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Withdrawn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
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
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
NCT ID
NCT01406639
First Posted
July 29, 2011
Last Updated
April 26, 2013
Sponsor
University of California, San Diego
Collaborators
Genentech, Inc.
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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