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Pazopanib for the Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia | Clinical Trials | Clareo Health

UNKNOWNPHASE1/PHASE2

Pazopanib for the Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia

An Open-label, Non-randomized Study of the Efficacy of Pazopanib for the Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)

NCT03850730•Cure HHT

View on ClinicalTrials.gov

Summary

Investigators will test the value of very low dose Pazopanib administered to patients with hereditary hemorrhagic telangiectasia for the reduction in the severity of nose bleeds in those with frequent and long duration bleeding episodes.

Detailed Description

Based on frequency and nose bleed duration, a non-randomized, single arm, open label study of 30 hereditary hemorrhagic telangiectasia patients will be treated with very low dose Pazopanib \[25mg-similar\] for between 16 and 24 weeks.. The primary endpoint is a reduction in bleeding duration of 50% or more, along with multiple secondary related endpoints, including bleed frequency, blood counts and quality of life; as compared to 6-12 weeks of baseline characteristics. If after the first 8 weeks of therapy benefit is suboptimal, dose advance to 50mg-similar daily can be considered.

Eligibility

Age

18 - 75 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•a definite diagnosis of hereditary hemorrhagic telangiectasia defined as having at least 3 of the following criteria:
•* Spontaneous and recurrent epistaxis.
•* Multiple telangiectasias at characteristic sites: lips, oral cavity, fingers, nose.
•* Visceral lesions: GI telangiectasia, pulmonary, hepatic, cerebral or spinal AVMs.
•* A first degree relative with hereditary hemorrhagic telangiectasia according to these criteria.
•* OR a gene sequencing diagnosis of hereditary hemorrhagic telangiectasia
•2\. Epistaxis due to hereditary hemorrhagic telangiectasia at least 2x per week, for a cumulative duration of at least 25 minutes per week
•3\. Epistaxis is clinically stable during the 12 weeks prior to screening in the clinical judgment of the investigator (i.e. no major changes in frequency or duration of epistaxis).
•4\. Participant agrees not to undergo cautery of nasal telangiectasias or take any experimental therapies for hereditary hemorrhagic telangiectasia other than the study drug while participating in the study.
•5\. Male or female \[non-child bearing potential\]

Exclusion Criteria

•1. Participant has known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to pazopanib that in the opinion of the investigator contradicts their participation.
•2. Currently has untreated cerebral arterio-venous malformations (AVMs), cerebral arteriovenous fistulae, or cerebral cavernous malformations (CCMs) (Note: MRI scan does not need to be repeated at screening if AVMs, arterio-venous fistulas and CCMs were absent on a scan at age ≥18 years).
•3. Currently has perfused pulmonary AVMs with feeding artery diameter \>3mm.
•4. Known significant bleeding sources other than nasal or gastrointestinal.
•5. Systemic use of a vascular endothelial growth factor inhibitor in the past 3 months or previous enrollment in this study.
•6. Active and recent onset of clinically significant diarrhea.
•7. Current or recent (in the last 5 years) malignancies (except non-melanoma skin cancers)
•8. Participant has had major surgery (e.g. surgical ligation of an AVM) or trauma within 28 days or had minor surgical procedures (e.g. central venous access line removal) within 7 days prior to dosing, the latter representing a recent wound, fracture or ulcer
•9. Participant has a planned surgery during the period to include active treatment and 6 weeks of follow up.
•10. Participant has clinically significant gastrointestinal abnormalities (other than hereditary hemorrhagic telangiectasia related vascular lesions)
+13 more criteria

Key Dates

Start Date

July 1, 2023

Primary Completion Date

June 30, 2025

Completion Date

December 31, 2025

Last Updated

September 8, 2022

Enrollment

ESTIMATED participants

Conditions

Hereditary Hemorrhagic TelangiectasiaEpistaxis

Interventions

Pazopanib

DRUG

Contacts

Nicole Schaefer

CONTACT

410-357-9932 nicole.schaefer@curehht.org

Dennis L Sprecher, MD

CONTACT

410-357-9932 dennis.sprecher@curehht.org

Treatment of Nosebleeds in the Emergency Department With Powder Tranexamic Acid

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 8, 2022Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Pazopanib for the Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia

Inclusion Criteria

Exclusion Criteria

Treatment of Nosebleeds in the Emergency Department With Powder Tranexamic Acid

Efficacy and Tolerance of Treatment With Bevacizumab for Severe Liver Involvement With High Cardiac Output in Hereditary Hemorrhagic Telangiectasia Within the French Hereditary Hemorrhagic Telangiectasia Network

Ultra-low-dose Chest CT for HHT

Doxycycline for Hereditary Hemorrhagic Telangiectasia

Genotype-phenotype Association in Hereditary Hemorrhagic Telangiectasia

Risk Factors of Epistaxis Recurrence in Adults