Ranibizumab for Neovascularization in Sickle Cell Retinopathy

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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A Phase I Study to Evaluate the Ocular and Non Ocular Safety of Ranibizumab in Treating Neovascularization Secondary to Sickle Cell Retinopathy

Conditions
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Sickle Cell Anemia, Retinopathy

Intervention / Treatment
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* Drug: Ranibizumab


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>Ranibizumab injection | Drug: Ranibizumab<br>* Ranibizumab 0.5 mg intravitreal injection<br>* Other names: Lucentis (ranibizumab);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ocular safety of a single dose of ranibizumab |  | Three months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in vision status |  | Three months | 
| To evaluate ocular hemorrhage |  | Three months. |