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Immunosuppressant

Tacrolimus for Hereditary Hemorrhagic Telangiectasia

Phase 2
Waitlist Available
Led By Marie E Faughnan, MD,MSc,FRCPC
Research Sponsored by Unity Health Toronto
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age > 18 years
Clinical HHT diagnosis or personal genetic diagnosis of HHT
Must not have
Specific contra-indications for study drug (detailed in the product monograph)
Acute infection
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, weeks; 12,18,24,30,36,42,48,60,66,72,78,84,96
Awards & highlights
No Placebo-Only Group

Summary

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.

Who is the study for?
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.
What is being tested?
The 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.
What are the potential side effects?
Tacrolimus may cause side effects such as increased risk of infection due to immune system suppression, kidney problems reflected by elevated creatinine levels, potential liver dysfunction indicated by raised transaminase levels (AST/ALT), and possible heart rhythm abnormalities detectable on an ECG.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am older than 18 years.
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I have been diagnosed with HHT either through clinical evaluation or genetic testing.
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I have nosebleeds lasting 15 minutes or more each week.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have no known allergies or reactions to the study drug.
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I currently have an infection.
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I am not pregnant, breastfeeding, nor planning to become pregnant during the study.
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I am a man and my partner can have children but we are not using effective birth control.
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My liver enzymes are twice as high as the normal limit.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, weeks; 12,18,24,30,36,42,48,60,66,72,78,84,96
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, weeks; 12,18,24,30,36,42,48,60,66,72,78,84,96 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
The change in epistaxis (nose bleeding) severity using low-dose Tacrolimus
Secondary study objectives
Change in Biomarkers
Change in Chronic Bleeding
Change in Epistaxis Severity Score (ESS)
+2 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

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

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Tacrolimus, a calcineurin inhibitor, works by suppressing the immune system and reducing inflammation. It inhibits the activity of T-cells, which are crucial in the immune response, thereby decreasing the production of inflammatory cytokines. This mechanism is particularly relevant for Osler-Weber-Rendu Syndrome (HHT) patients because it can help reduce the severity of recurrent nasal hemorrhages, a common and debilitating symptom of the disease. By targeting the underlying inflammatory pathways, Tacrolimus may help in managing the vascular malformations characteristic of HHT, potentially improving the quality of life for these patients.
Effect of olfactory ensheathing cells combined with chitosan on inhibition of P2×4 receptor over-expression-mediated neuropathic pain.Characteristics of Taiwanese patients of PNH in the international PNH registry.Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols.

Find a Location

Who is running the clinical trial?

Unity Health TorontoLead Sponsor
556 Previous Clinical Trials
454,376 Total Patients Enrolled
United States Department of DefenseFED
901 Previous Clinical Trials
332,935 Total Patients Enrolled
Marie E Faughnan, MD,MSc,FRCPCPrincipal InvestigatorUnity Health Toronto

Media Library

Tacrolimus (Immunosuppressant) Clinical Trial Eligibility Overview. Trial Name: NCT04646356 — Phase 2
Osler-Weber-Rendu Syndrome Research Study Groups: Tacrolimus immediate-release capsules
Osler-Weber-Rendu Syndrome Clinical Trial 2023: Tacrolimus Highlights & Side Effects. Trial Name: NCT04646356 — Phase 2
Tacrolimus (Immunosuppressant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04646356 — Phase 2
~6 spots leftby Oct 2025