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Clinical Trial 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.


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03850730
Study type Interventional
Source Cure HHT
Contact Nicole Schaefer
Phone 410-357-9932
Email nicole.schaefer@curehht.org
Status Not yet recruiting
Phase Phase 1/Phase 2
Start date July 1, 2023
Completion date December 31, 2025

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