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 ]

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).


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