NCT02484716: TEMPO

Efficacy of a Timolol Nasal Spray as a Treatment for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) - Randomized Trial Versus Placebo

Completed Phase 2 Last updated 5 August 2025

What this trial tests

Phase 2 trial testing Timolol nasal spray in Telangiectasia, Hereditary Hemorrhagic in 58 participants. Completed in 29 January 2018.

Timeline

1 June 2015

Primary endpoint
1 November 2017

29 January 2018

Quick facts

Lead sponsor	Hospices Civils de Lyon
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	58
Start date	1 June 2015
Primary completion	1 November 2017
Estimated completion	29 January 2018
Sites	1 location across France

Drugs / interventions tested

• Timolol nasal spray — full drug profile →
• Placebo nasal spray — full drug profile →

Conditions studied

• Telangiectasia, Hereditary Hemorrhagic — all drugs for Telangiectasia, Hereditary Hemorrhagic →
• Osler Rendu Disease — all drugs for Osler Rendu Disease →

Sponsor

Hospices Civils de Lyon — full company profile →

Who can join

18 and older, any sex, with Telangiectasia, Hereditary Hemorrhagic or Osler Rendu Disease. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Efficacy of timolol nasal spray on duration of nosebleeds for 3 months after the end of the treatment.
  Time frame: Day 0 (inclusion) ; up to 4 months
  comparison of mean monthly epistaxis duration 3 months before the treatment and 3 months after the end of the treatment.

Sponsor's own description

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.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding.
   Albiñana V, Cuesta AM, Rojas-P I, Gallardo-Vara E, et al · · 2020 · cited 20× · PMID 32517280 · DOI 10.3390/jcm9061766
2. Efficacy of TIMOLOL nasal spray as a treatment for epistaxis in hereditary hemorrhagic telangiectasia. A double-blind, randomized, placebo-controlled trial.
   Dupuis-Girod S, Pitiot V, Bergerot C, Fargeton AE, et al · · 2019 · cited 18× · PMID 31427745 · DOI 10.1038/s41598-019-48502-9
3. Epistaxis Prevention, Treatment, and Future Perspectives for Hereditary Hemorrhagic Telangiectasia.
   Ficany A, Del Alamo M, Bernabeu C, Shovlin CL, et al · · 2025 · PMID 41227119 · DOI 10.3390/jcm14217724