Macugen Observational Study

• Sponsor: Pfizer (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT00735943
• Collaborator: (none)
• Enrollment: 22
• Locations: 5
• Duration (Months): 21
• Patients Per Site: 4.4
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Vascular Endothelial Growth Factor; Macular Degeneration	Other: No intervention

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.

Study Design

• Study Type: Observational
• Actual Enrollment: 22 participants
• Observational Model: Case-Only
• Time Perspective: Prospective
• Official Title: Macugen Observational Study
• Study Start Date: Nov 1, 2008
• Actual Primary Completion Date: Aug 1, 2010
• Actual Study Completion Date: Aug 1, 2010

Outcome Measures

Primary Outcome Measures

1. 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.

2. 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.

3. 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 Outcome Measures

1. 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.

2. 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).

3. 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.

4. 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.

5. 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.

6. 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.

7. 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.

8. 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.

9. 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.

10. 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.

11. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 90 Years
• Sexes Eligible for Study: All
• Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Pfizer Investigational Site	Navrangpura, Ahemdabad	Gujarat	India	380009
2	Pfizer Investigational Site	Gurgaon	Haryana	India	122 002
3	Pfizer Investigational Site	Cochin	Kerala	India	682 015
4	Pfizer Investigational Site	Mumbai	Maharashtra	India	400 054
5	Pfizer Investigational Site	Jaipur	Rajasthan	India	302 004

Sponsors and Collaborators

• Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center, Pfizer

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• To obtain contact information for a study center near you, click here.

Publications

None provided.

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT00735943
• Other Study ID Numbers: A5751031
• First Posted: Aug 15, 2008
• Last Update Posted: Aug 29, 2011
• Last Verified: Aug 1, 2011

• Keywords provided by Pfizer: Pegaptanib; macular degeneration; vascular endothelial growth factor
• Additional relevant MeSH terms: Macular Degeneration

Study Results

Participant Flow

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Period Title: Overall Study
STARTED	22
COMPLETED	20
NOT COMPLETED	2

Baseline Characteristics

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Overall Participants	22
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	70.4 (10.9)
Sex: Female, Male (Count of Participants)
Female	8 36.4%
Male	14 63.6%

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA)
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	22
Improvement	18.2 82.7%
Stabilization	54.5 247.7%
Deterioration	18.2 82.7%

2. Primary Outcome

Title	Average Number of Injections to Achieve Stabilization of VA
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	12
Mean (Standard Deviation) [injections]	2.25 (1.14)

3. Primary Outcome

Title	Median Number of Injections to Achieve Stabilization of VA
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	12
Median (Full Range) [injections]	2.00

4. Secondary Outcome

Title	Percentage of Participants Receiving Macugen Monotherapy Versus Those Receiving a Combination Therapy
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	22
Macugen monotherapy	100 454.5%
Combination therapy	0 0%

5. Secondary Outcome

Title	Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	22
Improvement	13.6 61.8%
No improvement	54.5 247.7%
Lost to follow up	27.3 124.1%
No 'center subfield' value	4.5 20.5%

6. Secondary Outcome

Title	Percentage of Participants Showing Improvement in Fundus Fluorescein Angiography (FFA) Parameters
Description	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

Outcome Measure Data

Analysis Population Description

Due to small sample size, the analysis was not conducted.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

7. Secondary Outcome

Title	Percentage of Participants With Early Lesions Showing Stabilization and Improvement of VA
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

8. Secondary Outcome

Title	Average Number of Injections to Achieve Stabilization of VA in Participants With Early Lesions
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

9. Secondary Outcome

Title	Median Number of Injections to Achieve Stabilization of VA in Participants With Early Lesions
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

10. Secondary Outcome

Title	Percentage of Participants With Occult or Minimally Classic and Classic Lesions Showing Stabilization and Improvement in VA
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

11. Secondary Outcome

Title	Percentage of Participants Who Were Treatment Naive When Started on Macugen Versus Those Previously Treated by Any Other Therapy Except Macugen
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

12. Secondary Outcome

Title	Percentage of Participants Showing Stabilization or Improvement in VA in the Subgroup Previously Treated by Other Therapy
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

13. Secondary Outcome

Title	Percentage of Participants Showing Improvement in OCT in the Subgroup Previously Treated by Other Therapy
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

14. Secondary Outcome

Title	Percentage of Participants Showing Improvement in OCT in the Subgroup Which Were Not Treatment Naive
Description	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

Outcome Measure Data

Analysis Population Description

Subgroup analysis was not performed as the study was terminated due to slow rate of recruitment.

Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Measure Participants	0

Adverse Events

Adverse Event Reporting Description	The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Arm/Group Title	Pegaptanib
Arm/Group Description	Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
All Cause Mortality
	Pegaptanib
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Pegaptanib
	Affected / at Risk (%)	# Events
Total	0/22 (0%)
Other (Not Including Serious) Adverse Events
	Pegaptanib
	Affected / at Risk (%)	# Events
Total	3/22 (13.6%)
Eye disorders
Retinal haemorrhage	1/22 (4.5%)	2
Visual acuity reduced	1/22 (4.5%)	2
Subretinal fibrosis	1/22 (4.5%)	1

Limitations/Caveats

Outcome measures were not designated as primary or secondary measures as this study was an observational non-interventional study.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.