Sirolimus for Nosebleeds in HHT

• Status: Recruiting
• Trial end date: 2027-02-10
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Unity Health Toronto
• Collaborator: National Institutes of Health (NIH)
• Treatments: Sirolimus

Criteria

Inclusion Criteria:

1. Age > 18 years

2. Clinical HHT diagnosis (8) or genetic diagnosis of HHT

3. Epistaxis at least 15 min per week.

4. COVID-19 Vaccine (2 doses)

5. Ability to give written informed consent, including compliance with the requirements of the study.

Exclusion Criteria:

1. Allergy/intolerance to the study drug or related agents

2. Unstable medical illness

3. Acute infection

4. Creatinine > ULN (upper limit of normal)

5. Liver transaminases (AST or ALT) >= 2x ULN

6. Women participant who are pregnant or breastfeeding or plan to become pregnant during the duration of the study

7. Women of childbearing potential not on effective contraception.

8. Male participants of reproductive potential whose female partners are of childbearing potential and are not planning to use highly effective contraceptive method

9. Immunocompromised

10. History of malignancy

11. Known untreated dyslipidemia (20% above the ULN of total cholesterol and triglycerides)

12. Specific contra-indications for study drug (detailed in the product monograph)