Macular Degeneration, Vascular Endothelial Growth Factor
Conditions
Keywords
Pegaptanib, macular degeneration, vascular endothelial growth factor
Brief summary
The objective of this observational study is to evaluate the effectiveness and safety of Macugen for treatment of wet age-related macular degeneration (AMD) in Indian patients.Prospective, Observational, Non-interventional Study. The period of observation for the study will be 1 year
Detailed description
To be eligible for enrollment in this study, patients must receive the first injection of Macugen intravitreal in at least one eye for treatment of wet age-related macular degeneration (AMD). The decision to prescribe Macugen will necessarily precede and will be independent of the decision to enroll patient into the study. If both eyes of a patient receive injection Macugen, only one eye will be included in the study. If both eyes receive first injection Macugen after initiation of the study, only the first treated eye will be included in the analysis. If one eye has already received Macugen when the study starts and the second eye receives injection after the study initiation, the second eye will be included in the analysis. The study was prematurely discontinued due to delay in meeting pre-defined protocol recruitment milestones on August 30, 2010. There were no safety concerns regarding the study in the decision to terminate the trial.
Interventions
OTHERNo intervention
No intervention
Sponsors
Pfizer
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
* To be eligible for enrollment in this study, patients must receive the first injection of Macugen intravitreal in at least one eye for treatment of wet age-related macular degeneration (AMD).
Exclusion criteria
* Active or suspected ocular or periocular infection. * Known hypersensitivity to pegaptanib sodium or any other excipient in this product.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA) | Baseline through 12 months or last follow-up visit before study termination | VA was measured using ETDRS (Early Treatment Diabetic Retinopathy Study) chart at 4 meter distance, at 1 meter distance (if participant's VA was poor) or verifying if the participant was able only to count fingers, to perceive hand motion or light. VA was assessed as the number of ETDRS letters correctly read. VA statuses were defined as: Stabilization: loss of less than 15 letters in the best corrected VA (BCVA); Improvement: gain of more than or equal to 15 letters in the BCVA; Deterioration: loss of more than or equal to 15 letters in the BCVA. |
| Average Number of Injections to Achieve Stabilization of VA | 12 months or last follow-up visit before study termination | Stabilization of VA was defined as loss of less than 15 letters in the BCVA. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up. |
| Median Number of Injections to Achieve Stabilization of VA | 12 months or last follow-up visit before study termination | Stabilization of VA was defined as loss of less than 15 letters in the BCVA. Median number of injections to achieve stabilization of VA was estimated via the Kaplan Meier method. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Early Lesions Showing Stabilization and Improvement of VA | 12 months or last follow-up visit before study termination | Early lesions were defined by any 2 of the following criteria: occult lesion diagnosed on FFA; baseline VA of more than or equal to 54 ETDRS letters; lesion size of less than 2DA on FFA. Stabilization of VA was defined as loss of less than 15 letters in the BCVA. Improvement in the VA was defined as gain of more than or equal to 15 letters in the BCVA. |
| Average Number of Injections to Achieve Stabilization of VA in Participants With Early Lesions | 12 months or last follow-up visit before study termination | Stabilization of VA was defined as loss of less than 15 letters in the BCVA. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up. |
| Median Number of Injections to Achieve Stabilization of VA in Participants With Early Lesions | 12 months or last follow-up visit before study termination | Stabilization of VA was defined as loss of less than 15 letters in the BCVA. Median number of injections to achieve stabilization of VA in participants with early lesions was estimated via the Kaplan Meier method. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up. |
| Percentage of Participants With Occult or Minimally Classic and Classic Lesions Showing Stabilization and Improvement in VA | 12 months or last follow-up visit before study termination | FFA was utilized to characterize the lesions as follows: Classic lesion: more than 50% of the lesion had a well-demarcated area of hyperfluorescence; minimally classic lesion: less than or equal to 50% of lesion had well-demarcated area of hyperfluorescence; occult lesion: lesion with no well demarcated borders. VA statuses were defined as: stabilization: loss of less than 15 letters in the BCVA; improvement: gain of more than or equal to 15 letters in the BCVA. |
| Percentage of Participants Receiving Macugen Monotherapy Versus Those Receiving a Combination Therapy | 12 months or last follow-up visit before study termination | Macugen monotherapy: referred to participants receiving Macugen in the study eye during the study that is (i.e.) participants without any concomitant drug treatment or nondrug treatment for the study eye during the study. Combination therapy: referred to participants receiving combination therapy in the study eye (during the study) i.e. participants with any concomitant drug treatment or nondrug treatment for the study eye during the study. |
| Percentage of Participants Showing Stabilization or Improvement in VA in the Subgroup Previously Treated by Other Therapy | 12 months or last follow-up visit before study termination | VA was measured using ETDRS chart at 4 meter distance, at 1 meter distance (if patient's VA was poor) or verifying if the patient was able only to count fingers, to perceive hand motion or light. VA was measured as the number of ETDRS letters correctly read. VA statuses were defined as: stabilization: loss of less than 15 letters in the BCVA; improvement: gain of more than 15 letters in the BCVA. |
| Percentage of Participants Showing Improvement in OCT in the Subgroup Previously Treated by Other Therapy | 12 months or last follow-up visit before study termination | OCT, a noninvasive, noncontact, transpupillary imaging technology, was utilized to image retinal structures in vivo with a resolution of 10 to 17 microns. The anatomic layers within the retina, retinal thickness could be measured. Improvement in OCT parameters was defined as a reduction of more than or equal to 100 microns in the central macular thickness (Center subfield). Improvement in OCT parameters was measured based on this single parameter. |
| Percentage of Participants Showing Improvement in OCT in the Subgroup Which Were Not Treatment Naive | 12 months or last follow-up visit before study termination | OCT, a noninvasive, noncontact, transpupillary imaging technology, was utilized to image retinal structures in vivo with a resolution of 10 to 17 microns. The anatomic layers within the retina, retinal thickness could be measured. Improvement in OCT parameters was defined as a reduction of more than or equal to 100 microns in the central macular thickness (Center subfield). Improvement in OCT parameters was measured based on this single parameter. |
| Percentage of Participants Who Were Treatment Naive When Started on Macugen Versus Those Previously Treated by Any Other Therapy Except Macugen | 12 months or last follow-up visit before study termination | Participants were considered treatment naive when started on Macugen and without any previous drug or non drug treatment administered to the study eye. Participants were considered previously treated by any other therapy if received any other drug or non drug treatment to the study eye except Macugen. |
| Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters | 12 months or last follow-up visit before study termination | OCT, a noninvasive, noncontact, transpupillary imaging technology, was utilized to image retinal structures in vivo with a resolution of 10 to 17 microns. The anatomic layers within the retina, retinal thickness could be measured. Improvement in OCT parameters was defined as a reduction of more than or equal to 100 microns in the central macular thickness (Center subfield). |
| Percentage of Participants Showing Improvement in Fundus Fluorescein Angiography (FFA) Parameters | 12 months or last follow-up visit before study termination | A fluorescein angiogram provides information about the condition of the retina. Improvement in FFA parameters was defined as absence of progression of the lesion or decrease in the size of the lesion and absence of new lesions on FFA i.e. change in lesion size from baseline must be less than or equal to 0 disc area(DA) and no new lesions. |
Countries
India
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Pegaptanib Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye. | 22 |
| Total | 22 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Lost to Follow-up | 1 |
Baseline characteristics
| Characteristic | Pegaptanib |
|---|---|
| Age Continuous | 70.4 years STANDARD_DEVIATION 10.9 |
| Sex: Female, Male Female | 8 Participants |
| Sex: Female, Male Male | 14 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 3 / 22 |
| serious Total, serious adverse events | 0 / 22 |
Outcome results
Primary
Average Number of Injections to Achieve Stabilization of VA
Stabilization of VA was defined as loss of less than 15 letters in the BCVA. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up.
Time frame: 12 months or last follow-up visit before study termination
Population: FAS included all participants who received at least 1 injection of the study treatment in the study eye during the study. Missing values were imputed by LOCF technique. Analysis was conducted for those participants who received at least 1 injection of the study treatment in the study eye and achieved stabilization at the last follow-up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Pegaptanib | Average Number of Injections to Achieve Stabilization of VA | 2.25 injections | Standard Deviation 1.14 |
Primary
Median Number of Injections to Achieve Stabilization of VA
Stabilization of VA was defined as loss of less than 15 letters in the BCVA. Median number of injections to achieve stabilization of VA was estimated via the Kaplan Meier method. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up.
Time frame: 12 months or last follow-up visit before study termination
Population: FAS included all participants who received at least 1 injection of the study treatment in the study eye during the study. Missing values were imputed by LOCF technique. Analysis was conducted for those participants who received at least 1 injection of the study treatment in the study eye and achieved stabilization at the last follow-up.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pegaptanib | Median Number of Injections to Achieve Stabilization of VA | 2.00 injections |
Primary
Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA)
VA was measured using ETDRS (Early Treatment Diabetic Retinopathy Study) chart at 4 meter distance, at 1 meter distance (if participant's VA was poor) or verifying if the participant was able only to count fingers, to perceive hand motion or light. VA was assessed as the number of ETDRS letters correctly read. VA statuses were defined as: Stabilization: loss of less than 15 letters in the best corrected VA (BCVA); Improvement: gain of more than or equal to 15 letters in the BCVA; Deterioration: loss of more than or equal to 15 letters in the BCVA.
Time frame: Baseline through 12 months or last follow-up visit before study termination
Population: Full Analysis Set (FAS) included all participants who received at least 1 injection of the study treatment in the study eye during the study. Missing values were imputed by Last Observation Carried Forward (LOCF) technique.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pegaptanib | Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA) | Improvement | 18.2 percentage of participants |
| Pegaptanib | Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA) | Stabilization | 54.5 percentage of participants |
| Pegaptanib | Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA) | Deterioration | 18.2 percentage of participants |
Secondary
Average Number of Injections to Achieve Stabilization of VA in Participants With Early Lesions
Stabilization of VA was defined as loss of less than 15 letters in the BCVA. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.
Secondary
Median Number of Injections to Achieve Stabilization of VA in Participants With Early Lesions
Stabilization of VA was defined as loss of less than 15 letters in the BCVA. Median number of injections to achieve stabilization of VA in participants with early lesions was estimated via the Kaplan Meier method. For each participant, number of injections before reaching the first stabilization in VA (considered as an event) was counted. For participant having no event, the time to the number of injections to reach an event was unobserved at the number of injections before the last follow up.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.
Secondary
Percentage of Participants Receiving Macugen Monotherapy Versus Those Receiving a Combination Therapy
Macugen monotherapy: referred to participants receiving Macugen in the study eye during the study that is (i.e.) participants without any concomitant drug treatment or nondrug treatment for the study eye during the study. Combination therapy: referred to participants receiving combination therapy in the study eye (during the study) i.e. participants with any concomitant drug treatment or nondrug treatment for the study eye during the study.
Time frame: 12 months or last follow-up visit before study termination
Population: FAS included all participants who received at least 1 injection of the study treatment in the study eye during the study. Missing values were imputed by LOCF analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pegaptanib | Percentage of Participants Receiving Macugen Monotherapy Versus Those Receiving a Combination Therapy | Macugen monotherapy | 100 Percentage of participants |
| Pegaptanib | Percentage of Participants Receiving Macugen Monotherapy Versus Those Receiving a Combination Therapy | Combination therapy | 0 Percentage of participants |
Secondary
Percentage of Participants Showing Improvement in Fundus Fluorescein Angiography (FFA) Parameters
A fluorescein angiogram provides information about the condition of the retina. Improvement in FFA parameters was defined as absence of progression of the lesion or decrease in the size of the lesion and absence of new lesions on FFA i.e. change in lesion size from baseline must be less than or equal to 0 disc area(DA) and no new lesions.
Time frame: 12 months or last follow-up visit before study termination
Population: Due to small sample size, the analysis was not conducted.
Secondary
Percentage of Participants Showing Improvement in OCT in the Subgroup Previously Treated by Other Therapy
OCT, a noninvasive, noncontact, transpupillary imaging technology, was utilized to image retinal structures in vivo with a resolution of 10 to 17 microns. The anatomic layers within the retina, retinal thickness could be measured. Improvement in OCT parameters was defined as a reduction of more than or equal to 100 microns in the central macular thickness (Center subfield). Improvement in OCT parameters was measured based on this single parameter.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.
Secondary
Percentage of Participants Showing Improvement in OCT in the Subgroup Which Were Not Treatment Naive
OCT, a noninvasive, noncontact, transpupillary imaging technology, was utilized to image retinal structures in vivo with a resolution of 10 to 17 microns. The anatomic layers within the retina, retinal thickness could be measured. Improvement in OCT parameters was defined as a reduction of more than or equal to 100 microns in the central macular thickness (Center subfield). Improvement in OCT parameters was measured based on this single parameter.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.
Secondary
Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters
OCT, a noninvasive, noncontact, transpupillary imaging technology, was utilized to image retinal structures in vivo with a resolution of 10 to 17 microns. The anatomic layers within the retina, retinal thickness could be measured. Improvement in OCT parameters was defined as a reduction of more than or equal to 100 microns in the central macular thickness (Center subfield).
Time frame: 12 months or last follow-up visit before study termination
Population: FAS included all participants who received at least 1 injection of the study treatment in the study eye during the study. Missing values were imputed by LOCF technique.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pegaptanib | Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters | Improvement | 13.6 Percentage of participants |
| Pegaptanib | Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters | No improvement | 54.5 Percentage of participants |
| Pegaptanib | Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters | Lost to follow up | 27.3 Percentage of participants |
| Pegaptanib | Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters | No 'center subfield' value | 4.5 Percentage of participants |
Secondary
Percentage of Participants Showing Stabilization or Improvement in VA in the Subgroup Previously Treated by Other Therapy
VA was measured using ETDRS chart at 4 meter distance, at 1 meter distance (if patient's VA was poor) or verifying if the patient was able only to count fingers, to perceive hand motion or light. VA was measured as the number of ETDRS letters correctly read. VA statuses were defined as: stabilization: loss of less than 15 letters in the BCVA; improvement: gain of more than 15 letters in the BCVA.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.
Secondary
Percentage of Participants Who Were Treatment Naive When Started on Macugen Versus Those Previously Treated by Any Other Therapy Except Macugen
Participants were considered treatment naive when started on Macugen and without any previous drug or non drug treatment administered to the study eye. Participants were considered previously treated by any other therapy if received any other drug or non drug treatment to the study eye except Macugen.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.
Secondary
Percentage of Participants With Early Lesions Showing Stabilization and Improvement of VA
Early lesions were defined by any 2 of the following criteria: occult lesion diagnosed on FFA; baseline VA of more than or equal to 54 ETDRS letters; lesion size of less than 2DA on FFA. Stabilization of VA was defined as loss of less than 15 letters in the BCVA. Improvement in the VA was defined as gain of more than or equal to 15 letters in the BCVA.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.
Secondary
Percentage of Participants With Occult or Minimally Classic and Classic Lesions Showing Stabilization and Improvement in VA
FFA was utilized to characterize the lesions as follows: Classic lesion: more than 50% of the lesion had a well-demarcated area of hyperfluorescence; minimally classic lesion: less than or equal to 50% of lesion had well-demarcated area of hyperfluorescence; occult lesion: lesion with no well demarcated borders. VA statuses were defined as: stabilization: loss of less than 15 letters in the BCVA; improvement: gain of more than or equal to 15 letters in the BCVA.
Time frame: 12 months or last follow-up visit before study termination
Population: Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.