Sirolimus for Nosebleeds in HHT (NCT05269849) | Clinical Trial Compass
CompletedPhase 2
Sirolimus for Nosebleeds in HHT
Canada10 participantsStarted 2022-03-16
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Age \> 18 years
✓. Clinical HHT diagnosis (8) or genetic diagnosis of HHT
✓. Epistaxis at least 15 min per week.
✓. COVID-19 Vaccine (2 doses)
✓. 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
. 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