Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia (ETIC-HHT)
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|ClinicalTrials.gov Identifier: NCT04139018|
Recruitment Status : Recruiting
First Posted : October 25, 2019
Last Update Posted : December 6, 2019
|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)|
|Estimated Enrollment :||30 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|
|Estimated Primary Completion Date :||October 20, 2020|
|Estimated Study Completion Date :||October 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.
- Epistaxis Severity Scale [ Time Frame: Baseline to 8-week follow-up ]Assessment of epistaxis severity will be obtained by the validated instrument, the Epistaxis Severity Score (ESS).(Hoag et al. 2010) 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.(Yin et al. 2016) With the 8-week duration of our trial, patients will complete the Assisted Epistaxis Severity Scale (aESS), which references their epistaxis over the past 1 month.
- Clinical Global Impression - Improvement (CGI-I) Score [ Time Frame: Baseline to 8-week follow-up ]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." (Busner J, Targum SD. 2007).
- Mean Change in Hemoglobin [ Time Frame: Baseline to 8-week follow-up ]
Hemoglobin is a protein in red blood cells which contains iron.Hemoglobin is involved in the transport of oxygen and carbon dioxide. Red blood cells get their color from hemoglobin. Hemoglobin test measures serum hemoglobin level and levels lower than normal means a low red blood cell count or anemia. Anemia can have many different causes, including vitamin deficiencies, bleeding and chronic diseases. Hemoglobin serum values are often measured in patients with HHT to assess for anemia.
The normal range for hemoglobin is:
Men, 13.5 to 17.5 grams per deciliter Women, 12.0 to 15.5 grams per deciliter
Change in hemoglobin values are often used to measure the impact of epistaxis treatment.
- HHT endoscopic scale (HES) [ Time Frame: Baseline to 8-week follow-up ]The HHT Endoscopy Score (HES) is an endoscopy staging score developed as a way to quantify the severity of the patient's nasal telangiectasias as seen on physical exam.(Reh et al. 2014) The HES was shown to correlate with the ESS. Its parameters are telangiectasia sites, patterns, crusting, location, perforation, density, and relative AVMs.
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
|Contact: Andrew M Petersonemail@example.com|
|Contact: Sara Kukuljan, RNfirstname.lastname@example.org|
|Principal Investigator:||Jay F Piccirillo, MD||Washington University School of Medicine|