NCT03850730

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)

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Cure HHT · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hereditary Hemorrhagic Telangiectasia
• Epistaxis

Interventions

Type	Name	Description
DRUG	Pazopanib	Active pharmaceutical ingredient plus excipients filled into a capsule for the lower dosing necessary in this study

Timeline

• Start date: 2023-07-01
• Primary completion: 2025-06-30
• Completion: 2025-12-31
• First posted: 2019-02-22
• Last updated: 2022-09-08

Regulatory

• FDA-regulated drug study