Trial Outcomes & Findings for Efficacy, Safety and Tolerability Study of Subcutaneous C.E.R.A. in Pre-Dialysis Participants With Chronic Renal Anemia (NCT NCT00517881)

NCT ID: NCT00517881

Last Updated: 2016-05-27

Results Overview

Percentage of participants maintaining the mean hemoglobin concentration within +/- 1.0 g/dL of their reference hemoglobin value and within the target range of 10.5 to 12.5 g/dL during the efficacy evaluation period (EEP) was reported. The reference hemoglobin value was defined as the mean of the 5 assessments recorded during the stability verification period (SVP) at Weeks -4, -3, -2, -1 and 0. The mean hemoglobin concentration for each individual participant during the EEP (Week 17 to Week 24) was estimated as a time adjusted average.

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 29 participants
• Primary outcome timeframe: Week 17 up to Week 24
• Results posted on: 2016-05-27

Participant Flow

Participant milestones

Measure	C.E.R.A. Participants received subcutaneous methoxy polyethylene glycol-epoetin beta (C.E.R.A.) at starting dose of 120, 200, or 360 micrograms (mcg) every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Overall Study STARTED	29
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Measure	C.E.R.A. Participants received subcutaneous methoxy polyethylene glycol-epoetin beta (C.E.R.A.) at starting dose of 120, 200, or 360 micrograms (mcg) every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Overall Study Protocol Violation	2
Overall Study Adverse Event	1
Overall Study Renal transplantation	2

Baseline Characteristics

Efficacy, Safety and Tolerability Study of Subcutaneous C.E.R.A. in Pre-Dialysis Participants With Chronic Renal Anemia

Baseline characteristics by cohort

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Age, Continuous	60.6 years STANDARD_DEVIATION 11.29 • n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 17 up to Week 24

Population: The Intention-to-treat (ITT) population included all participants who received at least 1 dose of C.E.R.A. (week 0) and for whom data for at least 1 follow-up variable was available. Data missing at the end of the EEP (that is, the last measured hemoglobin value before Week 24) was handled using the last value carried forward method.

Percentage of participants maintaining the mean hemoglobin concentration within +/- 1.0 g/dL of their reference hemoglobin value and within the target range of 10.5 to 12.5 g/dL during the efficacy evaluation period (EEP) was reported. The reference hemoglobin value was defined as the mean of the 5 assessments recorded during the stability verification period (SVP) at Weeks -4, -3, -2, -1 and 0. The mean hemoglobin concentration for each individual participant during the EEP (Week 17 to Week 24) was estimated as a time adjusted average.

Outcome measures

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Percentage of Participants Maintaining Mean Hemoglobin Concentration Within Plus or Minus (+/-) 1 Gram Per Deciliter (g/dL) of Their Reference Hemoglobin and Within the Target Range	51.7 percentage of participants Interval 32.5 to 70.6

SECONDARY outcome

Timeframe: Week 17 up to Week 24

Population: ITT population; data missing at the end of the EEP was handled using the last value carried forward method.

The reference hemoglobin value was defined as the mean of the 5 assessments recorded during the SVP at Weeks -4, -3, -2, -1 and 0. The mean change of the time adjusted average of hemoglobin from reference value obtained during the SVP (Week -4 up to Week 0) and the value during EEP (Week 17 up to Week 24) was assessed.

Outcome measures

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Change in Hemoglobin Concentration Between Reference (SVP) and EEP	-0.3 g/dL Standard Deviation 1.04

SECONDARY outcome

Timeframe: Week 17 up to Week 24

Population: ITT Population; data missing at the end of the EEP was handled using the last value carried forward method.

Percentage of participants maintaining hemoglobin concentration within the target range of 10.5 to 12.5 g/dL during EEP (Week 17 to Week 24) was reported.

Outcome measures

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Percentage of Participants Maintaining Hemoglobin Concentration Within the Target Range During EEP	58.6 percentage of participants Interval 38.9 to 76.5

SECONDARY outcome

Timeframe: Week 17 up to Week 24

Population: ITT Population

Mean time spent by participants with hemoglobin concentration within the target range of 10.5 to 12.5 g/dL during the EEP (Week 17 to Week 24) was reported.

Outcome measures

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Mean Time Spent by Participants With Hemoglobin Concentration in the Target Range During the EEP	31 days Standard Deviation 17.2

SECONDARY outcome

Timeframe: Week 1 up to Week 16 and Week 17 up to Week 24

Population: ITT Population. Here, 'n' signifies the number of participants evaluable for specified category.

Percentage of participants requiring any adjustment in the dose of study drug during the dose titration period (DTP: Week 1 to Week 16) and EEP (Week 17 to Week 24) was reported.

Outcome measures

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Percentage of Participants Requiring Any Dose Adjustment DTP (n = 29)	62 percentage of participants
Percentage of Participants Requiring Any Dose Adjustment EEP (n = 26)	31 percentage of participants

SECONDARY outcome

Timeframe: Week 0 up to Week 24

Population: ITT Population

Percentage of participant who required red blood cell transfusion during the study was reported.

Outcome measures

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Percentage of Participants With Red Blood Cell Transfusion During the Study	7 percentage of participants

SECONDARY outcome

Timeframe: Week 0 up to Week 24

Population: Safety population

An AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both serious as well as non-serious AEs.

Outcome measures

Measure	C.E.R.A. n=29 Participants Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) AEs	22 participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) SAEs	9 participants

Adverse Events

C.E.R.A.

Serious events: 9 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Measure	C.E.R.A. n=29 participants at risk Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Blood and lymphatic system disorders Anaemia	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Cardiac disorders Atrial fibrillation	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
General disorders Oedema	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
General disorders Oedema peripheral	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Infections and infestations Diabetic foot infection	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Infections and infestations Pneumonia	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Infections and infestations Wound infection	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Injury, poisoning and procedural complications Arteriovenous fistula thrombosis	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Renal and urinary disorders Renal impairment	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Surgical and medical procedures Dialysis device insertion	3.4% 1/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.

Other adverse events

Measure	C.E.R.A. n=29 participants at risk Participants received subcutaneous C.E.R.A. at starting dose of 120, 200, or 360 mcg every 4 weeks for 20 weeks. Further dose adjustments were performed during the study depending on the participant's blood hemoglobin levels.
Gastrointestinal disorders Nausea	10.3% 3/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
General disorders Fatigue	10.3% 3/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Infections and infestations Nasopharyngitis	6.9% 2/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Musculoskeletal and connective tissue disorders Arthralgia	6.9% 2/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Nervous system disorders Dizziness	10.3% 3/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Nervous system disorders Headache	6.9% 2/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.
Vascular disorders Hypotension	6.9% 2/29 • Week 0 up to Week 24 Only adverse events with an onset date after the start of medication were included.

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