🏛️ Verified NIH Record • Updated 2026-03-17

Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz) (NCT03850964)

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

🔵 Active, Not Recruiting Phase2 / Phase3 💊 Investigational Drug / Biologic Sponsor: Cure HHT

🩺 Plain-English Summary (8th-Grade Level)

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 join: To join this study, participants generally need to meet the listed inclusion criteria (18 items) and avoid the listed exclusions (29 items).

Location: University of California - Los Angeles — Los Angeles, California

Age range: 18 Years to 85 Years

🧪 Interventions in This Study

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Usually NOT eligible if any of the following apply:

  • Exclusion Criteria:
  • 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.
  • Currently has incompletely treated cerebral arterio-venous malformations (AVMs) or cerebral arteriovenous fistulas (AVFs) that are symptomatic or have high-risk features detected on either MRI/MRA or digital subtraction angiography. High-risk features include: microhemorrhage seen on MRI; feeding artery aneurysm, nidus aneurysm or venous outflow stenosis seen on MRA, CTA, or catheter angiography. Non-shunting vascular brain lesions such as capillary vascular malformations, telangiectasias, and cavernous malformations are not an exclusion criterion. (Note: MRI scan does not need to be repeated at screening if AVMs and AVFs were absent on a scan at age ≥18 years).
  • Currently has perfused pulmonary AVMs with feeding artery diameter ≥ 3mm.
  • Known significant bleeding sources other than nasal or gastrointestinal.
  • Systemic use of a potent VEGF inhibitor (e.g., direct inhibitors of VEGF-receptor signaling such as sunitinib) in the 4 weeks prior to enrollment. Systemic use of bevacizumab in the 6 weeks prior to enrollment due to its longer half-life.
  • Active and recent onset of clinically significant diarrhea.
  • Current or recent (in the last 5 years) malignancies (except non-melanoma skin cancers)
  • 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
  • Participant has a planned surgery during periods of active treatment and 6 weeks of follow up; case by case evaluation if PI desires inclusion with medical monitor agreement.
  • Participant has clinically significant gastrointestinal abnormalities (other than hereditary hemorrhagic telangiectasia related vascular lesions).
  • Participant during the 6 months prior to first dose of study drug has a history of cerebrovascular accident (including transient ischemic attacks), pulmonary embolism, untreated deep vein thrombosis (DVT), myocardial infarction, or any other thrombotic event.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (11 Sites)

  • University of California - Los Angeles — Los Angeles, California
  • University of Colorado — Denver, Colorado
  • Augusta University — Augusta, Georgia
  • John Hopkins University — Baltimore, Maryland
  • Massachusetts General Hospital — Boston, Massachusetts
  • Mayo Clinic — Rochester, Minnesota
  • Washington University — St Louis, Missouri
  • University of North Carolina — Chapel Hill, North Carolina
  • Cleveland Clinic — Cleveland, Ohio
  • University of Texas - Southwestern — Dallas, Texas
  • University of Utah Medical Center — Salt Lake City, Utah

💰 Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Hereditary Hemorrhagic Telangiectasia

Explore by condition: All Hereditary Hemorrhagic Telangiectasia clinical trials

💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗