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

NCT01406639 | Withdrawn Phase 1

• 1 other identifier: 091219
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT); Nosebleeds

Keywords

Hereditary Hemorrhagic Telangiectasia (HHT); Nosebleeds; Ranibizumab; Lucentis

Outcome Measures

Primary Outcomes (1)

• 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.

  Monthly until 6 months from the first treatment.

Study Arms (1)

Ranibizumab

EXPERIMENTAL

Patients treated with topical ranibizumab.

Drug: Ranibizumab

Interventions

Ranibizumab DRUG

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).

Also known as: Lucentis

Ranibizumab

Eligibility Criteria

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

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• University of California, San Diego: lead
• Genentech, Inc.: collaborator

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic; Epistaxis

Interventions

Ranibizumab

Condition Hierarchy (Ancestors)

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; Nose Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Respiratory; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Terence Davidson, M.D.

  UCSD

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Dean/Professor

Study Record Dates

• First Submitted: July 29, 2011
• First Posted: August 1, 2011
• Study Start: July 1, 2011
• Primary Completion: May 1, 2012
• Study Completion: May 1, 2012
• Last Updated: April 29, 2013

Record last verified: 2013-04