Efficacy of a Bevacizumab Nasal Spray as a Treatment for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
Summary
Hereditary Hemorrhagic Telangiectasia (HHT) is a rare (\~ 1/6000) but ubiquitous genetic disease. It is associated with abnormal angiogenesis and autosomal dominant inheritance, leading to telangiectasias and arteriovenous fistulae. More than 95% of patients are concerned by epistaxis (nosebleeds). These events are spontaneous, repeated, irregular, both diurnal and nocturnal, a source of anemia, disabling and very socially embarrassing. Anti-angiogenic treatments, including bevacizumab, are a new therapeutic option in HHT. The aim of this study is to evaluate 3 months after the end of the treatment the efficacy on the duration of the nosebleeds with 3 different doses (25, 50 and 75 mg) of bevacizumab administered as a nasal spray in a repeated manner (3 administrations) in patients with Hereditary Hemorrhagic Telangiectasia complicated by nosebleeds. This randomized, double-blind, placebo-controlled, seamless phase II/III study is to be carried out on 4 groups of 20 patients for first step and 2 groups of 20 to 40 patients for second step
Timeline
- Start
- 2014-04
- Primary completion
- 2015-06
- Completion
- 2015-09
Outcome
Missed primary endpoint
Stopped: “DSMB's decision following the first step analysis”
paper Mean monthly epistaxis duration measured at 3 months was not significantly different in the 59 patients receiving bevacizumab in comparison with the placebo group (P = .57) PMID 27599328 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Bevacizumab | Monoclonal antibody | 25 mg | Intranasal |
| Subject | Bevacizumab | Monoclonal antibody | 50 mg | Intranasal |
| Subject | Bevacizumab | Monoclonal antibody | 75 mg | Intranasal |