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

Phase 1

Withdrawn

• Conditions: Hereditary Hemorrhagic Telangiectasia (HHT); Nosebleeds
• Interventions: Drug: Ranibizumab

• Registration Number: NCT01406639
• Lead Sponsor: University of California, San Diego

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.

Study Timeline

• Study Start: 2011-07
• Study First Submitted: 2011-07-29
• Study First Submitted QC: 2011-07-29
• Study First Posted: 2011-08-01
• Primary Completion: 2012-05
• Study Completion: 2012-05
• Status Verified: 2013-04
• Last Update Submitted: 2013-04-26
• Last Update Posted: 2013-04-29

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ranibizumab	Ranibizumab	Patients treated with topical ranibizumab.

Primary Outcome Measures

Name	Time	Method
Epistaxis as measured by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum feritin levels.	Monthly until 6 months from the first treatment.	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.