Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia (ETIC-HHT)
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|ClinicalTrials.gov Identifier: NCT04139018|
Recruitment Status : Completed
First Posted : October 25, 2019
Results First Posted : August 24, 2021
Last Update Posted : August 24, 2021
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Hemorrhagic Telangiectasia||Drug: Timolol Gel Drug: Placebo Gel||Phase 2|
This study is a double-blinded, placebo-controlled, 8-week randomized clinical trial investigating the efficacy of timolol gel in the management of epistaxis in adults with HHT.
The Specific Aims are to determine in adults with HHT-associated epistaxis:
- If topical timolol gel is more effective than placebo in reducing the frequency and severity of epistaxis.
- If topical timolol gel is more effective than placebo in improving hemoglobin levels.
- The frequency of adverse events, side effects, and safety profile of topical timolol gel delivered to the nasal mucosa.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||27 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Efficacy of a Timolol Gel in the Care for Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia: A Double-Blinded, Randomized Controlled Trial|
|Actual Study Start Date :||October 20, 2019|
|Actual Primary Completion Date :||May 20, 2020|
|Actual Study Completion Date :||May 20, 2020|
Experimental: Timolol Gel Arm
Participants in the timolol gel arm (active medication arm) will receive timolol nasal gel 0.1% with 0.5 mL applied to each nostril twice daily via a syringe that will amount to a 2 mg total daily dose.
Drug: Timolol Gel
Timolol nasal gel 0.1% will be prepared with a poloxamer gel (combination of poloxamer 188 and 407; pH adjusted to 4.5-6.5) and 0.5 ml applied to each nostril twice daily. The total daily dose would amount to 2 mg.
Placebo Comparator: Placebo Gel Arm
Participants in the placebo gel arm will receive the gel itself with no active medication.
Drug: Placebo Gel
Placebo gel is prepared with poloxamers and no active ingredients.
- Change in Assisted Epistaxis Severity Scale (aESS) Score From Baseline at 8 Week Follow-up [ Time Frame: Baseline to 8-week follow-up ]
Assessment of epistaxis severity will be obtained by the validated instrument, the Epistaxis Severity Score (ESS). To complete the ESS, patients are asked to consider typical symptoms over the previous 3 months. The ESS contains 6 items - frequency, duration, and intensity of nosebleeds, whether patient has sought medication attention, whether patient is anemic, and whether patient has received a blood transfusion. The overall score ranges from 0 to 10, with severity of nosebleed based on score graded as None composite score of 0-1, Mild 1-4, Moderate 4-7, and Severe as 7-10.The minimal important difference noticeable by both patients and clinicians in the ESS scoring system is estimated as a change of 0.71. The scoring and MCID of the aESS is the same as the ESS.
The aESS references a participant's epistaxis over the past 1 month, and the change in aESS was calculated as the aESS score at 8 weeks minus the aESS score at baseline.
- Number of Participants With Improved Response on Clinical Global Impression - Improvement (CGI-I) Scale [ Time Frame: Scores at 8-week follow-up only ]CGI-I is a global rating of improvement scale, which requires subjects to rate their degree of improvement on a seven-point scale: "Compared to your condition at admission to the project [prior to medication initiation], how would you rate your overall response: 1=very much improved since the initiation of treatment; 2=much improved; 3=minimally improved; 4=no change from baseline (the initiation of treatment); 5=minimally worse; 6= much worse; 7=very much worse since the initiation of treatment."
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04139018
|United States, Missouri|
|Saint Louis, Missouri, United States, 63110|
|Principal Investigator:||Jay F Piccirillo, MD||Washington University School of Medicine|