10 Participants Needed

Sirolimus for Nosebleeds in HHT

ME
NB
Overseen ByNegar Bagheri, BSc MSc
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Unity Health Toronto
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 4 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

ME

Marie E Faughnan, MD MSc FRCPC

Principal Investigator

Unity Health Toronto

Are You a Good Fit for This Trial?

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.
See 2 more

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.
See 9 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral sirolimus starting at 2 mg once daily, adjusted to maintain blood levels of 6-10 ng/ml for 3 months

12 weeks
Weekly visits for dose adjustment and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

9 months
Monthly visits for outcome measures and safety monitoring

What Are the Treatments Tested in This Trial?

Interventions

  • Sirolimus
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.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Oral sirolimus tabletsExperimental Treatment1 Intervention

Sirolimus is already approved in United States, European Union, Canada, Japan for the following indications:

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Approved in United States as Rapamune for:
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Approved in European Union as Rapamune for:
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Approved in Canada as Rapamune for:
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Approved in Japan as Rapamune for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Unity Health Toronto

Lead Sponsor

Trials
572
Recruited
470,000+

University of California, San Francisco

Collaborator

Trials
2,636
Recruited
19,080,000+

National Institutes of Health (NIH)

Collaborator

Trials
2,896
Recruited
8,053,000+
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