NOrth American Study for the Treatment of Recurrent epIstaxis With DoxycycLine: The NOSTRIL Trial

NCT04167085 | Completed Phase 4 Results FDA Drug

• 1 other identifier: 16-001827
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to determine whether Doxycycline can be used to control nosebleeds (epistaxis) for patients with Hereditary Hemorrhagic Telangiectasia (HHT). Patients with HHT will be randomized to one of 2 study arms: Doxycycline or Placebo for a period of 2 months followed by a 1-month washout period before switching treatments for a further 2 months period. Observation and evaluation will continue for a period of one month after treatment is completed.

Conditions

Epistaxis

Keywords

Epistaxis; Hereditary Hemorrhagic Telangiectasia; HHT; Doxycycline; Nosebleed

Outcome Measures

Primary Outcomes (3)

• Frequency of Epistaxis

  Change in number of bleeding episodes per week by participant self-report via nosebleed diary

  Baseline, post-washout - approximately 6 months

• Duration of Epistaxis

  Change in duration of bleeding per week by patient self-report via nosebleed diary

  Baseline, post-washout - approximately 6 months

• Change in Severity of Epistaxis

  Epistaxis Severity Scale (ESS) for Hereditary Hemorrhagic Telangiectasia, is based on 6 nosebleed variables such as frequency and duration which are reported by patients.The ESS has a minimum value of 0 and maximum value of 10, with 10 representing more severe epistaxis.

  Baseline, post-washout - approximately 6-months

Secondary Outcomes (5)

• Change in Quality of Life

  Baseline, post-washout - approximately 6 months

• Change in Ferritin Level

  Baseline, post-washout - approximately 6 months

• Change in Hemoglobin Level

  Baseline, post-washout (approximately 6 months)

• Change in Monthly Units of Packed Red Blood Cells (PRBCs) Transfused

  Baseline, post-washout (approximately 6 months)

• Incidence of Treatment Failure

  Baseline, post-washout (approximately 6 months)

Study Arms (2)

Doxycycline, then Placebo

EXPERIMENTAL

Doxycycline for a period of 2 months followed by a 1-month washout period, and then placebo for a further 2 months period followed by a 1-month washout period.

Drug: Doxycycline; Other: Placebo

Placebo, then Doxycycline

EXPERIMENTAL

Placebo for a period of 2 months followed by a 1-month washout period, and then Doxycycline for a further 2 months period followed by a 1-month washout period.

Drug: Doxycycline; Other: Placebo

Interventions

Doxycycline DRUG

Doxycycline, 100 mg twice a day for 2 months

Doxycycline, then Placebo; Placebo, then Doxycycline

Placebo OTHER

Matching placebo, twice a day for 2 months

Doxycycline, then Placebo; Placebo, then Doxycycline

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of definite HHT by the Curacao criteria or genetic diagnosis of HHT
• Epistaxis severity during observation month at least moderate by Epistaxis Severity Score (ESS) evaluation
• For female participants, a negative pregnancy test at Day -1 and agree to use birth control during treatment and for 28 days following cessation of Doxycycline

You may not qualify if:

• Use of medication(s) contraindicated with doxycycline use within 14 days prior to the study (including barbiturates, tegretol, dilantin, warfarin, isotretinoin)

Sponsors & Collaborators

• University of California, Los Angeles: lead

Study Sites (1)

100 UCLA Medical Plaza

Los Angeles, California, 90095, United States

Location

MeSH Terms

Conditions

Epistaxis; Telangiectasia, Hereditary Hemorrhagic

Interventions

Doxycycline

Condition Hierarchy (Ancestors)

Nose Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Respiratory; Signs and Symptoms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Tetracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Results Point of Contact

• Title: Justin P. McWilliams, MD, FSIR
• Organization: David Geffen School of Medicine at UCLA

jumcwilliams@mednet.ucla.edu

(310) 267-8773

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 14, 2019
• First Posted: November 18, 2019
• Study Start: December 18, 2017
• Primary Completion: June 1, 2021
• Study Completion: June 1, 2021
• Last Updated: July 20, 2022
• Results First Posted: July 20, 2022

Record last verified: 2022-06

Locations

US (1)