Clinical Trial: Efficacy of a Bevacizumab Nasal Spray as a Treatment for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: Efficacy of a Bevacizumab Nasal Spray as a Treatment for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
Brief 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
Detailed Summary:
Sponsor: Hospices Civils de Lyon
Current Primary Outcome: mean duration of epistaxis [ Time Frame: 3 months after treatment ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- adverse events [ Time Frame: before and 6 months after treatment ]Adverse events observed along a repeated administration of bevacizumab (nasal spray administration) : evaluation by epistaxis monitoring along the study and by a clinical exam before each treatment and 6 months after the end of the treatment.
- mean monthly epistaxis duration [ Time Frame: 6 months after the end of the treatment ]To evaluate the efficacy at 6 months after the end of the treatment on the duration of the nosebleeds for the dose retained versus placebo
- frequency and duration of epistaxis [ Time Frame: 3 months and 6 months after the end of the treatment ]Evolution of the frequency and the mean monthly duration of epistaxis at 3 and 6 months for the dose retained
- Quality of life [ Time Frame: 3 months and 6 months aftert the end of the treatment ]Evolution of the quality of life score (SF-36) between the inclusion, 3 months and 6 months after the end of the treatment
- Number of red blood cells transfusion [ Time Frame: 3 months and 6 months after the end of the treatment ]Evolution of the number of red blood cells transfusion between the inclusion and 3 and 6 months after the end of the treatment.
- Change in hemoglobinemia and serum ferritin [ Time Frame: 1 month, 3 months and 6 months ]Evolution of hemoglobinemia and serum ferritin at inclusion,3 and 6 months after the end of the treatment for the retained dose
- Kinetics of monthly epistaxis duration [ Time Frame: 6 months ]To describe the nosebleed kinetics for the dose retained and the placebo throughout the study
Original Secondary Outcome: Same as current
Information By: Hospices Civils de Lyon
Dates:
Date Received: April 1, 2014
Date Started: April 2014
Date Completion:
Last Updated: November 19, 2015
Last Verified: November 2015