Trial Outcomes & Findings for Complement Inhibition With Eculizumab for the Treatment of Non-Exudative Macular Degeneration (AMD) (NCT NCT00935883)
NCT ID: NCT00935883
Last Updated: 2017-05-30
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
60 participants
Primary outcome timeframe
6 months
Results posted on
2017-05-30
Participant Flow
Participant milestones
| Measure |
Eculizumab
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Saline
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
|---|---|---|
|
Treatment Period
STARTED
|
40
|
20
|
|
Treatment Period
COMPLETED
|
38
|
19
|
|
Treatment Period
NOT COMPLETED
|
2
|
1
|
|
Follow-Up Period
STARTED
|
38
|
19
|
|
Follow-Up Period
COMPLETED
|
38
|
19
|
|
Follow-Up Period
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
Eculizumab
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Saline
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
|---|---|---|
|
Treatment Period
Physician Decision
|
0
|
1
|
|
Treatment Period
Withdrawal by Subject
|
2
|
0
|
Baseline Characteristics
Complement Inhibition With Eculizumab for the Treatment of Non-Exudative Macular Degeneration (AMD)
Baseline characteristics by cohort
| Measure |
Saline
n=20 Participants
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Eculizumab
n=40 Participants
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Total
n=60 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
17 Participants
n=93 Participants
|
34 Participants
n=4 Participants
|
51 Participants
n=27 Participants
|
|
Age, Continuous
|
75.7 years
STANDARD_DEVIATION 8.64 • n=93 Participants
|
75.1 years
STANDARD_DEVIATION 8.0 • n=4 Participants
|
75.3 years
STANDARD_DEVIATION 8.2 • n=27 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=93 Participants
|
19 Participants
n=4 Participants
|
29 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=93 Participants
|
21 Participants
n=4 Participants
|
31 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=93 Participants
|
40 participants
n=4 Participants
|
60 participants
n=27 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: Geographic Atrophy Cohort included 10 eyes randomized to saline and 20 eyes randomized to Eculizumab
Outcome measures
| Measure |
Saline
n=10 Participants
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Eculizumab
n=20 Participants
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
|---|---|---|
|
Growth of Geographic Atrophy
|
0.18 millimeters
Standard Deviation 0.15
|
0.19 millimeters
Standard Deviation 0.12
|
PRIMARY outcome
Timeframe: 6 MonthsPopulation: Drusen Cohort included 10 eyes randomized to saline and 20 eyes randomized to Eculizumab
Outcome measures
| Measure |
Saline
n=10 Participants
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Eculizumab
n=20 Participants
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
|---|---|---|
|
Decrease in Drusen Volume
|
0.12 mm^3
Standard Deviation 0.08
|
0.15 mm^3
Standard Deviation 0.17
|
SECONDARY outcome
Timeframe: Baseline/ 6 MonthsPopulation: Drusen Cohort included 10 eyes randomized to saline and 20 eyes randomized to Eculizumab
Visual function was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better functioning. Maximum score would be 100 letters read and minimum would be count fingers, hand motion and light perception if there were no letters read on the chart.
Outcome measures
| Measure |
Saline
n=10 Participants
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Eculizumab
n=20 Participants
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
|---|---|---|
|
Change in Visual Acuity for Drusen Group
|
4.0 letters
Standard Deviation 7.0
|
2.4 letters
Standard Deviation 3.9
|
SECONDARY outcome
Timeframe: Baseline/ 6 MonthsPopulation: Geographic Atrophy Cohort included 10 eyes randomized to saline and 20 eyes randomized to Eculizumab
Visual function was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better functioning. Maximum score would be 100 letters read and minimum would be count fingers, hand motion and light perception if there were no letters read on the chart.
Outcome measures
| Measure |
Saline
n=10 Participants
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Eculizumab
n=20 Participants
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
|---|---|---|
|
Change in Visual Acuity for Geographic Atrophy Group
|
-2.6 letters
Standard Deviation 7.2
|
2.5 letters
Standard Deviation 4.1
|
Adverse Events
Saline
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Eculizumab
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Saline
n=20 participants at risk
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
Saline: Induction Period: patient will receive saline via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by saline for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive saline via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
Eculizumab
n=40 participants at risk
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
Eculizumab: Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg or 1200 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days) until week 24.
Observation period: patient will then be observed for 6 months off treatment with follow-up visits scheduled for 9 months and 12 months.
|
|---|---|---|
|
Cardiac disorders
chest pains
|
5.0%
1/20 • Number of events 1
|
0.00%
0/40
|
Additional Information
Philip J Rosenfeld
University of Miami Miller School of Medicine
Phone: 305-326-6148
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place