Status:
COMPLETED
Sirolimus for Nosebleeds in HHT
Lead Sponsor:
Unity Health Toronto
Collaborating Sponsors:
National Institutes of Health (NIH)
University of California, San Francisco
Conditions:
Hereditary Hemorrhagic Telangiectasia
Nosebleeds
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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 si...
Detailed Description
The most common symptom of the hereditary hemorrhagic telangiectasia (HHT) disease is epistaxis. HHT is characterized by vascular (blood vessel) malformations, of the skin and mucus membranes of the n...
Eligibility Criteria
Inclusion
- 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
- 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
- Immunocompromised
- History of malignancy
- Known untreated dyslipidemia (20% above the ULN of total cholesterol and triglycerides)
- Specific contra-indications for study drug (detailed in the product monograph)
Key Trial Info
Start Date :
March 16 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 2 2024
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT05269849
Start Date
March 16 2022
End Date
December 2 2024
Last Update
February 27 2025
Active Locations (1)
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1
St. Michael's Hospital
Toronto, Ontario, Canada, M5B 1W8