NEDenise - you can appreciate this one, a study using a nasal spray and Avastin already exists. You don't happen to have nosebleeds? Maybe you can sneak in on this one!
NORTH AMERICAN STUDY OF EPISTAXIS (NOSE Study)
Prinicipal Investigator - Dr. James Gossage, HHT Foundation Medical Director
UPDATE: We only need to recruit 24 more patients!! So far, the investigators have seen significant results. If you have mild to moderate nosebleeds on a regular basis, please contact a research coordinator at one of the participating HHT Centers listed below.
Research Plan
The largest multi-centered study of nosebleed treatment ever conducted. The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays for patients with HHT-related nosebleeds and definitively determine which of these are beneficial. Each of these sprays will approach the problem from a different mechanism of action. The U.S. Federal Drug Administration (FDA) has reviewed our application and approved the usage of these three agents.
140 patients with moderate to severe epistaxis (nosebleeds) secondary to HHT will be randomized to receive one of four intranasal sprays for a period of 12 weeks and then followed for an additional 12 weeks off therapy. Enrollment in this study will occur over a period of 12-18 months. The primary measure of efficacy will be the frequency of epistaxis. Secondary measures will include the duration of epistaxis, Hoag Epistaxis Severity Score (ESS), a quality of life survey, satisfaction of treatment, hemoglobin level, and transfusion requirements.
Nasal Sprays
- Saline spray (Placebo)
- Estriol (a low dose estrogen)
- Tranexamic Acid (a drug that promotes clotting)
- Bevacizumab (Avastin - a drug that can reduce blood vessel growth)
Avastin has both received hype about effectiveness and concerns about side effects. Nearly all of the serious complications that have been reported so far have occurred with an intravenous dose of 300-400 mg (the dose typically used to treat cancer). The dose that will be used in the NOSE Study is 4 mg per day, about 1% as potent as the intravenous dose. We expect the side effects will be minimal. A placebo has been included to allow an accurate estimate of both efficacy and safety, and to finally determine whether any of these treatments are truly beneficial.