Ranibizumab for Neovascularization in Sickle Cell Retinopathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00618644
Recruitment Status : Withdrawn (Withdrawn due to lack of eligible participants)
First Posted : February 20, 2008
Last Update Posted : September 27, 2012
Wayne State University
Genentech, Inc.
Information provided by:
Kresge Eye Institute

Brief Summary:
The purpose of this study is to determine the ocular and non-ocular safety of a single dose of ranibizumab in treating neovascularization secondary to sickle cell retinopathy.

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Retinopathy Drug: Ranibizumab Not Applicable

Detailed Description:

In the U.S., about 10% of African Americans have an abnormal hemoglobin gene. About 8% of African Americans are heterozygous for Hemoglobin S. In the United States, sickle cell anemia primarily occurs in the black population, with approximately 0.2% of African American children afflicted by this disease. It may be associated with other hemoglobinopathies as well. The prevalence in adults is lower because of the decrease in life expectancy. Systemically, the sickle cell anemia variation (SS) produces the most symptoms. With respect to the eye, the sickle cell disease mutation (SC) produces the most effects. Overall, the sickle cell trait expression (AS) produces the fewest complications.

  • Among patients with SC or SThal, the incidence of proliferation sickle cell retinopathy is 33% and 14% respectively.
  • Proliferative sickle cell retinopathy is the major cause of vision loss in sickle cell disease.

For sickle cell retinopathy, the commonly used therapeutic modalities include laser retinal photocoagulation, retinal cryotherapy, and vitrectomy/membranectomy depending on the severity of the disease. The most effective therapeutic modality with minimal postoperative complications appears to be scatter laser retinal photocoagulation.

A single case study of bevacizumab was found to effective in short term regression of neovascularization and improving vision after a single injection. Further study with ranibizumab is warranted.

Recent clinical trials (Marina and Anchor) have demonstrated that ranibizumab is effective in treating patients with CNV with age-related macular degeneration. Retinopathy in sickle cell disease has also been linked to VEGF. Therefore, patients with sickle cell retinopathy should respond to ranibizumab therapy.

This is an open-label single dose, phase I study of intravitreally administered ranibizumab in patients with sickle cell retinopathy.

Consented, enrolled subjects will receive a single open-label intravitreal injection of 0.5 mg ranibizumab.

Three subjects from one site in the United States will be enrolled.

Patients will receive one dose of 0.5 mg ranibizumab administered intravitreally.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase I Study to Evaluate the Ocular and Non Ocular Safety of Ranibizumab in Treating Neovascularization Secondary to Sickle Cell Retinopathy
Study Start Date : January 2010
Actual Primary Completion Date : June 2011
Actual Study Completion Date : June 2011

Resource links provided by the National Library of Medicine

Drug Information available for: Ranibizumab

Arm Intervention/treatment
Experimental: 1
Ranibizumab injection
Drug: Ranibizumab
Ranibizumab 0.5 mg intravitreal injection
Other Name: Lucentis (ranibizumab)

Primary Outcome Measures :
  1. Ocular safety of a single dose of ranibizumab [ Time Frame: Three months ]

Secondary Outcome Measures :
  1. Change in vision status [ Time Frame: Three months ]
  2. To evaluate ocular hemorrhage [ Time Frame: Three months. ]

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

Inclusion Criteria:

  • Patients with sickle cell anemia and retinopathy
  • Over age 18 years
  • Non-pregnant

Exclusion Criteria:

  • Pregnant
  • Glaucoma
  • Patients using anticoagulants (e.g., warfarin)
  • Retinal detachment

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 identifier (NCT number): NCT00618644

United States, Michigan
Kresge Eye Institute
Detroit, Michigan, United States, 48201
Sponsors and Collaborators
Kresge Eye Institute
Wayne State University
Genentech, Inc.
Principal Investigator: Vinay Shah, MD Kresge Eye Institute

Responsible Party: Vinay A. Shah, M.D., Kresge Eye Institute Identifier: NCT00618644     History of Changes
Other Study ID Numbers: 08-08
First Posted: February 20, 2008    Key Record Dates
Last Update Posted: September 27, 2012
Last Verified: September 2012

Additional relevant MeSH terms:
Retinal Diseases
Neovascularization, Pathologic
Anemia, Sickle Cell
Eye Diseases
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Immunologic Factors
Physiological Effects of Drugs
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents