Phase 1
N=6
Efalizumab to Treat Uveitis
Uveitis · Macular Edema
Bottom Line
View on ClinicalTrials.gov: NCT00280826 ↗Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Jan 2011
Primary outcome: Primary: Number of Participants With Systemic Toxicities, Adverse Events, or Infections — 6 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Efalizumab (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- National Eye Institute (NEI)
- Primary completion
- Feb 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Systemic Toxicities, Adverse Events, or Infections |
6 | — |
| SECONDARY Cystoid Macular Edema in the Worse Eye as Assessed by Optical Coherence Tomography (OCT). |
128 | — |
| SECONDARY Cystoid Macular Edema in the Better Eye as Assessed by Optical Coherence Tomography (OCT). |
57 | — |
| SECONDARY Change in Visual Acuity in the Worse Eye From Baseline to 16 Weeks |
6.7 | — |
| SECONDARY Change in Visual Acuity in the Better Eye From Baseline to 16 Weeks |
1.7 | — |
Summary
This study examined the safety and potential efficacy of the monoclonal antibody efalizumab (Raptiva) for treating sight-threatening uveitis (eye inflammation). Efalizumab controls the activity of white blood cells called lymphocytes that cause inflammation. The drug is currently approved in the United States to treat patients with moderate to severe psoriasis.
Participants 18 and older with sight-threatening intermediate or posterior uveitis of at least 3 months duration, causing persistent macular edema in one or both eyes, were eligible for this study. The uveitis required treatment with at least 20 milligrams per day of prednisone, or the equivalent, or a combination of two or more anti-inflammatory treatments such as prednisone, methotrexate, cyclophosphamide, cyclosporine, etc.
Participants underwent the following tests and procedures:
* Medical history and physical examination.
* Weekly efalizumab treatment.
* Weekly eye examination, including measurement of vision and pressure in the eyes, dilation of the eyes and examination of the front and back parts of the eye.
* Weekly blood tests to measure the number and types of cells in the blood and to check for signs of inflammation and treatment side effects. At some visits, blood samples were collected to measure how much efalizumab remains in the blood and whether the body has developed an immune response to the medicine.
* Blood draw at enrollment and at 2 and 4 months for research tests to examine how participants' immune response was operating.
* Fluorescein angiography at enrollment and 1 and 3 months after enrollment, unless additional tests are needed, for medical management. This test checked for abnormalities of eye blood vessels. A yellow dye was injected into an arm vein and travels to the blood vessels in the eyes. Pictures of the retina (the back portion of the eye) were taken with a special camera that flashes a blue light into the eye. The pictures show whether any dye has leaked from the vessels into the retina, indicating possible abnormalities.
* Monthly pregnancy test for women who could become pregnant.
Participants returned for treatment and clinic visits weekly for 16 weeks. After 16 weeks, participants whose macular edema had decreased and whose vision may have improved were offered to continue the injections.
Eligibility Criteria
Inclusion Criteria
- Participant is 18 years of age or older;
- Participant has a diagnosis of sight-threatening, intermediate or posterior uveitis of at least three months duration prior to original enrollment that is causing persistent cystoid macular edema in one or both eyes. Their disease requires treatment to control their intraocular inflammatory disease with at least 20 mg/day of prednisone (or equivalent) or any combination of two or more anti-inflammatory treatments for uveitis, including for example prednisone, cyclophosphamide, cyclosporine, azathioprine, mycophenolate mofetil, methotrexate, etc.
- Participant exhibits intolerance to the indicated systemic medications required for their uveitis or, though their uveitis may be under control, wish to be taken off their present medications due to potential or actual unacceptable side effects.
- Participant has visual acuity in at least one eye of 20/200 or better.
- Participant has normal renal or liver function or no worse than mild abnormalities as defined by the Common Toxicity Criteria.
- Participant is not currently pregnant or lactating.
- Both men and women with reproductive potential and who are sexually active agree to use acceptable birth control methods throughout the course of the study and for six weeks following the last administration of the study medication.
- Participant must have the ability to understand and sign an informed consent form.
Exclusion Criteria
- Participants who had received previous treatment with an intercellular adhesion molecule (ICAM) or lymphocyte function-associated antigen-1 (LFA-1) directed monoclonal antibody or any other investigational agent that would interfere with the ability to evaluate the safety, efficacy or pharmacokinetics of efalizumab.
- Participant has a significant active infection.
- Participant has a history of cancer (other than a non-melanoma skin cancer) diagnosed within the past 5 years.
Data sourced from ClinicalTrials.gov (NCT00280826). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.