NCT03850964

Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz)

A Phase II/III Randomized, Placebo Controlled, Double Blind Study to Evaluate the Effects of up to 24 Weeks of Low Dose Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 70 (estimated)
• Sponsor: Cure HHT · Academic / Other
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

During the Efficacy Study (Part B), the investigators will study whether Pazopanib, taken daily for 24 weeks, will reduce the severity of nose bleeds in patients with hereditary hemorrhagic telangiectasia (HHT). Patients will either be provided active drug or a placebo \[sugar - inactive pill\], and be tested for nose bleed severity throughout the trial, including particularly nose bleed duration. Investigators will also test for blood loss, as well as for safety. This study is funded by the US Department of Defense USAMRAA and FDA/OOPD.

Detailed description

Now that a single dose pharmacokinetics (PK) study (Part A) has been completed to properly establish similar exposure with the prior pilot 50mg tablet, a double blind, placebo controlled study will follow (Part B), which proposes to define primarily the value of low dose (150 mg) Pazopanib on nose bleed duration, in the context of assessing perceived nose bleed severity. After a patient completes Part B of the study, the patient will be invited to take part in an Extension Study (Part C) in which the patient will be provided with active drug equal to the dose they were assigned in Part B. All patients in Part C will receive active drug for 24 weeks. Part C will further assess the effects of Pazopanib on the severity of nose bleeds in patients with HHT and also support safety and efficacy elements. After the patient completes their treatment period (either Part B or Parts B and C), a 12 week follow-up period will follow to support safety and efficacy elements. Secondary endpoints will be assessed, including ongoing blood loss, use of iron and blood products, quality of life, and drug safety.

Conditions

• Hereditary Hemorrhagic Telangiectasia
• Epistaxis
• Anemia
• Nosebleed
• HHT

Interventions

Type	Name	Description
DRUG	Pazopanib	gel capsule, with 25mg-similar fills
DRUG	Placebo oral capsule	identical gel capsule without active pharmaceutical ingredient

Timeline

• Start date: 2023-05-08
• Primary completion: 2025-11-21
• Completion: 2026-07-01
• First posted: 2019-02-22
• Last updated: 2026-03-19

Locations

11 sites across 1 country: United States

Regulatory

• FDA-regulated drug study