Low-dose pazopanib for HHT nosebleeds and anemia
A Phase II/III randomized, placebo controlled, double blind study to evaluate the effects of up to 24 weeks of low dose pazopanib on HHT related epistaxis and anemia. IND#144808 June 25, 2020
Low-dose pazopanib given for up to 24 weeks aims to reduce nosebleeds and anemia in people with hereditary hemorrhagic telangiectasia (HHT).
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Hht Foundation International, INC. NIH-funded |
| Lab location | 1 site (Monkton, United States) |
| Project ID | NIH-11162362 on NIH RePORTER |
What this research studies
This randomized, placebo-controlled, double-blind Phase II/III trial gives people with HHT a chance to receive low-dose pazopanib or a placebo for up to 24 weeks without knowing which one they get. You would have regular clinic visits to record nosebleed frequency, complete blood counts, and any need for IV iron or blood transfusions. The drug is an anti-angiogenic cancer medication that reduces VEGF signaling and is being repurposed because small pilot studies and animal work suggested benefit. The HHT Foundation is sponsoring the effort under an IND to see if this treatment can safely lower bleeding and anemia in a larger group.
Who could benefit from this research
Good fit: People with a confirmed diagnosis of HHT who have frequent or severe nosebleeds and anemia, including those needing transfusions or IV iron, are the ideal candidates.
Not a fit: People without HHT, those whose bleeding comes from other causes, or patients with medical contraindications to pazopanib (for example pregnancy, significant liver or heart disease) are unlikely to benefit.
Why it matters
Potential benefit: If successful, this treatment could meaningfully reduce nosebleed frequency, lower transfusion or IV iron needs, and improve quality of life for people with HHT.
How similar studies have performed: Early pilot clinical cases, off-label use reports, and a mouse HHT model showed encouraging signals, but large randomized trials have been limited until now.
Where this research is happening
Monkton, United States
- Hht Foundation International, INC. — Monkton, United States (Active)
Researchers
- Principal investigator: Gossage, James Richard — Hht Foundation International, INC.
- Study coordinator: Gossage, James Richard
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.