Trial Outcomes & Findings for An Observational Study to Evaluate the Effectiveness of Mircera Treatment for Renal Anemia in Pre-dialysis Participants With Chronic Kideny Disease (CKD) (NCT NCT02547454)

NCT ID: NCT02547454

Last Updated: 2017-06-07

Results Overview

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

• Recruitment status: COMPLETED
• Target enrollment: 393 participants
• Primary outcome timeframe: Baseline
• Results posted on: 2017-06-07

Participant Flow

Participant milestones

Measure	Methoxy Polyethylene Glycol-Epoetin Beta Participants with chronic kidney disease (CKD), who did not require dialysis, received methoxy polyethylene glycol-epoetin beta (Mircera) either intravenously (IV) or subcutaneously (SC), as per routine clinical practice, and were followed for approximately 36 months.
Overall Study STARTED	393
Overall Study COMPLETED	221
Overall Study NOT COMPLETED	172

Reasons for withdrawal

Measure	Methoxy Polyethylene Glycol-Epoetin Beta Participants with chronic kidney disease (CKD), who did not require dialysis, received methoxy polyethylene glycol-epoetin beta (Mircera) either intravenously (IV) or subcutaneously (SC), as per routine clinical practice, and were followed for approximately 36 months.
Overall Study Due to hemodialysis	53
Overall Study Due to transplantation	1
Overall Study Due to no further need to treat	4
Overall Study Death	38
Overall Study Lost to Follow-up	26
Overall Study Other	39
Overall Study No data	11

Baseline Characteristics

An Observational Study to Evaluate the Effectiveness of Mircera Treatment for Renal Anemia in Pre-dialysis Participants With Chronic Kideny Disease (CKD)

Baseline characteristics by cohort

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Age, Continuous	73 years STANDARD_DEVIATION 11.5 • n=5 Participants
Sex: Female, Male Female	216 Participants n=5 Participants
Sex: Female, Male Male	177 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=390 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hemoglobin (Hb) Value Within the Target Range (110 to 120 Grams Per Liter [g/L]) at Baseline	22.8 Percentage of participants

PRIMARY outcome

Timeframe: At Months 1-3

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=259 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) at Months 1-3	34 Percentage of participants

PRIMARY outcome

Timeframe: At Months 4-6

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=296 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) at Months 4-6	35.1 Percentage of participants

PRIMARY outcome

Timeframe: At Months 7-9

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=232 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) at Months 7-9	32.3 Percentage of participants

PRIMARY outcome

Timeframe: At Months 10-12

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=199 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) at Months 10-12	35.2 Percentage of participants

PRIMARY outcome

Timeframe: At Months 13-15

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=170 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) at Months 13-15	33.5 Percentage of participants

PRIMARY outcome

Timeframe: At Months 16-18

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=172 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) at Months 16-18	31.4 Percentage of participants

PRIMARY outcome

Timeframe: At Months 19-21

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=96 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) at Months 19-21	26 Percentage of participants

PRIMARY outcome

Timeframe: After 21 Months up to 36 Months

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=35 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (110 to 120 g/L) After 21 Months up to 36 Months	31.4 Percentage of participants

PRIMARY outcome

Timeframe: At Baseline

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=390 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hemoglobin (Hb) Value Within the Target Range (100 to 130 g/L) at Baseline	71.3 Percentage of participants

PRIMARY outcome

Timeframe: At Months 1-3

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=259 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) at Months 1-3	77.2 Percentage of participants

PRIMARY outcome

Timeframe: At Months 4-6

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=296 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) at Months 4-6	73.6 Percentage of participants

PRIMARY outcome

Timeframe: At Months 7-9

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=232 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) at Months 7-9	74.1 Percentage of participants

PRIMARY outcome

Timeframe: At Months 10-12

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=199 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) at Months 10-12	75.9 Percentage of participants

PRIMARY outcome

Timeframe: At Months 13-15

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=170 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) at Months 13-15	78.2 Percentage of participants

PRIMARY outcome

Timeframe: At Months 16-18

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=172 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) at Months 16-18	79.7 Percentage of participants

PRIMARY outcome

Timeframe: At Months 19-21

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=96 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) at Months 19-21	75 Percentage of participants

PRIMARY outcome

Timeframe: After 21 Months up to 36 Months

Population: Analysis population included all enrolled participants who had at least one Hb assessment at the specified time frame.

If a participant had more than 1 assessment during the specified time frame, the last observed Hb value was considered for calculation.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=35 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Hb Value Within the Target Range (100 to 130 g/L) After 21 Months up to 36 Months	68.6 Percentage of participants

SECONDARY outcome

Timeframe: Up to 36 Months

Population: Analysis population included all enrolled participants.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=390 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Median Time in Which Hb Value Was Maintained Within Target Range of 110-120 g/L	NA Months Median time (full range) to obtain Hb value within the target range was not possible to analyze due to fluctuation within and outside of range.

SECONDARY outcome

Timeframe: Up to 36 Months

Population: Analysis population included all enrolled participants.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Median Time in Which Hb Value Was Maintained Within Target Range of 100-130 g/L	NA Months Median time (full range) to obtain Hb value within the target range was not analyzed due to fluctuation within and outside of range.

SECONDARY outcome

Timeframe: Baseline

Population: Analysis population included all enrolled participants.

Average dose of methoxy polyethylene glycol-epoetin beta administered at Baseline

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Average Dose of Methoxy Polyethylene Glycol-Epoetin Beta	88 microgram Standard Deviation 46

SECONDARY outcome

Timeframe: Month 1, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and After 21 Months up to 36 Months

Population: Analysis population included all enrolled participants. Here, n signifies participants with available data at specified time-point.

Median monthly dose of methoxy polyethylene glycol-epoetin beta administered in the study up to 36 months.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 1 (n=393)	100 microgram Interval 50.0 to 400.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 3 (n=263)	75 microgram Interval 50.0 to 480.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 6 (n=300)	75 microgram Interval 30.0 to 300.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 9 (n=239)	67 microgram Interval 30.0 to 400.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 12 (n=206)	50 microgram Interval 25.0 to 360.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 15 (n=175)	50 microgram Interval 30.0 to 360.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 18 (n=177)	50 microgram Interval 30.0 to 250.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta Month 21 (n=93)	50 microgram Interval 0.0 to 300.0
Median Dose of Methoxy Polyethylene Glycol-Epoetin Beta After 21 Months up to 36 Months (n=30)	50 microgram Interval 30.0 to 250.0

SECONDARY outcome

Timeframe: Up to 36 Months

Population: Analysis population included all enrolled participants.

Total number of changes (increase or decrease) in daily methoxy polyethylene glycol-epoetin beta doses. The reasons for dose-adaptations included: inflammation or infection; kidney function decline; over-response; iron deficiency; insufficient response; adverse effect; start of maintenance dose; kidney function improvement; re-introduction of treatment; and others (other reasons than specified).

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Number of Dose Adaptations Adverse effect	2 Events
Number of Dose Adaptations Start of maintenance phase	71 Events
Number of Dose Adaptations Total dose adaptations	729 Events
Number of Dose Adaptations Inflammation or infection	11 Events
Number of Dose Adaptations Kidney function decline	32 Events
Number of Dose Adaptations Over response	314 Events
Number of Dose Adaptations Iron deficiency	14 Events
Number of Dose Adaptations Insufficient response	213 Events
Number of Dose Adaptations Kidney function improvement	4 Events
Number of Dose Adaptations Re-introduction of treatment	41 Events
Number of Dose Adaptations Other	27 Events

SECONDARY outcome

Timeframe: Up to 36 Months

Population: Analysis population included all enrolled participants.

Percentage of participants who did not use methoxy polyethylene glycol-epoetin beta (Dose 0) at atleast one visit during study period.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Dose 0	32.6 Percentage of participants

SECONDARY outcome

Timeframe: Up to 36 Months

Population: Analysis population included all enrolled participants.

Iron replacement was given to the participants either in oral iron replacement or intravenous replacement or both.

Outcome measures

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 Participants Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Percentage of Participants With Iron Replacement Oral Iron Replacement	37.7 Percentage of participants
Percentage of Participants With Iron Replacement Intravenous Iron Replacment	31.6 Percentage of participants
Percentage of Participants With Iron Replacement Oral and Intravenous Iron Replacment	10.9 Percentage of participants
Percentage of Participants With Iron Replacement No Iron Replacment	19.8 Percentage of participants

Adverse Events

Methoxy Polyethylene Glycol-Epoetin Beta

Serious events: 3 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 participants at risk Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Nervous system disorders Cerebrovascular accident	0.25% 1/393 • Up to 36 Months
Nervous system disorders Epileptic seizure	0.25% 1/393 • Up to 36 Months
Cardiac disorders Orthostatic collapse	0.25% 1/393 • Up to 36 Months

Other adverse events

Measure	Methoxy Polyethylene Glycol-Epoetin Beta n=393 participants at risk Participants with CKD, who did not require dialysis, received methoxy polyethylene glycol-epoetin beta either IV or SC, as per routine clinical practice, and were followed for approximately 36 months.
Skin and subcutaneous tissue disorders Rash	0.25% 1/393 • Up to 36 Months
Gastrointestinal disorders Diarrhoea	0.25% 1/393 • Up to 36 Months

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800 821-8590

Email: genentech@druginfo.com

Results disclosure agreements

• Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
• Publication restrictions are in place

Restriction type: OTHER