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Phase 2 N=27 Randomized Quadruple-blind Treatment

Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia

Hereditary Hemorrhagic Telangiectasia

Enrolled (actual)
27
Serious AEs
0.0%
Results posted
Aug 2021
Primary outcome: Primary: Change in Assisted Epistaxis Severity Scale (aESS) Score From Baseline at 8 Week Follow-up — 2.32; 1.96 units on a scale

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Timolol Gel (Drug); Placebo Gel (Drug)
Age
Adult, Older Adult · 20+ yrs
Sex
All
Sponsor
Washington University School of Medicine
Primary completion
May 2020

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in Assisted Epistaxis Severity Scale (aESS) Score From Baseline at 8 Week Follow-up
2.32; 1.96
SECONDARY
Number of Participants With Improved Response on Clinical Global Impression - Improvement (CGI-I) Scale
0; 1; 4; 3; 7; 8

Summary

This study is a double-blinded, randomized controlled trial to evaluate the efficacy of an intranasal topical timolol gel in the care for epistaxis in adults with hereditary hemorrhagic telangiectasia.

Eligibility Criteria

Inclusion Criteria

  • Adults ages 20 and older
  • Confirmed clinical (meeting at least 3 of the 4 Curaçao Criteria) or genetic diagnosis of HHT
  • Epistaxis Severity Score (ESS) ≥ 4 and 2 or more nosebleeds per week with a cumulative nosebleed duration of at least 5 minutes per week
  • Stable nasal hygiene and medical regimen for preceding 1 month
  • Stable epistaxis pattern over the preceding 3 months

Exclusion Criteria

  • Contraindications for systemic β adrenergic blocker administration
  • Hypersensitivity to β adrenergic blockers
  • Asthma or bronchospasm
  • Congestive heart failure with LVEF 9%) or diabetic ketoacidosis within last 6 months
  • Hypotension (systolic blood pressure < 90)
  • Known hypersensitivity to timolol
  • Severe peripheral circulatory disturbances (Raynaud phenomenon)
  • Known intermediate or poor metabolizer variant of the liver enzyme CYP2D6
  • Current use of any of the following known strong CYP2D6 inhibitors: fluoxetine (Prozac), paroxetine (Paxil), bupropion (Welbutrin), quinidine, quinine, ritonavir (Norvir), and terbinafine (Lamisil)
  • Current use of the following other drugs known to pharmacodynamically interact with timolol: diltiazem, verapamil, digoxin, digitalis, propafenone, disopyramide, clonidine, flecainide, or lidocaine
  • Patients currently treated or who plan to initiate treatment with β-blockers
  • Use of any anti-angiogenic medication in the last month prior to recruitment, including bevacizumab, pazopanib, thalidomide, or lenalidomide
  • Illicit drug use, except marijuana
  • Known pheochromocytoma
  • Use of anticoagulants, antiplatelet, or fibrinolytic therapies within the last month prior to recruitment, except for low-dose (81 mg or less) of aspirin
  • Pregnancy or planned pregnancy in the next 6 months or currently breastfeeding
  • Inability to read or understand English
  • Inability to complete 8 weeks of therapy for any reason
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04139018). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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