Tacrolimus for Hereditary Hemorrhagic Telangiectasia

Recruiting in Palo Alto (17 mi)

Overseen byMarie 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

Approved in 5 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests oral low-dose tacrolimus for patients with a condition that causes severe nosebleeds. Tacrolimus helps by reducing new blood vessel growth and inflammation, which can decrease bleeding.

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 frequent nosebleeds, at least 15 minutes per week. Participants must have a clinical or genetic diagnosis of HHT and at least one visible telangiectasia. Pregnant or breastfeeding women, those not using effective contraception, people with certain allergies, acute infections, high creatinine levels, liver issues, abnormal ECGs or unstable illnesses cannot join.

Inclusion Criteria

I am older than 18 years.

I have been diagnosed with HHT either through clinical evaluation or genetic testing.

Ability to give written informed consent, including compliance with the requirements of the study

See 2 more

Exclusion Criteria

Unstable medical illness

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

I currently have an infection.

See 8 more

Treatment Details

Interventions

Tacrolimus (Immunosuppressant)

Trial OverviewThe study tests if low-dose tacrolimus capsules taken orally can reduce the severity of nosebleeds in HHT patients. It will also look for changes in vascular malformations and biomarkers related to HHT in blood and tissue. This Phase II trial involves giving subjects the drug twice daily for six months.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Tacrolimus immediate-release capsulesExperimental Treatment1 Intervention

subjects will be treated with a 6 months course of oral low-dose tacrolimus capsules to be taken twice daily starting dose of 0.025 mg/kg/day, adjusted to maintain drug blood levels of 2-5ng/ml

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