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Ranibizumab for the Management of Recurrent Nosebleeds in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

Primary Purpose

Hereditary Hemorrhagic Telangiectasia (HHT), Nosebleeds

Status
Withdrawn
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Ranibizumab
Sponsored by
University of California, San Diego
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Hemorrhagic Telangiectasia (HHT) focused on measuring Hereditary Hemorrhagic Telangiectasia (HHT), Nosebleeds, Ranibizumab, Lucentis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria

Subjects will be eligible if the following criteria are met:

  • Ability to provide written informed consent and comply with study assessments for the full duration of the study
  • Age > 18 years
  • Diagnosed with HHT
  • Deemed appropriate for topical Ranibizumab without laser, as determined by severity. For the Ranibizumab to be effective, the spray needs to reach the nasal mucosa. Patients will be instructed in nasal irrigation and will only be treated if they can keep their noses clean.
  • Severe patients will presumably not achieve this goal, so subjects will be patients with moderate disease as defined by the epistaxis severity score sheet (Appendix B)

Exclusion Criteria

Subjects who meet any of the following criteria will be excluded from this study:

  • Coagulopathy
  • Pregnancy (positive pregnancy test) or lactation
  • Premenopausal women not using adequate contraception. The following are considered effective means of contraception: surgical sterilization or use of oral contraceptives, barrier contraception with either a condom or diaphragm in conjunction with spermicidal gel, an IUD, or contraceptive hormone implant or patch
  • Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated
  • Participation in another simultaneous medical investigation or trial
  • Patients who have had bevacizumab therapy, injection, or spray
  • Patients with a known reaction based on macular degeneration treatment
  • Patients having received VEGF inhibitors in the last 2 years for the treatment of any ophthalmologic disease

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Ranibizumab

    Arm Description

    Patients treated with topical ranibizumab.

    Outcomes

    Primary Outcome Measures

    Epistaxis as measured by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum feritin levels.
    Epistaxis will be evaluated on a monthly basis by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum ferritin levels. This will continue through 6 months after the first treatment.

    Secondary Outcome Measures

    Full Information

    First Posted
    July 29, 2011
    Last Updated
    April 26, 2013
    Sponsor
    University of California, San Diego
    Collaborators
    Genentech, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01406639
    Brief Title
    Ranibizumab for the Management of Recurrent Nosebleeds in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
    Official Title
    Ranibizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2013
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    Study withdrawn due to lack of study population.
    Study Start Date
    July 2011 (undefined)
    Primary Completion Date
    May 2012 (Actual)
    Study Completion Date
    May 2012 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    University of California, San Diego
    Collaborators
    Genentech, Inc.

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    This study is for patients with recurrent epistaxis (nosebleeds) as a result of Hereditary Hemorrhagic Telangiectasia (HHT). The aim is to determine if ranibizumab, topically applied will diminish epistaxis in patients with HHT as measured by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum ferritin levels.
    Detailed Description
    This is an open-label, Phase I study of intranasal administered ranibizumab in subjects with HHT. Plans are to recruit patients with HHT from the UCSD Nasal Dysfunction Clinic. Those who come for evaluation and are deemed appropriate for topical ranibizumab without laser will be recruited for this study. Ten (10) consented, enrolled subjects will receive a pulsatile nasal irrigator to clean their nose twice daily. They will complete the initial ESS and have a blood test for Hct, Hgb, serum ferritin, liver function tests, renal function tests and if of child-bearing age, a urine pregnancy test. They will return 1 week later and will then receive intranasal sprays of ranibizumab administered once per week during clinic. Patients will be treated with a total of 4 applications of ranibizumab in a fine mist spray. This will be done with 2 mgs in 0.2cc; 0.1cc sprayed into each nostril a day; therefore 8 mgs will require 4 days of application. If, following the first treatments, they experience recurrent bleeding (equal to or less than a 75% reduction in their ESS number as compared to pre treatment), they will be offered the opportunity to be prescribed a second administration, to be completed in the same way (4 applications of 4 mg in 0.4 cc with 0.2cc per nostril). At the completion of the treatment, subjects will be observed monthly for examination and ESS until 6 months from the first treatment. At 3 and 6 months laboratory tests will be performed.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Hereditary Hemorrhagic Telangiectasia (HHT), Nosebleeds
    Keywords
    Hereditary Hemorrhagic Telangiectasia (HHT), Nosebleeds, Ranibizumab, Lucentis

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Ranibizumab
    Arm Type
    Experimental
    Arm Description
    Patients treated with topical ranibizumab.
    Intervention Type
    Drug
    Intervention Name(s)
    Ranibizumab
    Other Intervention Name(s)
    Lucentis
    Intervention Description
    Patients will be treated with a total of 4 applications of ranibizumab in a fine mist spray. This will be done with 2 mgs in 0.2cc; 0.1cc sprayed into each nostril a day; therefore 8 mgs will require 4 days of application. If, following the first treatments, they experience recurrent bleeding (equal to or less than a 75% reduction in their ESS number as compared to pre treatment), they will be offered the opportunity to be prescribed a second administration, to be completed in the same way (4 applications of 4 mg in 0.4 cc with 0.2cc per nostril).
    Primary Outcome Measure Information:
    Title
    Epistaxis as measured by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum feritin levels.
    Description
    Epistaxis will be evaluated on a monthly basis by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum ferritin levels. This will continue through 6 months after the first treatment.
    Time Frame
    Monthly until 6 months from the first treatment.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria Subjects will be eligible if the following criteria are met: Ability to provide written informed consent and comply with study assessments for the full duration of the study Age > 18 years Diagnosed with HHT Deemed appropriate for topical Ranibizumab without laser, as determined by severity. For the Ranibizumab to be effective, the spray needs to reach the nasal mucosa. Patients will be instructed in nasal irrigation and will only be treated if they can keep their noses clean. Severe patients will presumably not achieve this goal, so subjects will be patients with moderate disease as defined by the epistaxis severity score sheet (Appendix B) Exclusion Criteria Subjects who meet any of the following criteria will be excluded from this study: Coagulopathy Pregnancy (positive pregnancy test) or lactation Premenopausal women not using adequate contraception. The following are considered effective means of contraception: surgical sterilization or use of oral contraceptives, barrier contraception with either a condom or diaphragm in conjunction with spermicidal gel, an IUD, or contraceptive hormone implant or patch Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated Participation in another simultaneous medical investigation or trial Patients who have had bevacizumab therapy, injection, or spray Patients with a known reaction based on macular degeneration treatment Patients having received VEGF inhibitors in the last 2 years for the treatment of any ophthalmologic disease
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Terence Davidson, M.D.
    Organizational Affiliation
    UCSD
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Learn more about this trial

    Ranibizumab for the Management of Recurrent Nosebleeds in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

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