Can a transplant drug stop HHT nosebleeds? small study tests sirolimus
NCT ID NCT05269849
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This small pilot study tested the safety and effectiveness of the oral drug sirolimus for reducing nosebleeds in people with hereditary hemorrhagic telangiectasia (HHT). Ten adults with moderate to severe nosebleeds took 2 mg of sirolimus daily for 3 months. Researchers tracked bleeding duration, side effects, and lab values to see if the drug could help control this common HHT symptom.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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10 people
The number who actually took part.
- Started
-
Mar 2022
- Finished
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Dec 2024
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
St. Michael's Hospital
Toronto, Ontario, M5B 1W8, Canada
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- A pill to stop the bleeding? new hope for HHT nosebleeds
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- Blood thinners in HHT: a risky balancing act under the microscope
- New ointment could tame HHT nosebleeds
- New antibody aims to tame rare bleeding disorder