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Trial record 29 of 35 for:    HHT | United States

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

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ClinicalTrials.gov Identifier: NCT04167085
Recruitment Status : Recruiting
First Posted : November 18, 2019
Last Update Posted : December 10, 2019
Sponsor:
Information provided by (Responsible Party):
Justin McWilliams, MD, University of California, Los Angeles

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.

Condition or disease Intervention/treatment Phase
Epistaxis Drug: Doxycycline Other: Placebo Phase 4

Detailed Description:

This study will analyze the effect of doxycycline on nosebleeds in hereditary hemorrhagic telangiectasia (HHT), a rare and debilitating medical condition. Patients with HHT have a variety of vascular abnormalities, including changes in their nasal vasculature that result in chronic, severe nosebleeds. These nosebleeds have been shown to significantly reduce the quality of life in HHT and can be severe enough to require emergent blood transfusion. Current therapies to control nosebleeds in HHT, which include both medications and surgical treatments, are inconsistently effective and associated with a variety of problematic side effects and complications.

Doxycycline is a common, FDA-approved antibiotic medication that has been used by physicians for decades. It is a safe medication that can be used on an outpatient basis for extended periods of time with minimal side effects. Recent research has shown that doxycycline is a potent inhibitor of angiogenesis, the process of new blood vessel development. Since the vascular abnormalities in HHT form due to uncontrolled angiogenesis, it follows that doxycycline may have potential in treating HHT. A growing body of research demonstrates the efficacy of doxycycline in treating a variety of vascular conditions, including cancer of the breast, duodenum, liver prostate, and lung. Our trial will be the first to analyze the efficacy of doxycycline in treating HHT epistaxis.

This a randomized, double-blind, crossover study design. Each patient will take both placebo and doxycycline. Since this is a cross-over study, half the participants will start out with taking placebo and half start with doxycyline and then switch halfway through. After a baseline observation period, participants will be given doxycycline at 100 mg twice a day, or an identical-appearing placebo, for a period of three months. Each month they will be seen be a physician, who will take a detailed clinical history and perform a physical examination. Further, each month the patient will have blood testing to assess blood loss, and fill out scientifically validated surveys to assess quality of life and the severity of bleeding. Following this three month treatment period, participants will be observed for one additional month, with one additional blood test and clinical assessment performed after this month.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: NOrth American Study for the Treatment of Recurrent epIstaxis With DoxycycLine: The NOSTRIL Trial
Actual Study Start Date : December 18, 2017
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : July 2020


Arm Intervention/treatment
Experimental: Doxycycline, then Placebo
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
Doxycycline, 100 mg twice a day for 2 months

Other: Placebo
Matching placebo, twice a day for 2 months

Experimental: Placebo, then Doxycycline
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
Doxycycline, 100 mg twice a day for 2 months

Other: Placebo
Matching placebo, twice a day for 2 months




Primary Outcome Measures :
  1. Frequency of epistaxis [ Time Frame: Approximately 6 months ]
    Nosebleed diary

  2. Duration of epistaxis [ Time Frame: Approximately 6 months ]
    Nosebleed diary

  3. Severity of epistaxis [ Time Frame: Approximately 6 months ]
    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.


Secondary Outcome Measures :
  1. Participant's Quality of Life [ Time Frame: Approximately 6 months ]
    The 12-item Short Form (SF-12) is a survey designed for use with patients with multiple chronic conditions. This 12-item scale can be used to assess the physical and mental health of respondents. 10 of the 12 questions are answered on a 5-point Likert scale and 2 are answered on a 3-point Likert scale. The questions are then scored and weighted into 2 subscales, physical health and mental health. Respondents can have a score that ranges from 0-100 with 100 being the best score and indicating high physical or mental health.

  2. Ferritin level [ Time Frame: Approximately 6 months ]
  3. Hemoglobin level [ Time Frame: Approximately 6 months ]
  4. Units of packed red blood cells (PRBCs) transfused [ Time Frame: Approximately 6 months ]
  5. Incidence of treatment failure [ Time Frame: Approximately 6 months ]
    Treatment failure will be defined as need for nasal surgery or other epistaxis treatments



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

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

Information from the National Library of Medicine

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): NCT04167085


Contacts
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Contact: Stephanie Gilbert, BA 310-794-0376 ext 40376 SGilbert@mednet.ucla.edu
Contact: Saima Chaabane, PhD 310-794-8995 SChaabane@mednet.ucla.edu

Locations
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United States, California
100 UCLA Medical Plaza Recruiting
Los Angeles, California, United States, 90095
Contact: Stephanie Gilbert, BA    310-794-0376    SGilbert@mednet.ucla.edu   
Sponsors and Collaborators
University of California, Los Angeles
Investigators
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Principal Investigator: Justin McWilliams, MD University of California, Los Angeles

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Responsible Party: Justin McWilliams, MD, Principal Investigator, University of California, Los Angeles
ClinicalTrials.gov Identifier: NCT04167085    
Other Study ID Numbers: 16-001827
First Posted: November 18, 2019    Key Record Dates
Last Update Posted: December 10, 2019
Last Verified: December 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Justin McWilliams, MD, University of California, Los Angeles:
Epistaxis
Hereditary Hemorrhagic Telangiectasia
HHT
Doxycycline
Nosebleed
Additional relevant MeSH terms:
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Epistaxis
Nose Diseases
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Hemorrhage
Pathologic Processes
Signs and Symptoms, Respiratory
Signs and Symptoms
Doxycycline
Anti-Bacterial Agents
Anti-Infective Agents
Antimalarials
Antiprotozoal Agents
Antiparasitic Agents