Infliximab, Sirolimus and Daclizumab to Treat Age-Related Macular Degeneration

NCT ID: NCT00304954

Last Updated: 2016-11-29

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-02-28

Study Completion Date

2010-01-31

Brief Summary

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This study examined whether the anti-inflammatory medicines infliximab, sirolimus or daclizumab, when given with a participant's current therapies, would prevent the growth of new blood vessels in the eye in participants with age-related macular degeneration (AMD).

Participants 55 years of age and older with AMD and drusen larger than 63um may be eligible for this study. Vision in the study eye was between 20/20 and 20/400.

Participants were randomly assigned to one of three treatments - infliximab, sirolimus, or daclizumab - or to observation only. In addition, participants may have been treated by their ophthalmologist as needed for their AMD.

Infliximab and daclizumab were given intravenously (through a vein); infusions were given at enrollment in the study, then at 2 weeks, and then monthly.

Sirolimus was a pill that was taken every other day for the duration of the study. At 6 months, participants were evaluated to see whether continuing treatment would be beneficial.

In addition to treatment or observation, participants underwent the following procedures:

Physical examination at enrollment and 6 months.

Photographs of the back of the eye, fluorescein angiography, indocyanine green angiography and measurement of retinal thickness at enrollment and months 1, 3 and 6.

* Fluorescein angiography evaluated the eye's blood vessels. A yellow dye was injected into an arm vein and traveled to the blood vessels in the eyes. Pictures of the retina were taken using a camera that flashed a blue light into the eye. The pictures show if any dye has leaked from the vessels into the retina, indicating possible blood vessel abnormality.
* Indocyanine green angiography identified feeder vessels that may have supplied abnormal blood vessels. This procedure is similar to fluorescein angiography, but uses a green dye and flashes an invisible light.
* Optical coherence tomography measures retinal thickness. This test shines a light into the eye and produces cross-sectional pictures of the retina. These measurements are repeated during the study to determine whether retinal thickening is getting better or worse, or staying the same.

Tuberculin skin test and chest x-ray at enrollment and 6 months.

Blood tests at enrollment and months 1, 3 and 6.

Detailed Description

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There has been much interest in the possible role of the immune system in AMD. Experimental models and patient material have, to date, suggested a role for macrophages and complement. This study hypothesized that the underlying mechanism that leads to choroidal neovascularization (CNV) is similar to those at play in atherosclerosis. If this is the case, the investigators believed that CNV treatment should be amenable to new immunomodulatory agents directed against specific parts of the immune system.

After therapy with anti-angiogenic agents not leading to a persistent remission of CNV due to AMD, participants were treated with one of three immunomodulatory agents or were observed in conjunction with their continued anti-angiogenic therapy. Thus the participant continued with the anti-angiogenic therapy they received after randomization. The investigators hypothesized that this combination therapy would inhibit progression of CNV associated with AMD.

This was an open-label, phase II, randomized, single center clinical trial of 18 study participants randomized to receive one of three immunomodulatory agents or was observed in conjunction with their anti-angiogenic therapy.

Conditions

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Age-Related Macular Degeneration Choroidal Neovascularization

Keywords

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Age-Related Macular Degeneration Rapamycin Remicade Daclizumab Immunosuppression Infliximab Choroidal Neovascularization Sirolimus AMD Ocular Inflammation

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Intravenous Daclizumab

Participants randomly assigned to intravenous (IV) daclizumab received 8 mg/kg of IV daclizumab at baseline, then 4 mg/kg of IV daclizumab at Week 2 and then 2 mg/kg of IV daclizumab monthly for the rest of the 6-month study.

Group Type ACTIVE_COMPARATOR

Intravenous Daclizumab

Intervention Type DRUG

Participants randomly assigned to intravenous (IV) daclizumab received 8 mg/kg of IV daclizumab at baseline, then 4 mg/kg of IV daclizumab at Week 2 and then 2 mg/kg of IV daclizumab monthly for the rest of the 6-month study.

Intravenous Infliximab

Participants randomized to IV infliximab received 3 mg/kg of IV infliximab monthly for 6 months.

Group Type ACTIVE_COMPARATOR

Intravenous Infliximab

Intervention Type DRUG

Participants randomized to IV infliximab received 3 mg/kg of IV infliximab monthly for 6 months.

Oral Rapamycin

Participants randomly assigned to rapamycin received 2 mg in capsule form every other day for 6 months.

Group Type ACTIVE_COMPARATOR

Oral Rapamycin

Intervention Type DRUG

Participants randomly assigned to rapamycin received 2 mg in capsule form every other day for 6 months.

Observation

Participants randomly assigned to the observation group were given injections of either bevacizumab (1.25 mg/0.05 mL or 2.5 mg/0.1 mL) or ranibizumab (0.5 mg) if they presented with recurrence of intraretinal or subretinal fluid as seen on Stratus Optical Coherence Tomography.

Group Type OTHER

Observation

Intervention Type OTHER

Participants randomly assigned to the observation group were given injections of either bevacizumab (1.25 mg/0.05 mL or 2.5 mg/0.1 mL) or ranibizumab (0.5 mg) if they presented with recurrence of intraretinal or subretinal fluid as seen on Stratus Optical Coherence Tomography.

Interventions

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Intravenous Daclizumab

Participants randomly assigned to intravenous (IV) daclizumab received 8 mg/kg of IV daclizumab at baseline, then 4 mg/kg of IV daclizumab at Week 2 and then 2 mg/kg of IV daclizumab monthly for the rest of the 6-month study.

Intervention Type DRUG

Intravenous Infliximab

Participants randomized to IV infliximab received 3 mg/kg of IV infliximab monthly for 6 months.

Intervention Type DRUG

Observation

Participants randomly assigned to the observation group were given injections of either bevacizumab (1.25 mg/0.05 mL or 2.5 mg/0.1 mL) or ranibizumab (0.5 mg) if they presented with recurrence of intraretinal or subretinal fluid as seen on Stratus Optical Coherence Tomography.

Intervention Type OTHER

Oral Rapamycin

Participants randomly assigned to rapamycin received 2 mg in capsule form every other day for 6 months.

Intervention Type DRUG

Other Intervention Names

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Zenapax Remicade Sirolimus

Eligibility Criteria

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Inclusion Criteria

1. Understand and sign the institutional review board (IRB)-approved informed consent document for the study.
2. Age greater than 55 years.
3. In the study eye, diagnosis of AMD defined by the presence of drusen larger than 63 microns.
4. Any anti-angiogenic therapy in the study eye within 7 days of beginning immunosuppressive therapy.
5. In the study eye, the participant's study eye vision is between 20/20 and 20/400.
6. In the study eye, the presence of CNV under the fovea determined by the investigators and defined as any one of the following fluorescein angiographic (FA) features:

1. Early stippled hyperfluorescence of flat retinal pigment epithelium (RPE)and little or mild leakage in the late frames of the fluorescein (occult).
2. Irregular elevation of the RPE that does not exhibit discrete or bright hyperfluorescence in the early transit phase of the angiogram. Stippled hyperfluorescence may be present. Late frames may show persistent fluorescein staining or leakage within a sensory retinal detachment overlying this area (occult).
3. Late-phase leakage of undetermined source with leakage at the level of the RPE in the late-phase frames of the angiogram in which the source of the late leakage cannot be determined from earlier-phase frames of the angiogram (occult).
4. A well-demarcated area of bright hyperfluorescence in the early phase of the angiogram with leakage through the mid- and late-phase frames which obscures the boundaries of the area (classic).
7. For all CNV lesions considered to have occult CNV with no classic CNV, one of the following criteria must be met:

1. A documented loss of visual acuity (5 or more letters of best-corrected visual acuity if both measurements are made using an Early Treatment for Diabetic Retinopathy Study (ETDRS) chart or, a doubling of the visual angle if Snellen acuities are available from either an outside referral center or within the participating center (e.g., 20/80 to 20/160) a doubling of the visual angle is required because of the measurement variability of Snellen acuities).

OR
2. Documented FA evidence of a 10% increase in the lesion greatest linear dimension over the 3 months prior to enrollment.

OR
3. Documented blood associated with CNV.
8. The greatest linear dimension of the entire lesion (classic CNV, occult CNV and any features that could obscure the identification of classic or occult CNV) has to be less than or equal to 5400 microns in greatest linear dimension on the retina as measured by the treating ophthalmologist.
9. Retinal photographs and angiography of sufficient quality, allowing assessment of the macular area according to standard clinical practice, can be obtained.
10. Women of childbearing potential must not be pregnant or lactating, must have a negative pregnancy test at screening and must be practicing an adequate method of birth control. Acceptable methods of birth control include intrauterine device (IUD); oral, dermal, implanted or injected contraceptives; tubal ligation; and barrier methods with spermicide.
11. Willingness to comply with the protocol.

Exclusion Criteria

1. CNV, in the study eye, associated with other ocular diseases such as pathologic myopia, ocular histoplasmosis or posterior uveitis, etc.
2. Presence of geographic atrophy under the fovea in the study eye.
3. Evidence of retinal angiomatous proliferation as suspected by the presence of intraretinal hemorrhage, intraretinal leakage, adjoining serous pigment epithelial detachment (PED) or the presence of a connecting retinal vessel.
4. The presence of a chorio-retinal anastomosis.
5. Decreased vision, in the study eye, due to retinal disease not attributable to CNV, such as nonexudative forms of AMD, geographic atrophy, inherited retinal dystrophy, uveitis or epiretinal membrane. Participants who have any additional ocular diseases that have irreversibly compromised or, during follow-up, could likely compromise the visual acuity (VA) of the study eye including amblyopia, anterior ischemic optic neuropathy, clinically significant diabetic macular edema, severe non-proliferative diabetic retinopathy, or proliferative diabetic retinopathy.
6. Decreased vision, in the study eye, due to significant media opacity such as corneal disease or cataract, or opacity precluding photography of the retina; a tear (rip) of the RPE; a vitelliform-like lesion of the outer retina (e.g., as in pattern dystrophies or basal laminar drusen), idiopathic parafoveal telangiectasis, or central serous retinopathy.
7. Presence of fibrosis, hemorrhage, pigment epithelial detachments and other hypofluorescent lesions obscuring greater than 50% of the CNV lesion.
8. History of other systemic antiangiogenic treatment or treatment for CNV (not including photodynamic therapy and pegaptanib sodium injections) in the study eye with transpupillary thermotherapy or other local treatment (such as submacular surgery). Previous laser photocoagulation therapy is acceptable, provided it was not subfoveal.
9. Participant with a known underlying systemic disease with evidence of serious or potentially lethal uncontrolled active disease in one or more extraocular organ systems for which a defined effective medical regimen is indicated.
10. Participant with a corneal melting, necrotizing keratitis, or impending vision loss.
11. Participants with history of allergy to/or exposure to mouse protein.
12. Participant with scleritis of infectious etiology.
13. Participant receiving any other investigational therapy or another anti-tumor necrosis factor (TNF) agent that would interfere with the ability to evaluate the safety or efficacy of infliximab.
14. Participant has significant active infection requiring hospitalization.
15. Participant with multiple sclerosis.
16. Participant has severe (class 3/4) congestive heart failure.
17. Participant has a history of cancer within the past 5 years other than basal or squamous cell carcinoma.
18. Participant is pregnant or lactating.
19. Participant with posterior scleritis.
20. Participant has evidence of liver disease (any etiology). History of moderate to severe abnormal liver function, unless documented evidence of normal liver enzymes is provided.
21. Participant has positive PPD (tuberculosis test) unless cleared by Internal Medicine.
22. Participant has positive Chest X-ray showing acute pulmonary disease.
23. Participant has unexplained hematuria.
24. Participant has a history of alkylating therapy use.
25. Current exam evidence of ocular toxoplasmosis; pseudoexfoliation; external ocular infection, including conjunctivitis; chalazion; significant blepharitis; or aphakia in the study eye (pseudophakic participants are eligible).
26. Intraocular surgery (including lens replacement surgery) within 6 weeks prior to randomization.
27. Recent history of (within the last 6 months), or current acute ocular or periocular infection (including any history of ocular herpes zoster or simplex).
28. Known hypersensitivity/allergy to verteporfin, porfimer sodium, or other porphyrins, porphyria or other porphyrin sensitivity, or hypersensitivity to sunlight or bright artificial light. Participation in any other clinical study or are receiving, or have received any experimental systemic treatment for AMD (e.g., retinoic acid, thalidomide). Local therapy for AMD is permitted.
29. Medical problems that make consistent follow-up over the treatment period unlikely (e.g., stroke, severe myocardial infarction (MI), end stage malignancy), any contraindications to performing the necessary diagnostic studies (i.e., known allergy to fluorescein dyes etc.), or in general a poor medical risk because of other systemic diseases or active uncontrolled infections.
30. Participant has a history of moderate to severe abnormal liver function, unless documented evidence of normal liver enzymes is provided.
31. Participant has a history of active pulmonary tuberculosis.
32. Participant has a history of active viral hepatitis.
33. Participant has chronic continued Ketoconazole use.
Minimum Eligible Age

55 Years

Maximum Eligible Age

92 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Eye Institute (NEI)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Robert Nussenblatt, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

National Eye Institute (NEI)

Locations

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National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, United States

Site Status

Countries

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United States

References

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Aeberli D, Oertle S, Mauron H, Reichenbach S, Jordi B, Villiger PM. Inhibition of the TNF-pathway: use of infliximab and etanercept as remission-inducing agents in cases of therapy-resistant chronic inflammatory disorders. Swiss Med Wkly. 2002 Jul 27;132(29-30):414-22. doi: 10.4414/smw.2002.10031.

Reference Type BACKGROUND
PMID: 12428187 (View on PubMed)

Antoni C, Dechant C, Hanns-Martin Lorenz PD, Wendler J, Ogilvie A, Lueftl M, Kalden-Nemeth D, Kalden JR, Manger B. Open-label study of infliximab treatment for psoriatic arthritis: clinical and magnetic resonance imaging measurements of reduction of inflammation. Arthritis Rheum. 2002 Oct 15;47(5):506-12. doi: 10.1002/art.10671.

Reference Type BACKGROUND
PMID: 12382299 (View on PubMed)

Bian ZM, Elner SG, Strieter RM, Kunkel SL, Lukacs NW, Elner VM. IL-4 potentiates IL-1beta- and TNF-alpha-stimulated IL-8 and MCP-1 protein production in human retinal pigment epithelial cells. Curr Eye Res. 1999 May;18(5):349-57. doi: 10.1076/ceyr.18.5.349.5353.

Reference Type BACKGROUND
PMID: 10372996 (View on PubMed)

Nussenblatt RB, Byrnes G, Sen HN, Yeh S, Faia L, Meyerle C, Wroblewski K, Li Z, Liu B, Chew E, Sherry PR, Friedman P, Gill F, Ferris F 3rd. A randomized pilot study of systemic immunosuppression in the treatment of age-related macular degeneration with choroidal neovascularization. Retina. 2010 Nov-Dec;30(10):1579-87. doi: 10.1097/IAE.0b013e3181e7978e.

Reference Type RESULT
PMID: 20847709 (View on PubMed)

Other Identifiers

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06-EI-0111

Identifier Type: OTHER

Identifier Source: secondary_id

060111

Identifier Type: -

Identifier Source: org_study_id