Conditions: Telangiectasia, Hereditary Hemorrhagic Epistaxis
Keywords: epistaxis HHT bevacizumab tranexamic acid nosebleed estrogen
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 123
Healthy volunteers: no

Brief summary:
The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays (bevacizumab, estriol, and tranexamic acid), compared to placebo, for the treatment of HHT related nosebleeds.

Detailed description:
140 patients with moderate to severe epistaxis 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 will occur over a period of 18-36 months. The primary endpoint will be the frequency of epistaxis. Secondary endpoints will include duration of epistaxis, the Hoag Epistaxis Severity Score (ESS), a quality of life survey, satisfaction with treatment, hemoglobin and ferritin levels, transfusion requirements, and treatment failure. The sprays will be: saline spray (Placebo); estriol 0.1% in methylcellulose suspension (EST); tranexamic acid 10% in saline (TA), and bevacizumab 1% in saline (BEV). All sprays will be applied to the nasal mucosa by an identical spray bottle at a dose of 0.1 ml per nostril twice daily (total dose of 0.4 ml daily). Thus, the delivered doses will be: EST, 0.4 mg/day; TA, 40 mg/day; BEV, 4 mg/day.

Arms:
arm 1: sterile saline arm 2: bevacizumab 1% arm 3: Estriol 0.1% arm 4: tranexamic acid 10%

Interventions: intervention 1: Sterile saline intervention 2: Bevacizumab intervention 3: Estriol intervention 4: Tranexamic Acid
Intervention descriptions: intervention 1: 0.9%, 0.1 ml spray in each nostril bid intervention 2: 1% solution in saline, 0.1 ml spray in each nostril bid intervention 3: 0.1% suspension in methylcellulose, 0.1 ml spray in each nostril bid intervention 4: 10% solution in saline, 0.1 ml spray in each nostril bid

Protocol document: