Sirolimus for Nosebleeds in HHT

Recruiting in Palo Alto (17 mi)

Overseen by Marie E Faughnan, MD MSc FRCPC

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Unity Health Toronto

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Research Team

Marie E Faughnan, MD MSc FRCPC

Principal Investigator

Unity Health Toronto

Eligibility Criteria

This trial is for adults over 18 with Hereditary Hemorrhagic Telangiectasia (HHT) who suffer from moderate to severe nosebleeds lasting at least 15 minutes per week. Participants must have received two COVID-19 vaccine doses and not be pregnant, breastfeeding, or planning pregnancy without effective contraception. They should not have a history of cancer, acute infections, unstable illnesses, high creatinine levels, liver issues twice above normal limits or untreated dyslipidemia.

Inclusion Criteria

I experience nosebleeds lasting over 15 minutes each week.

I have received 2 doses of a COVID-19 vaccine.

I am older than 18 years.

Exclusion Criteria

I have no known allergies or reactions to the study drug.

I am a man and my partner can have children but we are not using effective birth control.

My immune system is weak.

Treatment Details

Interventions

• Sirolimus (mTOR inhibitor)

Trial Overview The study tests the safety and effectiveness of oral sirolimus in reducing nosebleeds in HHT patients. Participants will take a daily dose of sirolimus to maintain specific blood levels for three months. The impact on nosebleeds will be measured against their situation before the trial using patient-reported outcomes.

Participant Groups

Group I: Oral sirolimus tablets Experimental Treatment 1 Intervention

Sirolimus starting dose of 2 mg once daily, orally adjusted as need to maintain drug blood levels of 6-10 ng/ ml The first dose will be given at the week 12 visit and participants will be observed for 30 min

Sirolimus is already approved in Canada, Japan for the following indications:

Approved in Canada as Rapamune for:

• Prevention of organ rejection in kidney transplant patients
• Treatment of lymphangioleiomyomatosis (LAM)

Approved in Japan as Rapamune for:

• Prevention of organ rejection in kidney transplant patients

Who Is Running the Clinical Trial?

Unity Health Toronto

Lead Sponsor

Trials

572

Recruited

470,000+

Dr. Sharon Straus

Unity Health Toronto

Chief Medical Officer

MD and MSc in Clinical Epidemiology, University of Toronto

Altaf Stationwala

Unity Health Toronto

Chief Executive Officer

Bachelor's degree in Health Administration, University of Ottawa

University of California, San Francisco

Collaborator

Trials

2,636

Recruited

19,080,000+

Suresh Gunasekaran

University of California, San Francisco

Chief Executive Officer since 2022

MBA from Southern Methodist University

Dr. Lukejohn Day

University of California, San Francisco

Chief Medical Officer

MD from Stanford University School of Medicine

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

Dr. Jeanne Marrazzo

National Institutes of Health (NIH)

Chief Medical Officer

MD from University of California, Los Angeles

Dr. Jay Bhattacharya

National Institutes of Health (NIH)

Chief Executive Officer

MD, PhD from Stanford University