North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01408030
• Date of registration: 01/08/2011
• Prospective Registration: Yes
• Primary sponsor: James Gossage
• Public title: North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) NOSE
• Scientific title: North American Study of Epistaxis in HHT (NOSE)
• Date of first enrolment: August 2011
• Target sample size: 123
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01408030
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: James R Gossage, MD
• Affiliation: Augusta University

Key inclusion & exclusion criteria

Inclusion Criteria:

1. A diagnosis of definite or possible HHT by the Curacao criteria (Shovlin 2000) or a
positive DNA test for HHT (as characterized by a disease causing mutation in the gene
coding for endoglin, activin like kinase 1, or SMAD-4). According to the Curacao
criteria, a definite diagnosis of HHT is defined as having at least 3 of the following
criteria while a possible diagnosis is defined as 2 criteria:

1. Spontaneous and recurrent epistaxis.

2. Multiple telangiectasias at characteristic sites (lips, oral cavity, fingers,
nose).

3. Visceral lesions such as gastrointestinal telangiectasias and arteriovenous
malformations (AVM) in lung, brain, spine and liver.

4. A history of definite HHT in a first degree relative using these same criteria.

2. Epistaxis of at least 1 minute (on average) and which occurs at least once weekly when
averaged during the preceding 8 weeks.

3. Epistaxis severity score (ESS) of at least 3.0.

4. Age of at least 18 years.

5. Written and informed consent obtained prior to study entry.

6. Subject is able and willing to return for outpatient visits.

7. The epistaxis is considered to be clinically stable during the past 8 weeks in the
clinical judgment of the investigator (i.e. no major changes in frequency or duration
of epistaxis or in transfusion requirements).

8. Negative pregnancy test at enrollment.

Exclusion Criteria:

1. Allergy to any of the active treatment agents or their spray additives.

2. Estimated life expectancy less than 1 year.

3. A psychiatric or substance abuse problem that is expected to interfere with study
compliance.

4. History of deep venous thrombosis (DVT), pulmonary embolism (PE), acute myocardial
infarction (MI), arterial thromboembolism, or ischemic stroke in the past 6 months.6.
History of receiving more than 12 units of red blood cells in the past 12 weeks.

7. Presence of an untreated coagulopathy that is felt to be contributing to the 5. History
of estrogen receptor positive breast cancer. epistaxis. 8. Presence of active disseminated
intravascular coagulation. 9. Uncontrolled hypertension (systolic BP >160 and/or diastolic
BP >100). 10. Presence of untreated brain AVM. 11. Presence of active malignancy in the
brain, lung, or colon. 12. Presence of symptomatic heart failure. 13. Use of estrogens,
epsilon aminocaproic acid, tranexamic acid, or thalidomide by any route for more than 1
week in the past 12 weeks. Any use of a VEGF inhibitor by any route in the past 24 weeks.

14. Baseline use of the following anticoagulants is not allowed: warfarin or other vitamin
K antagonists at any dose; unfractionated or low molecular weight heparins at standard
doses for treatment of venous thromboembolism (VTE); or aspirin at >325 mg/day. Baseline
use of the following anticoagulants is allowed: heparins at standard doses for VTE
prophylaxis; clopidogrel; or aspirin at =325 mg/day.

15. Addition of new treatments for epistaxis in the past 12 weeks (including laser ablation
of nasal telangiectasias and over the counter medications).

16. Presence of another overt cause (e.g. overt gastrointestinal bleeding) that is felt to
be significantly contributing to anemia.

17. Lactating women.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Telangiectasia, Hereditary Hemorrhagic

Epistaxis

Intervention(s)

Drug: Sterile saline

Drug: Bevacizumab

Drug: Tranexamic Acid

Drug: Estriol

Primary Outcome(s)

Frequency of Epistaxis [Time Frame: Weeks 5-12 of active treatment phase]

Secondary Outcome(s)

Duration of Epistaxis [Time Frame: 5-12 weeks of active treatment]

Hemoglobin Level [Time Frame: 12 weeks]

Hoag Epistaxis Severity Score [Time Frame: 12 weeks]

Number of Participants With Treatment Failure [Time Frame: Baseline through 12 weeks]

Number of Participants Requiring Red Blood Cell (RBC) Transfusion [Time Frame: 12 weeks]

Secondary ID(s)

GHSU 1008041

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

HHT Foundation International

Results

• Results available: Yes
• Date Posted: 19/10/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01408030