Trial Outcomes & Findings for Macugen Observational Study (NCT NCT00735943)

NCT ID: NCT00735943

Last Updated: 2011-08-29

Results Overview

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.

• Recruitment status: TERMINATED
• Target enrollment: 22 participants
• Primary outcome timeframe: Baseline through 12 months or last follow-up visit before study termination
• Results posted on: 2011-08-29

Participant Flow

Participant milestones

Measure	Pegaptanib Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Overall Study STARTED	22
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Pegaptanib Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Overall Study Lost to Follow-up	1
Overall Study Adverse Event	1

Baseline Characteristics

Macugen Observational Study

Baseline characteristics by cohort

Measure	Pegaptanib n=22 Participants Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Age Continuous	70.4 years STANDARD_DEVIATION 10.9 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Pegaptanib n=22 Participants Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA) Improvement	18.2 percentage of participants
Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA) Stabilization	54.5 percentage of participants
Percentage of Participants Showing Stabilization, Improvement or Deterioration of Visual Acuity (VA) Deterioration	18.2 percentage of participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Pegaptanib n=12 Participants Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Average Number of Injections to Achieve Stabilization of VA	2.25 injections Standard Deviation 1.14

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Pegaptanib n=12 Participants Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Median Number of Injections to Achieve Stabilization of VA	2.00 injections Interval 1.0 to 4.0

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Pegaptanib n=22 Participants Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Percentage of Participants Receiving Macugen Monotherapy Versus Those Receiving a Combination Therapy Macugen monotherapy	100 Percentage of participants
Percentage of Participants Receiving Macugen Monotherapy Versus Those Receiving a Combination Therapy Combination therapy	0 Percentage of participants

SECONDARY outcome

Timeframe: 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.

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).

Outcome measures

Measure	Pegaptanib n=22 Participants Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters Improvement	13.6 Percentage of participants
Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters No improvement	54.5 Percentage of participants
Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters Lost to follow up	27.3 Percentage of participants
Percentage of Participants Showing Improvement in Optical Coherence Tomography (OCT) Parameters No 'center subfield' value	4.5 Percentage of participants

SECONDARY outcome

Timeframe: 12 months or last follow-up visit before study termination

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

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome data not reported

Adverse Events

Pegaptanib

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Pegaptanib n=22 participants at risk Macugen 0.3 mg (pegaptanib sodium) administered once every 6 weeks by intravitreal injection into the study eye.
Eye disorders Retinal haemorrhage	4.5% 1/22 • Number of events 2 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.
Eye disorders Visual acuity reduced	4.5% 1/22 • Number of events 2 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.
Eye disorders Subretinal fibrosis	4.5% 1/22 • Number of events 1 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.

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer, Inc.

Phone: 1-800-718-1021

Email: ClinicalTrials.gov_Inquiries@pfizer.com

Results disclosure agreements

• Principal investigator is a sponsor employee 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.
• Publication restrictions are in place

Restriction type: OTHER