Trial Outcomes & Findings for IAI Versus Sham as Prophylaxis Against Conversion to Neovascular AMD (NCT NCT02462889)

NCT ID: NCT02462889

Last Updated: 2025-11-14

Results Overview

Percentage of subjects converting to exudative AMD at 24 months as confirmed by independent masked reading center.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

128 participants

Primary outcome timeframe

24 months

Results posted on

2025-11-14

Participant Flow

Of 128 patients enrolled, 127 (63 in the IAI group and 64 in the sham group) were included in the analysis. This is because shortly after enrollment of the excluded patient, it was determined that they should not have been enrolled due to not meeting eligibility criteria at screening.

Participant milestones

Participant milestones
Measure	Intravitreal aflibercept injection Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Overall Study STARTED	63	64
Overall Study COMPLETED	60	53
Overall Study NOT COMPLETED	3	11

Reasons for withdrawal

Reasons for withdrawal
Measure	Intravitreal aflibercept injection Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Overall Study Withdrawal by Subject	2	6
Overall Study Death	1	5

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intravitreal Aflibercept Injection n=63 Participants Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo n=64 Participants Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection	Total n=127 Participants Total of all reporting groups
Age, Continuous	76.1 years STANDARD_DEVIATION 7.4 • n=63 Participants	76.9 years STANDARD_DEVIATION 8.8 • n=64 Participants	76.5 years STANDARD_DEVIATION 8.1 • n=127 Participants
Sex: Female, Male Female	27 Participants n=63 Participants	32 Participants n=64 Participants	59 Participants n=127 Participants
Sex: Female, Male Male	36 Participants n=63 Participants	32 Participants n=64 Participants	68 Participants n=127 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Duration of Fellow Eye nvAMD < 2 Years	30 Participants n=63 Participants	31 Participants n=64 Participants	61 Participants n=127 Participants
Non-exudative Macular NV	4 Participants n=63 Participants	6 Participants n=64 Participants	10 Participants n=127 Participants
Mean BCVA	78.7 letters read on ETDRS eye chart STANDARD_DEVIATION 8.8 • n=63 Participants	77.3 letters read on ETDRS eye chart STANDARD_DEVIATION 10.1 • n=64 Participants	78.0 letters read on ETDRS eye chart STANDARD_DEVIATION 9.45 • n=127 Participants

PRIMARY outcome

Timeframe: 24 months

Percentage of subjects converting to exudative AMD at 24 months as confirmed by independent masked reading center.

Outcome measures

Outcome measures
Measure	Intravitreal aflibercept injection n=63 Participants Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo n=64 Participants Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Percentage of Subjects Converting to Exudative AMD at 24 Months.	6 Participants	7 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Patients with a history of exudative AMD in their fellow eye no longer than 2 years at Baseline by treatment group.

Percentage of patients converted to exudative AMD in study eye who had exudative AMD in fellow eye ≤ 2 years at Baseline.

Outcome measures

Outcome measures
Measure	Intravitreal aflibercept injection n=30 Participants Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo n=31 Participants Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Percentage of Patients Converted to Exudative AMD Who Had Exudative AMD in Fellow Eye ≤ 2 Years at Baseline.	4 Participants	5 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Number of subjects with nonexudative CNV present at Month 24 based on OCT angiography.

Percentage of patients converted to exudative AMD compared to all patients with presence of nonexudative CNV in the study eye at Month 24, as confirmed by independent reading center evaluation of OCT angiography images.

Outcome measures

Outcome measures
Measure	Intravitreal aflibercept injection n=11 Participants Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo n=13 Participants Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Percentage of Patient Converted to Exudative AMD Based on Presence of Nonexudative CNV.	3 Participants	4 Participants

SECONDARY outcome

Timeframe: 24 months

Mean change in best-corrected visual acuity (BCVA) at 24 months compared to baseline according to ETDRS protocol. BCVA is evaluated by counting the number of letters correctly read by the patient on an ETDRS eye chart. A higher value indicates that the patient read more letter correctly which indicates a better outcome.

Outcome measures

Outcome measures
Measure	Intravitreal aflibercept injection n=63 Participants Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo n=64 Participants Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Mean Change in Best-corrected Visual Acuity at 24 Months Compared to Baseline	2.8 number of letters read on eye chart Interval 0.7 to 6.64	0.2 number of letters read on eye chart Interval -4.39 to 2.52

SECONDARY outcome

Timeframe: 24 months

Mean change in growth of geographic atrophy from baseline to Month 24.

Outcome measures

Outcome measures
Measure	Intravitreal aflibercept injection n=63 Participants Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo n=64 Participants Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Mean Change in Growth of Geographic Atrophy.	0.34 millimeters squared Interval -0.07 to 0.36	0.43 millimeters squared Interval 0.11 to 0.96

Adverse Events

Intravitreal aflibercept injection

Serious events: 14 serious events

Other events: 9 other events

Deaths: 1 deaths

Placebo

Serious events: 27 serious events

Other events: 8 other events

Deaths: 5 deaths

Serious adverse events

Other adverse events

Additional Information

Jeffrey S. Heier, MD

Ophthalmic Consultants of Boston

Phone: 617-314-2694

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Intravitreal aflibercept injection n=63 participants at risk Subjects will be randomized to receive intravitreal aflibercept injection every three months for 24 months. Intravitreal aflibercept injection: Intravitreal aflibercept injection	Placebo n=64 participants at risk Subjects will be randomized to receive sham injection every three months for 24 months. Placebo: Sham injection
Musculoskeletal and connective tissue disorders Abdominal pain	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Musculoskeletal and connective tissue disorders Back spasm	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Eye disorders Decreased visual acuity x 6 lines	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Hepatobiliary disorders Elevated LFTs	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Gastrointestinal disorders Hematochezia	0.00% 0/63 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Musculoskeletal and connective tissue disorders Left cavernous sinus lesion	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Musculoskeletal and connective tissue disorders Leg pain	0.00% 0/63 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Reproductive system and breast disorders Lump in right breast	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Musculoskeletal and connective tissue disorders Meniscus tear, left	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Vascular disorders Nosebleed	0.00% 0/63 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Musculoskeletal and connective tissue disorders Spinal stenosis	0.00% 0/63 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Cardiac disorders Syncopal episode due to hypotension	0.00% 0/63 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Endocrine disorders Thyroid nodule, benign	0.00% 0/63 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Gastrointestinal disorders Vomiting	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Skin and subcutaneous tissue disorders Vulva intraepithelial neoplasia (VIN 3)	1.6% 1/63 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	0.00% 0/64 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.
Eye disorders Worsening of cataract	0.00% 0/63 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.	1.6% 1/64 • Number of events 1 • 2 years A SAE is any untoward medical occurrence that at any dose: * Results in death. * Is life threatening. * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is an important medical event. Events with severity score of Severe on 3-point scale of mild, moderate, or severe are reported.