Tacrolimus Trial for Hereditary Hemorrhagic Telangiectasia (HHT)
Canada10 participantsStarted 2020-10-20
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Age \> 18 years
✓. Clinical HHT diagnosis or personal genetic diagnosis of HHT
✓. Epistaxis at least 15 min per week (mean for past month)
✓. At least one telangiectasia (skin or mucosal) available for micro-imaging.
✓. Ability to give written informed consent, including compliance with the requirements of the study.
Exclusion criteria
✕. Allergy/intolerance to the study drug or related agents
✕. Unstable medical illness
✕. Acute infection
✕. Creatinine \> ULN (upper limit of normal)
✕. Liver transaminases (AST or ALT) \>= 2x ULN
✕. Women participant who are pregnant or breastfeeding or plan to become pregnant during the duration of the study
What they're measuring
1
The change in epistaxis (nose bleeding) severity using low-dose Tacrolimus
✕. Women of childbearing potential not on effective contraception. Male participants of reproductive potential whose female partners are of childbearing potential and are not planning to use highly effective contraceptive method