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Sirolimus for Nosebleeds in HHT

NCT05269849 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-02-27

No results posted yet for this study

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.

Conditions

  • Hereditary Hemorrhagic Telangiectasia
  • Nosebleeds
  • Epistaxis

Interventions

DRUG

Sirolimus

Sirolimus (1, 2, or 5 mg tablets) given for 3 months followed by a washout period of 3 months

Sponsors & Collaborators

Principal Investigators

  • Marie E Faughnan, MD MSc FRCPC · Unity Health Toronto

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-16
Primary Completion
2024-12-02
Completion
2024-12-02

Countries

  • Canada

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05269849 on ClinicalTrials.gov