Investigating the effects of pazopanib on nosebleeds and anemia in patients with hereditary hemorrhagic telangiectasia.
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
This study is looking at whether a low dose of pazopanib, a drug usually used for cancer, can help people with hereditary hemorrhagic telangiectasia (HHT) by reducing nosebleeds and improving anemia, and it’s designed for those who experience these symptoms.
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-10704999 on NIH RePORTER |
What this research studies
This research focuses on evaluating the effects of low-dose pazopanib, a drug originally developed for cancer treatment, on patients suffering from hereditary hemorrhagic telangiectasia (HHT), a rare disorder that causes frequent and severe nosebleeds and anemia. The study will involve a randomized, placebo-controlled, double-blind design, meaning that neither the participants nor the researchers will know who is receiving the actual medication versus a placebo. Over a period of up to 24 weeks, participants will be monitored for improvements in their symptoms, particularly the frequency of nosebleeds and levels of anemia. The goal is to determine if pazopanib can provide significant relief and improve the quality of life for those affected by HHT.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hereditary hemorrhagic telangiectasia who experience frequent nosebleeds and anemia.
Not a fit: Patients who do not have hereditary hemorrhagic telangiectasia or those who do not experience significant nosebleeds or anemia may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new treatment option that significantly reduces the frequency of nosebleeds and alleviates anemia for patients with HHT.
How similar studies have performed: Previous studies have shown promising results with similar approaches, including preclinical models and small pilot studies indicating potential benefits of pazopanib for HHT patients.
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.