Timolol is a nonselective β-blocker commonly used in the treatment of glaucoma. Recently it has been used topically for the treatment of superficial hemangiomas. Because of its potential mechanism of action, it is possible that timolol could also be useful for the treatment of epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT). Moreover a case was reported in 2012 showing an improvement of nosebleeds with the use of topical nasal timolol. The aim of the study is to evaluate timolol nasal spray efficacy in HHT. The main objective of this trial is to evaluate, 3 months after the end of the treatment, the efficacy on the duration of nosebleeds of a 4 weeks timolol intranasal treatment in HHT patients with nosebleeds (\>20 min/month). Secondary objectives are to evaluate the tolerance, the efficacy at 6 months after the end of the treatment, and the efficacy on anemia and on clinical parameters (nosebleeds, quality of life and blood transfusions). This is a prospective double blind phase II study, randomized versus placebo using an allocation ratio of 1:1. A total of 58 patients will be included. The product (solution with timolol at 0.5% or placebo) is self-administered by the patient with a posology of one spray (50 µL) in each nostril twice a day for 28 consecutive days.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
58
Timolol 0.5% is administered by the patient with a posology of one spray (50 µL) in each nostril twice a day for 4 weeks.
Placebo (NaCl) is administered by the patient with a posology of one spray (50 µL) in each nostril twice a day for 4 weeks.
Hospices Civils de Lyon - Hôpital Femme Mère Enfant / Service de génétique Clinique
Bron, France
Efficacy of timolol nasal spray on duration of nosebleeds for 3 months after the end of the treatment.
comparison of mean monthly epistaxis duration 3 months before the treatment and 3 months after the end of the treatment.
Time frame: Day 0 (inclusion) ; up to 4 months
Tolerance of timolol nasal spray in patients with HHT-related epistaxis
Tolerance will be evaluated by observing adverse effects and clinical examinations during the follow up period.
Time frame: up to 7 months
Efficacy on clinical criteria : epistaxis frequency .
Comparison of number of epistaxis before and after treatment.
Time frame: Day 0 (inclusion) ; up to 4 months
Efficacy on clinical criteria : biological parameters (hemoglobin and ferritin level).
Comparison of hemoglobin and ferritin level before and after treatment.
Time frame: Day 0 (inclusion) ; up to 4 months
Efficacy on clinical criteria : quality of life (SF36).
Comparison of SF36 questionnaire before and after treatment.
Time frame: Day 0 (inclusion) ; up to 4 months
Efficacy of timolol nasal spray on duration of nosebleeds for 6 months after the end of the treatment.
Comparison of mean monthly epistaxis duration 3 months before the treatment and 6 months after the end of the treatment.
Time frame: Day 0 (inclusion) ; up to 7 months
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