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 range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Electrolytes

Timeframe: 9 months

Hemoglobin

Timeframe: 9 months

Renal Function

Timeframe: 9 months

Liver Function

Timeframe: 9 months

Change in Ferritin Levels

Timeframe: 9 months

Blood Glucose Level

Timeframe: 9 months

Lipid Assessment

Timeframe: 9 months

Total Number of Adverse Events (AEs)

Timeframe: 3 months

Trial details

NCT IDNCT05269849

SponsorUnity Health Toronto

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-12-02

Results submitted2025-06-25

View on ClinicalTrials.gov More Hereditary Hemorrhagic Telangiectasia trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Electrolytes

Timeframe: 9 months

Hemoglobin

Timeframe: 9 months

Renal Function

Timeframe: 9 months

Liver Function

Timeframe: 9 months

Change in Ferritin Levels

Timeframe: 9 months

Blood Glucose Level

Timeframe: 9 months

Lipid Assessment

Timeframe: 9 months

Total Number of Adverse Events (AEs)

Timeframe: 3 months