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Ranibizumab

Phase 3

Age-related Macular Degeneration | Small molecule | Ophthalmology |Novartis AG|Last Updated: Feb 13, 2023

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLED
Total Trials1
Total Enrollment650

FDA Designations

No designations recorded

Clinical trial landscape

Ranibizumab · 36 trials · 28 indications

Phase 3 28Phase 2 7Phase 1 1
NCT02640664Rainbow Extension StudyRetinopathy of Prematurity (ROP)
COMPLETED180 Analytics
NCT02375971RAINBOW Study: RAnibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of PrematurityRetinopathy of Prematurity
COMPLETED224 Analytics
NCT02259088A 12-month, Randomized, Efficacy and Safety Study of 0.5 mg Ranibizumab vs Laser in Chinese Diabetic Macular Edema (DME) PatientsDiabetic Macular Edema
COMPLETED384 Analytics
NCT02034006A Study of the Criteria Establishing the Need for Re-treatment With Ranibizumab Upon Relapse in Patients With Visual Impairment Due to Choroidal Neovascularization Secondary to Pathologic Myopia.Choroidal Neovascularization Secondary to Pathologic Myopia
COMPLETED200 Analytics
NCT01948830Randomized Study for Efficacy and Safety of Ranibizumab 0.5mg in Treat-extend and Monthly Regimens in Patients With nAMDAge-related Macular Degeneration
COMPLETED650 Analytics
NCT01976338Ranibizumab Intravitreal Injections Versus Sham Control in Patients With Branch Retinal Vein Occlusion (BRVO)Macular Edema Secondary to Branch Retinal Vein Occlusion
COMPLETED283 Analytics
NCT01976312Ranibizumab Intravitreal Injections Versus Sham Control in Patients With Central Retinal Vein Occlusion (CRVO)Central Retinal Vein Occlusion
COMPLETED252 Analytics
NCT01846299To Assess the Efficacy and Safety of Intravitreal Ranibizumab in People With Vision Loss Due to Macular EdemaMacular Edema (ME)
COMPLETED181 Analytics
NCT01908816An Open-label Extended Clinical Protocol of Ranibizumab to Evaluate Safety and Efficacy in Rare VEGF Driven Ocular Diseases.Choroidal Neovascularization
COMPLETED270 Analytics
NCT01922102Efficacy and Safety of Ranibizumab 0.5 vs Verteporfin PDT in Patients With Visual Impairment Due to Choroidal Neovascularization Secondary to Pathologic MyopiaVisual Impairment Due to Choroidal Neovascularization (CNV) Secondary to Pathologic Myopia (PM)
COMPLETED457 Analytics
PHASE3COMPLETED
Rainbow Extension Study
Retinopathy of Prematurity (ROP)Unlock trial analytics
PHASE3COMPLETED
RAINBOW Study: RAnibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of Prematurity
Retinopathy of PrematurityUnlock trial analytics
PHASE3COMPLETED
A 12-month, Randomized, Efficacy and Safety Study of 0.5 mg Ranibizumab vs Laser in Chinese Diabetic Macular Edema (DME) Patients
Diabetic Macular EdemaUnlock trial analytics
PHASE3COMPLETED
A Study of the Criteria Establishing the Need for Re-treatment With Ranibizumab Upon Relapse in Patients With Visual Impairment Due to Choroidal Neovascularization Secondary to Pathologic Myopia.
Choroidal Neovascularization Secondary to Pathologic MyopiaUnlock trial analytics
PHASE3COMPLETED
Randomized Study for Efficacy and Safety of Ranibizumab 0.5mg in Treat-extend and Monthly Regimens in Patients With nAMD
Age-related Macular DegenerationUnlock trial analytics
PHASE3COMPLETED
Ranibizumab Intravitreal Injections Versus Sham Control in Patients With Branch Retinal Vein Occlusion (BRVO)
Macular Edema Secondary to Branch Retinal Vein OcclusionUnlock trial analytics
PHASE3COMPLETED
Ranibizumab Intravitreal Injections Versus Sham Control in Patients With Central Retinal Vein Occlusion (CRVO)
Central Retinal Vein OcclusionUnlock trial analytics
PHASE3COMPLETED
To Assess the Efficacy and Safety of Intravitreal Ranibizumab in People With Vision Loss Due to Macular Edema
Macular Edema (ME)Unlock trial analytics
PHASE3COMPLETED
An Open-label Extended Clinical Protocol of Ranibizumab to Evaluate Safety and Efficacy in Rare VEGF Driven Ocular Diseases.
Choroidal NeovascularizationUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Ranibizumab 0.5 vs Verteporfin PDT in Patients With Visual Impairment Due to Choroidal Neovascularization Secondary to Pathologic Myopia
Visual Impairment Due to Choroidal Neovascularization (CNV) Secondary to Pathologic Myopia (PM)Unlock trial analytics

Study Endpoints

Primary Endpoints

Visual Acuity (VA) of the Better-seeing Eye at the Participant's Fifth Birthday Visit - Comparison Between Treatment Arms
at the participant's fifth birthday visit (maximum 5 years and 4 months post core baseline visit)

The VA assessment at the child's 5th birthday visit was performed using Early Treatment Diabetic Retinopathy Study (ETDRS) methodology. VA measurements were taken in a sitting position at an initial test distance of 3 meters using Lea Symbols charts. Scores represented the number of optotypes (Lea symbols) the participant identified and ranged from 0 to 100, with higher scores indicating better visual acuity. VA was tested in each eye, using the child's current refractive index. The better-seeing eye was defined as the eye with the higher ETDRS score at the 5th birthday visit. If both eyes had the same ETDRS score, then the right eye was assigned as the better-seeing eye.

Percentage of Participants With Absence of Active ROP and Absence of Unfavorable Structural Outcomes in Both Eyes at Week 24
Week 24

To achieve this outcome, patients must fulfill all the following criteria, 1) survival, 2) no intervention with a second modality for ROP, 3) absence of active ROP and 4) absence of unfavorable structural outcome. Retinopathy of prematurity (ROP) is a pathologic process that occurs in the incompletely vascularized, developing retina of low birth-weight preterm neonates.

Mean Average Change From Baseline in Best-Corrected Visual Acuity (BCVA) (Letters) to Month 1 Through 12
Baseline, Monthly from Month 1 through Month 12

Visual acuity with eyeglasses was assessed in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like charts at an initial testing distance of 4 meters. A letter score was calculated based on the number of letters correctly identified at the specified distance. Twelve monthly BCVA values were averaged \[(Month1+Month2+...+Month12)/12\], and the baseline BCVA value was subtracted from the average. A positive change value indicates an improvement in visual acuity, while a negative change value indicates a worsening. One eye (study eye) contributed to the analysis.

Number of Patients Treated and Re-treated Based on Presence/Absence of Active Leakage
Screening, Month 2, Month 6

Presence of active leakage on fluorescein angiography (FAG) was assessed at screening (14 to 3 days before baseline visit), month 2 and month 6. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of presence of active leakage (Yes/No). For retreated patients, the presence/absence of active leakage was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis. \*NE = Not evaluable

Number of Patients Treated and Re-treated Based on Presence/Absence of Macular Edema
Screening, Month 2, Month 6, Month 12

Presence of macular edema from optical coherence tomography (OCT) was assessed at screening (14 to 3 days before baseline visit), month 2, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of presence of macular edema (Yes/No). For retreated patients, the presence/absence of macular edema was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis. \*NE = Not evaluable

Number of Patients Treated and Re-treated Based on Presence/Absence of Cysts
Screening, Month 2, Month 6, Month 12

Presence of cysts from optical coherence tomography (OCT) was assessed at screening (14 to 3 days before baseline visit), month 2, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of presence of cysts (Yes/No). For retreated patients, the presence/absence of cysts was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis. \*NE = Not evaluable

Number of Patients Treated and Re-treated Based on Presence/Absence of Intra-retinal Fluid
Screening, Month 2, Month 6, Month 12

Presence of Intra-retinal fluid from optical coherence tomography (OCT) was assessed at screening (14 to 3 days before baseline visit), month 2, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of presence of Intra-retinal fluid (Yes/No). For retreated patients, the presence/absence of Intra-retinal fluid was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis. \*NE = Not evaluable

Change in Central Subfield Thickness (CSFT)
Screening, Month 2, Month 6, Month 12

Central subfield thickness (CSFT) from optical coherence tomography (OCT) was assessed at screening (14 to 3 days before baseline visit), month 2, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of change in CSFT versus previous visit. For retreated patients, the change in CSFT was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis.

Change in Central Subfield Volume (CSV)
Screening, Month 2, Month 6, Month 12

Central subfield volume (CSV) from optical coherence tomography (OCT) was assessed at screening (14 to 3 days before baseline visit), month 2, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of change in CSV versus previous visit. For retreated patients, the change in CSV was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis.

Number of Patients Treated and Re-treated Based on Presence/Absence of Sub-retinal Fluid
Screening, Month 2, Month 6, Month 12

Presence of sub-retinal fluid from optical coherence tomography (OCT) was assessed at screening (14 to 3 days before baseline visit), month 2, month 6 and month 12. The regression model for sub-retinal fluid was not valid because "Yes" was reported in almost all subjects causing a quasi-complete separation of data points. \*NE = Not evaluable

Number of Patients Treated and Re-treated Based on Presence/Absence of Clinically Significant Abnormalities
Baseline, Month 1, Month 2, Month 3, Month 6, Month 12

Presence of clinically significant abnormalities was assessed at baseline, month 1, month 2, month 3, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of presence of clinically significant abnormalities (Yes/No). For retreated patients, the presence/absence of clinically significant abnormalities was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis.

Number of Patients Treated and Re-treated Based on Improvement in Best Corrective Visual Acuity (BCVA) < 5 Letters
Baseline, Month 1, Month 2, Month 3, Month 6, Month 12

Improvement in BCVA \< 5 letters (Yes/No) was assessed at month1, month 2, month 3, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of improvement in BCVA \< 5 letters (Yes/No) which was reported as Gain \>= 5 letters versus Gain \< 5 letters. For retreated patients, Gain \>= 5 letters and Gain \< 5 letters were considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis.

Number of Patients Treated and Re-treated Based on Improvement in Best Corrective Visual Acuity (BCVA) < 10 Letters
Baseline, Month 1, Month 2, Month 3, Month 6, Month 12

Improvement in BCVA \< 10 letters (Yes/No) was assessed at month1, month 2, month 3, month 6 and month 12. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of improvement in BCVA \< 10 letters (Yes/No) which was reported as Gain \>= 10 letters versus Gain \< 10 letters. For retreated patients, Gain \>= 10 letters and Gain \< 10 letters were considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis.

Number of Patients in Different Categories of Changes From Baseline in BCVA
Baseline, Month 1, Month 2, Month 3, Month 6, Month 12

Changes from baseline in BCVA are described for the ETDRS parameter considering the following categories at each assessment: "no change" if the change was equal to 0 letter, "worsening" if change \< 0 letter , "improvement" if change \> 0 letter. A univariate logistic regression model was applied expressing the presence/absence of the first retreatment in function of change from baseline in BCVA (improved/worsened/stable) which was reported as Improved versus no change and worsened versus no change. For retreated patients, this variable was considered at the closest time-point to the first re-treatment: the last scheduled assessment immediately before the first re-treatment was considered. For treated patients, the last scheduled assessment available was considered. In case of missing value on the scheduled assessment, the value was considered as missing for this analysis.

Change in Best Corrected Visual Acuity (BCVA) From Baseline to Month 12
Baseline to month 12

Best-Corrected Visual Acuity (BCVA) letters was measured using Early Treatment Diabetic Retinopathy Study (ETDRS)-like charts while participants were in a sitting position at a testing distance of 4 meters. The range of ETDRS is 0 to 100 letters. A positive average change from baseline of BCVA indicates improvement

Average Change in Best Corrected Visual Acuity (BCVA) From Baseline to Month 1 Through Month 6
Baseline to Month 1 through Month 6

Best Corrected Visual Acuity (BCVA) was assessed in a sitting position using ETDRS-like visual acuity testing charts at an initial testing distance of 4 meters. Mean Visual Acuity was averaged over all monthly assessments from month 1 to month 6 and compared to Baseline.

Average Change in Visual Acuity (Letters) From Baseline to Month 1 Through Month 3
Baseline, 3 Months

Best Corrected Visual Acuity (BCVA) was assessed in a sitting position using ETDRS-like visual acuity testing charts at an initial testing distance of 4 meters. Mean Visual Acuity was averaged over all monthly assessments from month 1 to month 3 and compared to Baseline.

Change From Baseline in Best-corrected Visual Acuity (BCVA) in Study Eye
Baseline, Month 2

BCVA was assessed in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like visual acuity (VA) testing charts at an initial testing distance of 4 meters. A positive change from baseline indicated improvement.

Number of Participants With Adverse Events as a Measure of Safety and Tolerability
24 months

Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs) for ocular and non-ocular events. Due to early termination, only descriptive analysis was conducted.

Change From Baseline BCVA to the Average Level of BCVA Over All Monthly Assessments From Month 1 to Month 3
From Baseline to Month 3

Best corrected visual acuity (BCVA) was tested using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity (VA) charts testing protocol. VA measurements were taken in a sitting position at an initial test distance of 4 meters using ETDRS charts. The overall BCVA score was calculated using the BCVA worksheet, which was kept in the source data and the score was recorded in the eCRF.

Change From Baseline in Best-corrected Visual Acuity (BCVA) in Study Eye to Month 2
Baseline, Month 2

BCVA was assessed in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like visual acuity (VA) testing charts at an initial testing distance of 4 meters. The data were analyzed using mixed model repeated measures (MMRM) which contained scheduled visit, the type of underlying pathophysiologic mechanism (angloid streaks versus others) and treatment group as fixed effect factors, centered baseline BCVA as a continuous covariate and treatment group by visit and visit by centered baseline BCVA interactions. A positive change from baseline indicated improvement.

Average Best-corrected Visual Acuity (BCVA) (Letters) Change up to Month 12
up to Month 12

Visual acuity (VA) was assessed during every study visit using best correction determined from protocol refraction. VA measurements (number of letters correctly identified) were performed with the patient in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like VA testing charts at a testing distance of 4 meters. This outcome measure describes the difference between the Visual Acuity averaged up to Month 12 Level of VA (Letters) of the Study Eye.

Mean Change in Visual Acuity: BCVA Change at Month 6 Compared to Baseline in Patients With Visual Impairment Due to Branch Retinal Vein Occlusion (BRVO)
Baseline, 6 Months

Best-Corrected Visual Acuity (BCVA) letters was measured using Early Treatment Diabetic Retinopathy Study (ETDRS) -like chart while participants were in a sitting position at a testing distance of 4 meters. The range of ETDRS is 0 to 100 letters. For the mean change of best corrected visual acuity at Month 6 compare to Baseline, the 95% confidence interval and P value (related to the null hypothesis that this mean change is equal to zero) based on a t distribution/t test were calculated and assessed by an ANOVA model.

Mean Change in Best Corrected Visual Acuity (BCVA) at Month 12 Compared to Baseline
Baseline to month 12

Best-Corrected Visual Acuity (BCVA) letters was measured using Early Treatment Diabetic Retinopathy Study (ETDRS)-like chart while participants were in a sitting position at a testing distance of 4 meters. The range of ETDRS is 0 to 100 letters. A positive average change from baseline of BCVA indicates improvement

Mean Average BCVA Change From Month 1 Through Month 6 to Baseline
Baseline, month 6

the average of the changes in BCVA (letters) from baseline to any post-baseline visit, i.e. the mean of six differences to baseline for the six post-baseline visits at month 1 to 6. BCVA score was based on the number of letters read correctly on the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart assessed at a starting distance of 4 meters. An ETDRS visual acuity score of 85 is pproximately 20/20. An increased score indicates improvement in acuity

Mean Average Best Corrected Visual Acuity (BCVA) Change From Month 1 Through Month 6 to Baseline
Baseline, month 6

the average of the changes in BCVA (letters) from baseline to any post-baseline visit, i.e. the mean of six differences to baseline for the six post-baseline visits at month 1 to 6

Measure: Efficacy of 3 consecutive monthly intravitreal injections with 0.5 mg ranibizumab as assessed by the mean average change in best-corrected visual acuity (BCVA) from Month 1 through Month 3 compared to baseline
3 months
Average Change From Baseline to Month 1 Through Month 3 on Visual Acuity of the Study Eye
Baseline, Month 1 through Month 3

The Best Corrected Visual Acuity (BCVA) was tested using the Early Treatment Diabetic Retinopathy Study (ETDRS) Visual Acuity (VA) testing protocol. VA measurements were taken in a sitting position at an initial test distance of 4 meters using ETDRS charts at baseline and compared to the average from month 1 to month 3.

Visual Acuity of the Study Eye: Average Change From Baseline to Month 1 Through Month 12
Baseline to Month 12

Visual acuity was assessed at every study visit for the study eye using best correction determined from protocol refraction. The BCVA measurements were taken in a sitting position using ETDRS-like VA testing charts at a starting distance of 4 meters.

Mean Change From Baseline in Best Corrected Visual Acuity- (BCVA) at Month 12
Baseline and 12 months

Best-Corrected Visual Acuity (BCVA) letters was measured using Early Treatment Diabetic Retinopathy Study (EDTRS)-like chart while participants were in a sitting position at a testing distance of 4 meters. The range of BCVA (EDTRS) is 0 to 100 letters. A positive change from baseline of BCVA indicates improvement.

Average Change From Baseline of Best-Corrected Visual Acuity (BCVA) Over 12 Months (From Month 1 to Month 12 Compared to Baseline)
12 months

Best-Corrected Visual Acuity (BCVA) letters was measured using Early Treatment Diabetic Retinopathy Study (EDTRS)-like chart while participants were in a sitting position at a testing distance of 4 meters. The range of EDTRS is 0 to 100 letters. A positive average change from baseline of BCVA indicates improvement.

To evaluate efficacy of ranibizumab 0.5 mg by mean change in best-corrected visual acuity (BCVA) from Baseline to Month 4 as assessed with early treatment of diabetic retinopathy study (ETDRS) like charts at a distance of 4 meters.
12 months
The change in ETDRS visual acuity letter scores from baseline. The change in the 1-mm central retinal thickness as measured by OCT from baseline.
every 4 weeks (up to 52 weeks)
Core Study: Difference Between the Baseline Level of Visual Acuity (Letters) of the Study Eye and the Mean Visual Acuity Averaged Over All Monthly Post-baseline Assessments From Month 1 to Month 12
Baseline through the end of study (Month 12)

Visual acuity (VA) was assessed on both eyes during every study visit using best correction determined from protocol refraction. VA measurements (number of letters correctly identified) were performed with the patient in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like visual acuity testing charts at a testing distance of 4 meters.

Extension Study: Percentage of Participants With Ocular Adverse Events (AEs) in the Study Eye in the 24 Month Extension Study
Extension baseline (Month 12 -end of core study) to Month 36 (end of extension study) [24 Months]

Participants with ocular (occurring in the eye) serious adverse events (SAEs) and non-serious AEs in the study eye. The study eye is the eye that received the treatment. AEs are the appearance or worsening of any undesirable sign, symptom, or medical condition occurring after starting the study drug even if the event is not considered to be related to study drug. A serious adverse event is defined as an event that is fatal or life-threatening, results in persistent or significant disability/incapacity, constitutes a congenital anomaly/birth defect, requires inpatient hospitalization or prolongation of existing hospitalization or is medically significant. Additional information about adverse events can be found in the Adverse Event section.

Extension Study: Percentage of Participants With Non-Ocular Adverse Events (AEs) in the 24 Month Extension Study
Extension baseline (Month 12 -end of core study) to Month 36 (end of extension study) [24 Months]

Participants with non-ocular (not occurring in the eye) serious adverse events (SAEs) and non-serious AEs. AEs are the appearance or worsening of of any undesirable sign, symptom, or medical condition occurring after starting the study drug even if the event is not considered to be related to study drug. A serious adverse event is defined as an event that is fatal or life-threatening, results in persistent or significant disability/incapacity, constitutes a congenital anomaly/birth defect, requires inpatient hospitalization or prolongation of existing hospitalization or is medically significant. Additional information about adverse events can be found in the Adverse Event section.

Mean change in best-corrected visual acuity (BCVA) from Baseline, Month 4 and Month 12 as assessed with ETDRS (Early Treatment for Diabetic Retinopathy Study)-like charts at a distance of four meters
Baseline, Month 4 and Month 12
The primary efficacy outcome measure is the mean change from baseline in BCVA score
6 months
The change in ETDRS visual acuity letter scores from baseline.
every 4 weeks (up to 52 weeks)
Percentage of Patients With Ocular Adverse Events (AEs) in the Study Eye
Baseline through end of study (12 month treatment period)

Percentage of patients with ocular adverse events in the study eye over the one year (12 month) treatment period.

Percentage of Patients With Targeted Grade 3 Adverse Events (AEs) in the Study Eye
Baseline through end of study (12 month treatment period)

Grade 3 targeted AEs included: * 4+ ocular inflammation or 2-3+ ocular inflammation failing to decrease to ≤ 1+ within 30 days * ≥ 30 letter decrease in BCVA that developed within 14 days of ranibizumab injection * sustained (\>15 minutes) loss of light perception due to elevated intraocular pressure (IOP) or a \>20 mm Hg change in IOP persisting longer than 14 days * new retinal tear or detachment involving the macula * new vitreous hemorrhage \>2+ severity not resolving within 14 days * new or increase of previous retinal hemorrhage \>1 disc area in size and involving the fovea

To investigate whether iL given monthly for 6 months in patients with Predominantly PED lesions (secondary to AMD) will significantly improve mean visual acuity from baseline on a standardized ETDRS chart.
6 months
Adverse Events
12 months

Percentage of participants with ocular adverse events and other adverse events as identified by eye examination, physical examination, subject reporting and changes in vital signs one year post-operatively.

Change From Baseline in Area of Neovascularizations (NVs) at End of Core Study (EOCS)
Baseline, EOCS

The area of neovascularizations (NV) was assessed by a central reading center via fluorescein angiography (FA) images. The area of NV was calculated as the sum of area of neovascularization of the disc (NVD) and neovascularization elsewhere (NVE) and was recorded in square millimeters. A higher positive change value may indicate a greater formation of new, abnormal blood vessels and thus disease progression. One eye (study eye) contributed to the analysis.

The number of patients in whom, by consensus, no further treatment is required.
Data will be collected at every patient visit which will take place every 4-5 weeks, and analysed at 12 months follow-up

Intravitreal Ranibizumab will be given at baseline, month 1 and month 2 . Subsequent 4-5 weekly injections will be given according to clinical need. There will be a total of 12 months of follow-up.

Change in CRT as measured by Spectralis spectral domain OCT.
at baseline visit then at 6 and 12 months.
The difference from baseline in mean Best Corrected Visual Acuity (BCVA)
12 months
Difference Between the Baseline Level of Visual Acuity (Letters) of the Study Eye and the Mean Visual Acuity Averaged Over All Monthly Post-baseline Assessments From Month 1 to Month 12
Baseline through the end of study (Month 12)

Visual acuity (VA) was assessed on both eyes during every study visit using best correction determined from protocol refraction. VA measurements were performed with the patient in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like visual acuity testing charts at a testing distance of 4 meters as described in the Study Operations Manual.

Mean Change From Baseline in Visual Acuity (Letters) of the Study Eye at Month 12
Baseline through the end of study (Month 12)

Visual acuity (VA) was assessed on both eyes during every study visit using best correction determined from protocol refraction. VA measurements were performed with the patient in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like visual acuity testing charts at a testing distance of 4 meters as described in the Study Operations Manual.

Mean Change From Baseline in the Best Corrected Visual Acuity Score of the Study Eye at Month 6 in Group B
Baseline and Month 6

The efficacy assessment was based on Group B patients. Best Corrected Visual Acuity (BCVA) was assessed during all study visits using best correction determined from protocol refraction at a starting test distance of 2 meters. VA measurements were taken in a sitting position using Early Treatment Diabetic Retinopathy Study (ETDRS)-like visual acuity testing charts at a starting test distance of 2 meters. The BCVA score is the number of letters read correctly by the patient, hence an increase in score indicates improvement in acuity.

Secondary Endpoints

Number of Participants With Ocular Adverse Events (AEs) Regardless of Study Treatment or Procedure Relationship by Preferred Term
throughout the study, approximately 5 years
Number of Participants With Non-ocular Adverse Events (AEs) Regardless of Study Treatment or Procedure Relationship (Greater Than or Equal to 3% in Any Arm) by Preferred Term
throughout the study, approximately 5 years
Visual Acuity (VA) of the Worse-seeing Eye at the Participant's Fifth Birthday Visit - Comparison Between Treatment Arms
at the participant's fifth birthday visit (maximum 5 years and 4 months post core baseline visit)
Unlock Study Endpoints

Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Ranibizumab 0.2 mgEXPERIMENTAL1 intravitreal injection in both eyes on Day 1 (Baseline), with up to 2 re-treatments allowed for each eye if required
Ranibizumab 0.1 mgEXPERIMENTAL1 intravitreal injection in both eyes on Day 1 (Baseline), with up to 2 re-treatments allowed for each eye if required
Laser therapyNO_INTERVENTIONLaser therapy
Ranibizumab (RFB002)EXPERIMENTAL3 initial intravitreal injections to the study eye at Day 1, Month 1 and Month 2, followed by as-needed intravitreal injections of ranibizumab 0.5 mg guided by VA stabilization, plus sham laser
LaserACTIVE_COMPARATORLaser photocoagulation applied to the study eye at Day 1, followed by active laser photocoagulation at intervals no shorter than 3 months apart, plus sham injections
RanibizumabEXPERIMENTALPatients treated with a single ranibizumab 0.5 mg/0.05ml intravitreal injection
Group I ranibizumab 0.5 mg monthlyACTIVE_COMPARATORRanibizumab 0.5 mg/0.05 mL (Monthly regimen) up to month 11
Group II ranibizumab 0.5 mg TERACTIVE_COMPARATORRanibizumab 0.5 mg/0.05 mL (TER) treat and Extend regimen up to month 11
Ranibizumab 0.5 mgEXPERIMENTALPRN Intravitreal injection
Sham injectionSHAM_COMPARATORAs of Month 6 ranibizumab 0.5 mg PRN intravitreal injection
Sham controlSHAM_COMPARATORSham injection was given to the study eye at baseline, and then treatment was given based on evidence of disease activity. At month 1, if treatment was needed, sham was administered. At month 2, participants switched to open-label ranibizumab on an as needed basis.
Group IEXPERIMENTAL0.5 mg ranibizumab driven by visual acuity stability criteria
Group IIEXPERIMENTAL0.5 mg ranibizumab driven by disease activity criteria
Group IIIACTIVE_COMPARATORverteporfin PDT
RBZ 0.5 mg: VA only (Group I)EXPERIMENTALRBZ 0.5 mg: Visual Acuity (VA) only (Group I) 0.5 mg intravitreal injections of ranibizumab with retreatment based on best-corrected visual acuity (BCVA) loss due to neovascular (wet) age-related macular degeneration (nAMD)
RBZ 0.5 mg: VA and/or OCT (Group II)EXPERIMENTALRBZ 0.5 mg: VA and/or OCT (Group II) 0.5 mg intravitreal injections of ranibizumab with retreatment based on best-corrected visual acuity (BCVA)loss due to neovascular (wet) age-related macular degeneration (nAMD) and/or signs of wet AMD disease activity on optical coherence tomography (OCT).
1-ranibizumab monotherapyEXPERIMENTALRanibizumab 0.5 mg
2-ranibizumab with laserEXPERIMENTALRanibizumab 0.5 mg + laser
3-laser monotherapyACTIVE_COMPARATORLaser monotherapy with Ranibizumab 0.5 mg from Month 6
Ranibizumab armEXPERIMENTALIntravitreal injection with standard dose of 0.5 mg/0.05mL PRN
Standard of CareACTIVE_COMPARATOR -
DexamethasoneSHAM_COMPARATORIntravitreal implant as per commercial label (700 μg Dexamethasone; long acting release (LAR) over 6 months
ranibizumab intravitreal injectionEXPERIMENTAL -
Ranibizumab driven by disease activityEXPERIMENTALParticipants received active ranibizumab on day 1. Thereafter they received ranibizumab treatment based on disease activity criteria
Ranibizumab driven by stabilization criteriaEXPERIMENTALParticipants received ranibizumab on day 1 and month 1. Thereafter they received ranibizumab based on stabilization criteria for visual acuity
Verteporfin PDTACTIVE_COMPARATORParticipants received active vPDT on day 1. From month 3 onwards participants could receive ranibizumab, vPDT or a combination of the two if needed.
TE Ranibizumab 0.5 mg and LaserEXPERIMENTALOn Day 1, all patients received an intravitreal injection with 0.5 mg ranibizumab and subsequently entered Phase A which comprised of monthly injections. Laser therapy was applied at Day 1. It could then be re-administered according to ETDRS criteria at any visit with 0.5 mg ranibizumab treatment if deemed necessary by the Treating Investigator with a minimal treatment interval between laser treatments of 3 months. Laser therapy was administered ≥ 30 minutes prior to the ranibizumab injection.
TE Ranibizumab 0.5 mg aloneEXPERIMENTALPatients received ranibizumab intravitreal injection therapy only.
PRN Ranibizumab 0.5 mgACTIVE_COMPARATORPatients received ranibizumab intravitreal injection therapy as needed according to signs and symptoms of disease.
Combination TherapyEXPERIMENTALParticipants received ranibizumab intravitreal injection and laser photocoagulation treatments
Ranibizumab MonotherapyEXPERIMENTALParticipants received ranibizumab intravitreal injection therapy only
Laser MonotherapyACTIVE_COMPARATORParticipants received Laser photocoagulation therapy only
Adjunctive treatmentEXPERIMENTALAdjunctive administration of ranibizumab 0.5 mg intravitreal injections and active laser.
Monotherapy treatmentEXPERIMENTALMonotherapy ranibizumab 0.5 mg intravitreal injections plus sham laser.
Laser controlACTIVE_COMPARATORActive laser treatment plus sham intravitreal injections.
1EXPERIMENTAL -
Ranibizumab 0.5 mg + laserEXPERIMENTALRanibizumab 0.5 mg was administered monthly by intravitreal injection in the study eye for 3 months. After the third injection, treatment was suspended if either one of the following criteria was met: Improvement in best corrected visual acuity (BCVA) could not be attributed to treatment at the last 2 visits, in the opinion of the investigator, or BCVA \> 84 letters (approximate Snellen equivalent of 20/20) was observed at the last 2 last visits. Patients also received active laser treatment on Day 1 and subsequently at intervals of at least 3 months, if deemed necessary by the evaluating physician. Active/sham laser treatment was always administered before (sham) intravitreal injections. The minimum interval between the 2 treatments was 30 minutes. In the extension study at the investigator's discretion, patients received open-label ranibizumab 0.5 mg intravitreal injections once a month until stable vision was reached (a maximum of 24 injections) and could receive laser therapy.
AACTIVE_COMPARATOR0.5 ml 10mg/ml (0.5 mg) ranibizumab for intravitreal injection
BSHAM_COMPARATOR -
LucentisEXPERIMENTALAll patients receive iL for for first 6 months of study. At 6 months - patients are classified as "responders" or "non-responders". "Responders" receive iL PRN based on OCT,clinical exam etc. "Non-responders" are seen again at 12 months for repeat investigations.
A: Ranibizumab 0.5mg (0.05mL) injectionEXPERIMENTALRanibizumab 0.5mg (0.05mL) injection at end of trabeculectomy surgery. This intra-operative adjunct therapy was administered sub-conjunctivally 8-10mm posteriorly to the limbus as an antifibrotic agent.
B: Mitomycin C 0.4 mg/ml spongeACTIVE_COMPARATORMitomycin C 0.4 mg/ml soaked sponge applied to sclera (for up to 2 min) after flap is made during trabeculectomy surgery. This is the typical method used as an antifibrotic agent.
Ranibizumab monoEXPERIMENTALInterventional Core Phase: One intravitreal injection of ranibizumab 0.5 mg to the study eye monthly until stability regarding morphological parameters is confirmed (ie, no further improvement of morphology or no worsening of morphology for 3 consecutive months)
PRP monoACTIVE_COMPARATORInterventional Core Phase: Panretinal laser photocoagulation (PRP) treatment administered to the study eye in accordance with the modified diabetic retinopathy study (DRS) guidelines for panretinal laser photocoagulation procedures
Ranibizumab+PRPEXPERIMENTALInterventional Core Phase: Ranibizumab 0.5 mg as described for the ranibizumab mono arm and PRP treatment as described for the PRP mono arm until stability regarding morphological parameters is confirmed
ranibizumab 0.5mgEXPERIMENTAL -
Ranibizumab 0.3 mgEXPERIMENTALParticipants received monthly intravitreal injections with 0.3 mg ranibizumab (6 mg/ml) for up to 12 months. At each monthly visit from month 1 and onwards, the evaluating physician decided whether an increase in the dose to 0.6 mg was needed according to set criteria. If the dose was increased, all subsequent administrations were of the higher dose unless treatment had been withheld for more than 45 days (for any reason), in which case injections restarted with the initial dose. Laser photocoagulation was permitted as rescue treatment for the study eye after 3 consecutive monthly injections.
Group A: Ranibizumab 0.3 mgEXPERIMENTALIn the single dose phase, all patients randomized in Group A received a single intravitreal injection of 0.3 mg of ranibizumab into the study eye. Those patients who successfully completed this phase entered the multiple dose phase, where they received an intravitreal injection of 0.3 mg of ranibizumab once a month for an additional 11 months. Subsequently patients enrolling in the extension phase received an intravitreal injection of 0.3 mg of ranibizumab according to an individualized flexible interval regimen guided by monthly best corrected visual acuity scores and other ophthalmic examinations. In the extension phase patients received the same dose level as they received in the multiple dose phase of the study, for an average of 1.70 years.
Group A: Ranibizumab 0.5 mgEXPERIMENTALIn the single dose phase, all patients randomized in Group A received a single intravitreal injection of 0.5 mg of ranibizumab into the study eye. Those patients who successfully completed this phase entered the multiple dose phase, where they received an intravitreal injection of 0.5 mg of ranibizumab once a month for an additional 11 months. Subsequently Group A patients enrolling in the extension phase received an intravitreal injection of 0.5 mg of ranibizumab according to an individualized flexible interval regimen guided by monthly best corrected visual acuity scores and other ophthalmic examinations. In the extension phase patients received the same dose level as they received in the multiple dose phase of the study, for an average of 1.93 years.
Group B: Ranibizumab 0.3 mgEXPERIMENTALGroup B patients received a total of 12 monthly intravitreal injections of 0.3 mg of ranibizumab into the study eye in the multiple dose phase of the study. Group B patients who enrolled in the extension phase received an intravitreal injection of 0.3 mg of ranibizumab according to an individualized flexible interval regimen guided by monthly best corrected visual acuity scores and other ophthalmic examinations. In the extension phase patients received the same dose level as they received in the multiple dose phase of the study, for an average of 1.45 years.
Group B: Ranibizumab 0.5 mgEXPERIMENTALGroup B patients received a total of 12 monthly intravitreal injections of 0.5 mg of ranibizumab into the study eye in the multiple dose phase of the study. Group B patients who enrolled in the extension phase received an intravitreal injection of 0.5 mg of ranibizumab according to an individualized flexible interval regimen guided by monthly best corrected visual acuity scores and other ophthalmic examinations. In the extension phase patients received the same dose level as they received in the multiple dose phase of the study, for an average of 1.36 years.

Interventions

NameTypeDescription
RanibizumabDRUG1 intravitreal injection in both eyes on Day 1 (Baseline), with up to 2 re-treatments allowed for each eye if required
Laser therapyPROCEDURETranspupillary diode or frequency-doubled yttrium aluminum garnet (YAG) laser ablative therapy, following anesthesia or sedation
Ranibizumab (RFB002)DRUGRanibizumab 0.5 mg/0.05 mL for intravitreal injection
LaserPROCEDURELaser photocoagulation according to Early Treatment Diabetic Retinopathy Study (ETDRS) guidelines
Sham injectionDRUGImitation of an intravitreal injection consisting of an empty vial and an injection syringe without a needle
Sham laserPROCEDUREImitation of an active laser
Ranibizumab 0.5mgDRUG0.5 mg ranibizumab (intravitreal injections) prefilled syringe)
Ranibizumab 0.5 mgDRUGintravitreal injection of 0.05 ml
Sham controlOTHERThe sham vial did not contain active drug (empty sterile vial). The sham injection was an imitation of an intravitreal injection using an injection syringe without a needle touching the eye.
Verteporfin PDTDRUGVerteporfin for intravenous injection delivered by intravenous infusion followed by the light application
Ranibizumab 0.5 mg/0.05 mlDRUGPatients will receive the first dose at Baseline, as an intravitreal injection with a standard dose of 0.5 mg/0.05 ml. Patients will receive at least 3 study treatments at monthly intervals (Day 1, Month 1 and Month 2). The last mandatory dose during treatment initiation will be administered approximately 60 days after the first study treatment. If there is no improvement in VA over the course of the first 3 injections, continued treatment is not recommended and the patient may receive alternative treatment at the investigator's discretion.
Dexamethasone implant and sham injectionsDRUG -
Dexamethasone ImplantOTHERComparator Ozurdex® (dexamethasone) was formulated as a rod shaped implant to be inserted into the eye by an applicator. The implant as well as the respective applicator were suitable for single use only.
Sham RanibizumabDRUGEmpty vial to mimic the intravitreal injection
Sham verteporfin PDTDRUGSham vPDT intravenous infusion of dextrose 5% solution followed by light application (PDT).
Laser photocoagulationPROCEDUREActive laser treatment administered at day 1. Subsequent laser treatments administered if needed at intervals no shorter than 3 months from previous laser treatment.
Sham laser photocoagulationPROCEDURESham laser treatment administered at day 1.
Sham to ranibizumabDRUGSham to ranibizumab administered as an intravitreal injection.
Mitomycin (MMC)DRUGMitomycin (MMC) C 0.4 mg/ml applied with soaked pledget inserted in the sub-tenon's space during glaucoma surgery.
Panretinal laser photocoagulationPROCEDUREPRP treatment following DRS guidelines
Ranibizumab 0.3 mgDRUG6 mg/ml ranibizumab solution for intravitreal injection
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Eligibility Criteria

Age Range6 Months to 5 Years
SexALL
Healthy VolunteersNo
Study Sites73

Inclusion Criteria: * Signed informed consent from parent(s) or legal guardian(s), in compliance with local requirements * The patient successfully completed the core study H2301, as defined by providing assessments at the Visit 112 (the last scheduled visit in the core study) or, if appropriate, a...

Countries:United StatesAustriaBelgiumCroatiaCzechiaDenmarkEgyptEstoniaFranceGermanyGreeceHungaryIndiaItalyJapanLithuaniaMalaysiaRomaniaRussiaSaudi ArabiaSlovakiaTaiwanTurkey (Türkiye)United KingdomMexicoPolandChinaChileIsraelPortugalSloveniaSouth KoreaSpainSwitzerlandHong KongIndonesiaPhilippinesVietnamAustraliaCanadaLatviaNetherlandsSingaporeThailandPeruArgentinaColombiaFinlandGuatemalaIrelandPanamaSwedenNorway
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Frequently asked questions about Ranibizumab

What is Ranibizumab used for?

Ranibizumab is used for choroidal neovascularization (CNV), pigment epithelial detachment, uveitis related cystoid macular edema, visual impairment due to CNV secondary to pathologic myopia, age-related macular degeneration, and macular edema. It is an ophthalmology drug developed by Novartis AG.

What does Ranibizumab target?

Ranibizumab is a small molecule that targets vascular endothelial growth factor A (VEGF-A). By binding to VEGF-A, it inhibits angiogenesis and vascular permeability, which are key drivers of retinal diseases such as age-related macular degeneration and macular edema.

Who makes Ranibizumab?

Ranibizumab is developed by Novartis AG, a company traded on the New York Stock Exchange under the ticker NVS. Novartis is responsible for the clinical development and commercialization of the drug.

What phase is Ranibizumab in?

Ranibizumab is in Phase 3 clinical development. It has completed six trials with a total enrollment of 1,890 participants. The drug is investigational and not yet approved, as it is still undergoing clinical trials to evaluate its safety and efficacy.

What clinical trials is Ranibizumab in?

Ranibizumab has completed six clinical trials, including NCT00288561, NCT00942864, NCT01535261, and NCT01846299. These trials studied ranibizumab in conditions such as age-related macular degeneration, retinal vein occlusion, and macular edema across multiple countries.

Is Ranibizumab the same as Lucentis?

Ranibizumab is the same as Lucentis, a brand name used for the drug. It is also known by other names, but Lucentis is the most common alternative name. This information helps readers identify the drug when searching under different names.