treated with a 6-month course of tacrolimus twice daily.
Phase:
Phase 2
Details
Lead Sponsor:
St. Michael's Hospital, Toronto Unity Health Toronto