Active — Not RecruitingPhase 2/3

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

United States70 participantsStarted 2023-05-08

Plain-language 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.

Who can participate

Age range18 Years – 85 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Definite clinical HHT defined as having at least 3 of the following criteria:
✓. OR a definite diagnosis of HHT based on a pathogenic genetic mutation for HHT.
✓. OR probable HHT based on having 2 of the above criteria with high clinical suspicion of HHT.

What they're measuring

Change in epistaxis duration in minutes

Timeframe: Average duration in weeks 19-24 (last 6 weeks of blinded phase) versus baseline.

Hemoglobin Response rate increase in hemoglobin

Timeframe: Average duration in weeks 19-24 (last 6 weeks of blinded phase) versus baseline.

Trial details

NCT IDNCT03850964

SponsorCure HHT

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-11-21

View on ClinicalTrials.gov More Hereditary Hemorrhagic Telangiectasia trials