UveitisPhase 1NCT00280826

Efalizumab to Treat Uveitis

A Phase 1 study testing Efalizumab in people with Uveitis and Macular Edema, run by National Eye Institute (NEI) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Uveitis, Macular Edema
What is being tested?: Efalizumab
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 6 participants
Where: 1 siteUnited States
Study dates: Jan 2006to Feb 2009
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

United States1 site

National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: Treatment of Non-Infectious Intermediate and Posterior Uveitis Associated Macular Edema With Humanized Anti-CD11a Antibody Therapy

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.

Read the detailed description

Background: Uveitis refers to intraocular inflammatory diseases that are an important cause of visual loss. Standard systemic immunosuppressive medications for uveitis can cause significant adverse effects. Consequently, an effective treatment with a safer side effect profile is highly desirable.

Aims: This protocol evaluated the safety and potential efficacy of subcutaneous (SC) efalizumab (anti-CD11a) treatments for uveitis while reducing or eliminating standard medications commensurate with the standard of care. If the therapeutic benefit was sustained using the SC formulation, then maintenance therapy was continued as clinically indicated.

Methods: This was an open-label, non-randomized, clinical pilot study.

Conditions: Uveitis, Macular Edema
Being tested: Efalizumab
Keywords: OCT, Retinal Disease, Adhesion Molecule, Ocular Inflammation, Raptiva, Macular Edema, Uveitis, Immunosuppression
Medical subject headings: Uveitis, Macular Edema, Retinal Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

EfalizumabDrug
Also called Raptiva
Participants who qualified for the study received weekly subcutaneous treatments of efalizumab, with the first dose being a test dose of 0.7 mg/kg and subsequent doses of 1 mg/kg (not to exceed 200 mg per dose), for a total treatment duration of 16 weeks.

Study groups in NCT00280826
Study group	Role	What they receive
Efalizumab	Experimental	Not described Receives: Drug: Efalizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Systemic Toxicities, Adverse Events, or Infections
Safety outcomes were recorded by observing and tabulating the nature, severity and frequency of systemic toxicities, adverse events and infections throughout the study. Safety assessments were made by the investigators continuously during the study, with a review of the previous visit interval performed at each scheduled visit. Each participant was also encouraged to report any apparent adverse events between scheduled visits and could return for additional evaluations or treatment between scheduled visits if needed.
Measured: 16 weeks

Secondary outcomes (4)

Cystoid Macular Edema in the Worse Eye as Assessed by Optical Coherence Tomography (OCT).
Worse eye indicates the eye with the worst visual acuity (VA).
Measured: Baseline and 16 weeks
Cystoid Macular Edema in the Better Eye as Assessed by Optical Coherence Tomography (OCT).
Better eye indicates the eye with better VA.
Measured: Baseline and 16 weeks
Change in Visual Acuity in the Worse Eye From Baseline to 16 Weeks
Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. This acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters the Snellen measurement is 20/20.
Measured: Baseline and 16 weeks
Change in Visual Acuity in the Better Eye From Baseline to 16 Weeks
Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. This acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters the Snellen measurement is 20/20.
Measured: Baseline and 16 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2006Feb 2009

First posted23 Jan 2006 (estimated)
Study startJan 2006
Primary completionFeb 2009
Study completionFeb 2009

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (4)Papers the sponsor has linked to this study.
Other trials for UveitisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 6 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00280826
Sponsor's protocol ID: 060046
Other IDs: 06-EI-0046
Status last verified: Jan 2011
First submitted: 21 Jan 2006
Last updated: 2 Feb 2011
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Djalilian AR, Nussenblatt RB. Immunosuppression in uveitis. Ophthalmol Clin North Am. 2002 Sep;15(3):395-404, viii. doi: 10.1016/s0896-1549(02)00036-6.
PubMed 12434489
Leonardi CL. Efalizumab: an overview. J Am Acad Dermatol. 2003 Aug;49(2 Suppl):S98-104. doi: 10.1016/s0190-9622(03)01141-1.
PubMed 12894132
Whitcup SM, Hikita N, Shirao M, Miyasaka M, Tamatani T, Mochizuki M, Nussenblatt RB, Chan CC. Monoclonal antibodies against CD54 (ICAM-1) and CD11a (LFA-1) prevent and inhibit endotoxin-induced uveitis. Exp Eye Res. 1995 Jun;60(6):597-601. doi: 10.1016/s0014-4835(05)80001-6.
PubMed 7641842
Faia LJ, Sen HN, Li Z, Yeh S, Wroblewski KJ, Nussenblatt RB. Treatment of inflammatory macular edema with humanized anti-CD11a antibody therapy. Invest Ophthalmol Vis Sci. 2011 Sep 1;52(9):6919-24. doi: 10.1167/iovs.10-5896.
PubMed 21498606

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Uveitis trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Feb 2011. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Treatment of Non-Infectious Intermediate and Posterior Uveitis Associated Macular Edema With Humanized Anti-CD11a Antibody Therapy
ClinicalTrials.gov ID: NCT00280826
Acronym / sponsor ID: 060046
Conditions: Uveitis, Macular Edema
Keywords: OCT, Retinal Disease, Adhesion Molecule, Ocular Inflammation, Raptiva, Macular Edema, Uveitis, Immunosuppression
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 6 participants (actual)
Lead sponsor: National Eye Institute (NEI)US National Institutes of Health
Study start: Jan 2006
Primary completion: Feb 2009 (actual)
Study completion: Feb 2009 (actual)
First posted: 23 Jan 2006 (estimated)
Results: Posted 27 Jan 2011

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

Methods: This was an open-label, non-randomized, clinical pilot study.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Efalizumab	Experimental	Drug: Efalizumab

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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.

Key exclusion criteria (3 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
EfalizumabAlso: RaptivaParticipants who qualified for the study received weekly subcutaneous treatments of efalizumab, with the first dose being a test dose of 0.7 mg/kg and subsequent doses of 1 mg/kg (not to exceed 200 mg per dose), for a total treatment duration of 16 weeks.	Drug	Efalizumab

Primary outcome

Number of Participants With Systemic Toxicities, Adverse Events, or Infections
Time frame: 16 weeks
Safety outcomes were recorded by observing and tabulating the nature, severity and frequency of systemic toxicities, adverse events and infections throughout the study. Safety assessments were made by the investigators continuously during the study, with a review of the previous visit interval performed at each scheduled visit. Each participant was also encouraged to report any apparent adverse events between scheduled visits and could return for additional evaluations or treatment between scheduled visits if needed.

4 secondary outcomes

Cystoid Macular Edema in the Worse Eye as Assessed by Optical Coherence Tomography (OCT).
Time frame: Baseline and 16 weeks
Cystoid Macular Edema in the Better Eye as Assessed by Optical Coherence Tomography (OCT).
Time frame: Baseline and 16 weeks
Change in Visual Acuity in the Worse Eye From Baseline to 16 Weeks
Time frame: Baseline and 16 weeks
Change in Visual Acuity in the Better Eye From Baseline to 16 Weeks
Time frame: Baseline and 16 weeks

Results and safety

Results posted to the registry on 27 Jan 2011, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Efalizumab	6	6

Primary outcome: Number of Participants With Systemic Toxicities, Adverse Events, or Infections

Time frame: 16 weeks. Number. Unit: Participants

Measure	Efalizumab n = 6
Value	6

Population analysed: Analysis was per protocol

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were collected between October 2006 and July 2008.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Efalizumab	0 of 6 (0%)	6 of 6 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2011.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 12434489Djalilian AR, Nussenblatt RB. Immunosuppression in uveitis. Ophthalmol Clin North Am. 2002 Sep;15(3):395-404, viii. doi: 10.1016/s0896-1549(02)00036-6.
Background: PubMed 12894132Leonardi CL. Efalizumab: an overview. J Am Acad Dermatol. 2003 Aug;49(2 Suppl):S98-104. doi: 10.1016/s0190-9622(03)01141-1.
Background: PubMed 7641842Whitcup SM, Hikita N, Shirao M, Miyasaka M, Tamatani T, Mochizuki M, Nussenblatt RB, Chan CC. Monoclonal antibodies against CD54 (ICAM-1) and CD11a (LFA-1) prevent and inhibit endotoxin-induced uveitis. Exp Eye Res. 1995 Jun;60(6):597-601. doi: 10.1016/s0014-4835(05)80001-6.
Derived from this study: PubMed 21498606Faia LJ, Sen HN, Li Z, Yeh S, Wroblewski KJ, Nussenblatt RB. Treatment of inflammatory macular edema with humanized anti-CD11a antibody therapy. Invest Ophthalmol Vis Sci. 2011 Sep 1;52(9):6919-24. doi: 10.1167/iovs.10-5896.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Robert Nussenblatt, MD, MPH principal investigator

National Eye Institute (NEI)

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Efalizumab in people with Uveitis and Macular Edema, run by National Eye Institute (NEI) at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Feb 2011. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 Jan 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Systemic Toxicities, Adverse Events, or Infections (time frame: 16 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00280826, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Feb 2011.