NCT02634333

Brief Summary

Multiple studies have implicated vascular endothelial growth factor VEGF as a major causative factor in human eye diseases characterized by neovascularization including proliferative diabetic retinopathy (PDR) and vascular permeability including diabetic macular edema (DME). While there is strong evidence that PDR outcomes are markedly reduced in eyes that are treated with monthly anti-VEGF therapy (A Study of Ranibizumab Injection in Subjects With Clinically Significant Macular Edema (ME) With Center Involvement Secondary to Diabetes Mellitus: RIDE/RISE) and moderately reduced in eyes that received fairly frequent dosing during the 1st year of treatment (Diabetic Retinopathy Clinical Research Network protocol I), it is unknown whether or not an earlier but less frequent dosing regimen would result in similar, favorable anatomic outcomes, and whether favorable anatomic outcomes subsequently would result in favorable visual acuity outcomes. If this study demonstrates that intravitreous aflibercept treatment is effective and safe for reducing the onset of PDR or center involved- DME (CI-DME) in eyes that are at high risk for these complications, a new strategy to prevent vision threatening complications of diabetes will be available for patients. The application of intravitreous aflibercept earlier in the course of disease (i.e., at the time when an eye has baseline severe non-proliferative diabetic retinopathy) could help to reduce future potential treatment burden in patients, at the same time resulting in similar or better long-term visual outcomes, if PDR and DME are prevented. The primary objectives of this protocol are to 1) determine the efficacy and safety of intravitreous aflibercept injections versus sham injections (observation) for prevention of PDR or CI-DME in eyes at high risk for development of these complications and 2) compare long-term visual outcomes in eyes that receive anti-VEGF therapy early in the course of disease with those that are observed initially, and treated only if high-risk PDR or CI-DME with vision loss develops. Secondary objectives include:

  • Comparing other visual acuity outcomes between treatment groups, such as proportion of eyes with at least 10 or at least 15 letter loss from baseline, or gain or loss of at least 5 letters at the consecutive study visit just before and at the 2- or 4-year visit
  • Comparing optical coherence tomography (OCT) outcomes, such as mean change in OCT central subfield thickness and volume from baseline
  • Comparing proportion of eyes with at least 2 and 3-step worsening or improvement of diabetic retinopathy severity level (scale for individual eyes) by central reading center from baseline
  • Comparing associated treatment and follow-up exam costs between treatment groups
  • Comparing safety outcomes between treatment groups

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
399

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Jan 2016

Longer than P75 for phase_3

Geographic Reach
2 countries

85 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 11, 2015

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 18, 2015

Completed
14 days until next milestone

Study Start

First participant enrolled

January 1, 2016

Completed
4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2020

Completed
1.6 years until next milestone

Results Posted

Study results publicly available

November 24, 2021

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 11, 2022

Completed
Last Updated

June 18, 2023

Status Verified

May 1, 2023

Enrollment Period

4.3 years

First QC Date

December 11, 2015

Results QC Date

June 21, 2021

Last Update Submit

May 22, 2023

Conditions

Keywords

anti-vascular endothelial growth factornonproliferative diabetic retinopathy

Outcome Measures

Primary Outcomes (2)

  • Development of PDR and/or DME (Whichever Came First)

    CI-DME = center-involved diabetic macular edema, PDR = proliferative diabetic macular edema. First development of criteria meeting end point. Eyes that met any criteria are then censored from contributing to the next criteria. Eyes that did not meet the outcome were censored at the time of the last completed visit. Each outcome appears only once under "First PDR and/or DME criteria met." Outcomes appear under "Development of PDR" if PDR developed at any time in the study (regardless of if or when DME developed) and outcomes appear under "Development of DME" if DME developed at any time in the study (regardless of if or when PDR developed)

    2 years

  • Change in Visual Acuity From Baseline

    Visual acuity is measured as a continuous integer letter score from 0 to 100, with higher numbers indicating better visual acuity. A letter score of 85 is approximately 20/20 and a letter score of 70 is approximately 20/40, the legal unrestricted driving limit in most states. A 5-letter change for an individual is approximately equal to a 1-line change on a vision chart.

    2 years

Secondary Outcomes (2)

  • Change in Visual Acuity From Baseline

    4 years

  • Development of PDR and/or DME (Whichever Came First)

    4 years

Study Arms (2)

Observation (Prompt Sham)

SHAM COMPARATOR

Sham injection in study eye at randomization and at visits at 1, 2, and 4 months and then every 4 months thereafter. Deferred aflibercept may be given if center-involved diabetic macular edema or proliferative diabetic retinopathy develops and deferred laser may subsequently be added to intravitreal aflibercept if certain criteria are met.

Procedure: Prompt ShamProcedure: Deferred laserDrug: Deferred aflibercept

Prompt aflibercept

EXPERIMENTAL

Aflibercept injection in study eye at randomization and at visits at 1, 2, and 4 months and then every 4 months thereafter. More frequent aflibercept may be given if center-involved diabetic macular edema or proliferative diabetic retinopathy develops and deferred laser may subsequently be added to intravitreal aflibercept if certain criteria are met.

Drug: Prompt afliberceptProcedure: Deferred laserDrug: Deferred aflibercept

Interventions

Prompt ShamPROCEDURE

A sham injection (syringe without a needle pressed against the injection site) is performed on the day of randomization and visits at 1, 2, and 4 months and then every 4 months thereafter.

Observation (Prompt Sham)

Intravitreal injection of 2.0mg aflibercept is performed on the day of randomization and visits at 1, 2, and 4 months and then every 4 months thereafter.

Also known as: intravitreal anti-vascular endothelial growth factor, Eylea
Prompt aflibercept

Laser (either focal/grid laser for diabetic macular edema or panretinal photocoagulation for proliferative diabetic retinopathy) is added following initiation of anti-vascular endothelial growth factor injections for center-involved diabetic macular edema or proliferative diabetic retinopathy only if certain criteria are met

Also known as: focal/grid photocoagulation, panretinal photocoagulation
Observation (Prompt Sham)Prompt aflibercept

Intravitreal injection of 2.0mg aflibercept performed once proliferative diabetic retinopathy or center-involved diabetic macular edema develops and then up to every 4 weeks using defined treatment criteria.

Also known as: intravitreal anti-vascular endothelial growth factor, Eylea
Observation (Prompt Sham)Prompt aflibercept

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age \>= 18 years
  • Diagnosis of diabetes mellitus (type 1 or type 2)
  • Any one of the following will be considered to be sufficient evidence that diabetes is present:
  • Current regular use of insulin for the treatment of diabetes
  • Current regular use of oral anti-hyperglycemia agents for the treatment of diabetes
  • Documented diabetes by American Diabetes Association and/or World Health Organization criteria
  • Able and willing to provide informed consent.
  • Meets all of the following ocular criteria in at least one eye:
  • Best corrected Electronic-Early Treatment Diabetic Retinopathy Study (E-ETDRS) visual acuity letter score ≥79 (approximate Snellen equivalent 20/25 or better)
  • Severe NPDR is defined as:
  • All 4 midperipheral quadrants show severe hemorrhages or microaneurysms (at least as great as Standard photograph 2A, approximately 20 dot and blot hemorrhages), or
  • At least 2 fields of definite venous beading in the midperipheral quadrants or at least 1 field at least as severe as Standard photograph 6A, or
  • At least 1 field of moderate intraretinal microvascular abnormalities (IRMA) in the midperipheral quadrants, at least as severe as Standard photograph 8A
  • No evidence of neovascularization (NV) on fluorescein angiography within the 7-modified ETDRS fields, confirmed by the central Reading Center prior to randomization.
  • No center-involved diabetic macular edema (CI-DME) on clinical exam and optical coherence tomography (OCT) central subfield thickness must be below the following gender and OCT-machine specific thresholds:
  • +6 more criteria

You may not qualify if:

  • History of chronic renal failure requiring dialysis or kidney transplant.
  • A condition that, in the opinion of the investigator, would preclude participation in the study (e.g., unstable medical status including blood pressure, cardiovascular disease, and glycemic control).
  • Initiation of intensive insulin treatment (a pump or multiple daily injections) within 4 months prior to randomization or plans to do so in the next 4 months.
  • Participation in an investigational trial that involved treatment within 30 days of randomization with any drug that has not received regulatory approval for the indication being studied.
  • Note: study participants cannot participate in another investigational trial that involves treatment with an investigational drug while participating in the study.
  • Known allergy to any component of the study drug or any drug used in the injection prep (including povidone iodine prep).
  • Known allergy to fluorescein dye.
  • Blood pressure \> 180/110 (systolic above 180 or diastolic above 110). • If blood pressure is brought below 180/110 by anti-hypertensive treatment, individual can become eligible.
  • Systemic anti-VEGF or pro-VEGF treatment within 4 months prior to randomization.
  • These drugs should not be used during the study.
  • For women of child-bearing potential: pregnant or lactating or intending to become pregnant within the next 2 years.
  • Women who are potential study participants should be questioned about the potential for pregnancy. Investigator judgment is used to determine when a pregnancy test is needed.
  • Individual is expecting to move out of the area of the clinical center to an area not covered by another Diabetic Retinopathy Clinical Research Network certified clinical center during the next 2 years.
  • Individual has any of the following ocular characteristics in the eye(s) being evaluated:
  • Exam or photographic evidence of vitreous hemorrhage or preretinal hemorrhage presumed to be from PDR.
  • +11 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (85)

Arizona Retina and Vitreous Consultants

Phoenix, Arizona, 85021, United States

Location

University of Arizona Medical Center/Department of Ophthalmology

Tucson, Arizona, 85711, United States

Location

Atlantis Eye Care

Huntington Beach, California, 92647, United States

Location

Loma Linda University Health Care, Department of Ophthalmology

Loma Linda, California, 92354, United States

Location

East Bay Retina Consultants, Inc.

Oakland, California, 94609, United States

Location

Southern California Desert Retina Consultants, MC

Palm Desert, California, 92211, United States

Location

Shashi D Ganti, MD PC

Porterville, California, 93257, United States

Location

Retina Consultants of Southern California

Redlands, California, 92374, United States

Location

U.C. Davis Eye Center

Sacramento, California, 95817, United States

Location

California Retina Consultants

Santa Barbara, California, 93103, United States

Location

Retinal Consultants of Southern California Medical Group, Inc.

Westlake Village, California, 91361, United States

Location

New England Retina Associates

Hamden, Connecticut, 06518, United States

Location

Retina Group of Florida

Fort Lauderdale, Florida, 33308, United States

Location

National Ophthalmic Research Institute

Fort Myers, Florida, 33912, United States

Location

University of Florida College of Med., Department of Ophthalmology, Jacksonville Health Science Cent

Jacksonville, Florida, 32209, United States

Location

Florida Retina Institute-Jacksonville

Jacksonville, Florida, 32216, United States

Location

Florida Retina Consultants

Lakeland, Florida, 33805, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

Magruder Eye Institute

Orlando, Florida, 32803, United States

Location

Florida Retina Institute

Orlando, Florida, 32806, United States

Location

Southeast Eye Institute, P.A. dba Eye Associates of Pinellas

Pinellas Park, Florida, 33782, United States

Location

Fort Lauderdale Eye Institute

Plantation, Florida, 33324, United States

Location

Sarasota Retina Institute

Sarasota, Florida, 34239, United States

Location

Retina Associates of Florida, P.A.

Tampa, Florida, 33609, United States

Location

Emory Eye Center

Atlanta, Georgia, 30322, United States

Location

Southeast Retina Center, P.C.

Augusta, Georgia, 30909, United States

Location

Marietta Eye Clinic

Marietta, Georgia, 30060, United States

Location

Thomas Eye Group

Sandy Springs, Georgia, 30328, United States

Location

Gailey Eye Clinic

Bloomington, Illinois, 61704, United States

Location

Northwestern Medical Faculty Foundation

Chicago, Illinois, 60611, United States

Location

University of Illinois at Chicago Medical Center

Chicago, Illinois, 60612, United States

Location

Springfield Clinic, LLP

Springfield, Illinois, 62703, United States

Location

Raj K. Maturi, M.D., P.C.

Indianapolis, Indiana, 46290, United States

Location

Medical Associates Clinic, P.C.

Dubuque, Iowa, 52002, United States

Location

Wolfe Eye Clinic

West Des Moines, Iowa, 50266, United States

Location

Retina Associates, P.A.

Shawnee Mission, Kansas, 66204, United States

Location

Paducah Retinal Center

Paducah, Kentucky, 42001, United States

Location

Eye Associates of Northeast Louisiana dba Haik Humble Eye Center

West Monroe, Louisiana, 71291, United States

Location

Elman Retina Group, P.A.

Baltimore, Maryland, 21237, United States

Location

Wilmer Eye Institute at Johns Hopkins

Baltimore, Maryland, 21287, United States

Location

Mid Atlantic Retina Specialists

Hagerstown, Maryland, 21740, United States

Location

Valley Eye Physicians and Surgeons

Ayer, Massachusetts, 01432, United States

Location

Joslin Diabetes Center

Boston, Massachusetts, 02215, United States

Location

Henry Ford Health System, Dept of Ophthalmology and Eye Care Services

Detroit, Michigan, 48202, United States

Location

Vitreo-Retinal Associates

Grand Rapids, Michigan, 49546, United States

Location

Retina Center, PA

Minneapolis, Minnesota, 55404, United States

Location

Mid-America Retina Consultants, P.A.

Kansas City, Missouri, 64111, United States

Location

Retinal and Ophthalmic Consultants, PC

Northfield, New Jersey, 08225, United States

Location

Eye Associates of New Mexico

Albuquerque, New Mexico, 87109, United States

Location

The New York Eye and Ear Infirmary/Faculty Eye Practice

New York, New York, 10003, United States

Location

MaculaCare

New York, New York, 10021, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

Retina-Vitreous Surgeons of Central New York, PC

Syracuse, New York, 13224, United States

Location

Western Carolina Clinical Research, LLC

Asheville, North Carolina, 28803, United States

Location

Kittner Eye Center

Chapel Hill, North Carolina, 27517, United States

Location

Charlotte Eye, Ear, Nose and Throat Assoc., PA

Charlotte, North Carolina, 28210, United States

Location

Retina Associates of Cleveland, Inc.

Beachwood, Ohio, 44122, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Oregon Retina, LLP

Eugene, Oregon, 97401, United States

Location

Retina Northwest, PC

Portland, Oregon, 97210, United States

Location

Casey Eye Institute

Portland, Oregon, 97239, United States

Location

Retina Vitreous Consultants

Monroeville, Pennsylvania, 15146, United States

Location

University of Pennsylvania Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

Location

Carolina Retina Center

Columbia, South Carolina, 29223, United States

Location

Palmetto Retina Center

West Columbia, South Carolina, 29169, United States

Location

Southeastern Retina Associates

Chattanooga, Tennessee, 37421, United States

Location

Southeastern Retina Associates, P.C.

Knoxville, Tennessee, 37909, United States

Location

Southwest Retina Specialists

Amarillo, Texas, 79106, United States

Location

Austin Retina Associates

Austin, Texas, 78705, United States

Location

Retina Research Center

Austin, Texas, 78705, United States

Location

Robert E. Torti, MD, PA dba Retina Specialists

DeSoto, Texas, 75115, United States

Location

Retina Center of Texas

Grapevine, Texas, 76051, United States

Location

Retina and Vitreous of Texas

Houston, Texas, 77025, United States

Location

Baylor Eye Physicians and Surgeons

Houston, Texas, 77030, United States

Location

Retina Consultants of Houston, PA

Houston, Texas, 77030, United States

Location

Texas Retina Associates

Lubbock, Texas, 79424, United States

Location

Valley Retina Institute

McAllen, Texas, 78503, United States

Location

Retinal Consultants of San Antonio

San Antonio, Texas, 78240, United States

Location

Retina Institute of Virginia

Richmond, Virginia, 23235, United States

Location

Virginia Commonwealth University, Dept. of Ophthalmology

Richmond, Virginia, 23298, United States

Location

University of Wisconsin-Madison, Dept of Ophthalmology/Retina Service

Madison, Wisconsin, 53705, United States

Location

UBC/VCHA Eye Care Centre

Vancouver, British Columbia, V5Z 3N9, Canada

Location

Nova Scotia District Health Authority

Halifax, Nova Scotia, B3H 2Y9, Canada

Location

Toronto Retina Institute (TRI)

North York, Ontario, M3C 0G9, Canada

Location

University Health Network - Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

Location

Related Publications (2)

  • Maturi RK, Glassman AR, Josic K, Baker CW, Gerstenblith AT, Jampol LM, Meleth A, Martin DF, Melia M, Punjabi OS, Rofagha S, Salehi-Had H, Stockdale CR, Sun JK; DRCR Retina Network. Four-Year Visual Outcomes in the Protocol W Randomized Trial of Intravitreous Aflibercept for Prevention of Vision-Threatening Complications of Diabetic Retinopathy. JAMA. 2023 Feb 7;329(5):376-385. doi: 10.1001/jama.2022.25029.

  • Maturi RK, Glassman AR, Josic K, Antoszyk AN, Blodi BA, Jampol LM, Marcus DM, Martin DF, Melia M, Salehi-Had H, Stockdale CR, Punjabi OS, Sun JK; DRCR Retina Network. Effect of Intravitreous Anti-Vascular Endothelial Growth Factor vs Sham Treatment for Prevention of Vision-Threatening Complications of Diabetic Retinopathy: The Protocol W Randomized Clinical Trial. JAMA Ophthalmol. 2021 Jul 1;139(7):701-712. doi: 10.1001/jamaophthalmol.2021.0606.

MeSH Terms

Conditions

Diabetic Retinopathy

Interventions

aflibercept

Condition Hierarchy (Ancestors)

Retinal DiseasesEye DiseasesDiabetic AngiopathiesVascular DiseasesCardiovascular DiseasesDiabetes ComplicationsDiabetes MellitusEndocrine System Diseases

Results Point of Contact

Title
Adam Glassman
Organization
JAEB CENTER FOR HEALTH RESEARCH

Study Officials

  • Jennifer K. Sun, MD, MPH

    Joslin Diabetes Center

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, OUTCOMES ASSESSOR
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 11, 2015

First Posted

December 18, 2015

Study Start

January 1, 2016

Primary Completion

May 1, 2020

Study Completion

May 11, 2022

Last Updated

June 18, 2023

Results First Posted

November 24, 2021

Record last verified: 2023-05

Locations