Trial Outcomes & Findings for Iron Isomaltoside/Ferric Derisomaltose vs Iron Sucrose for Treatment of Iron Deficiency Anemia in Non-Dialysis-Dependent Chronic Kidney Disease (NCT NCT02940860)

Last Updated: 2020-10-06

Results Overview

Efficacy Evaluate the effect of iron isomaltoside/ferric derisomaltose vs iron sucrose in subjects with non-dialysis-dependent chronic kidney disease (NDD-CKD) and iron deficiency anaemia (IDA). Response was defined as change from baseline in hemoglobin (Hb) to week 8, i.e. ability to increase Hb in subjects with NDD-CKD and IDA, when oral iron preparations were ineffective or could not be used, or in whom the Hb measurement at screening in Investigators' opinion were sufficiently low to require rapid repletion of iron stores.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

1538 participants

Primary outcome timeframe

Baseline to week 8

Results posted on

2020-10-06

Participant Flow

A total of 2560 subjects were screened and 1538 subjects were randomised into the trial.

Participant milestones

Participant milestones
Measure	Iron Isomaltoside/Ferric Derisomaltose Iron isomaltoside/ferric derisomaltose (Monofer®/Monoferric®; 100 mg/mL) was the test product in this trial. Subjects received iron isomaltoside/ferric derisomaltose (Monofer®/Monoferric®; 100 mg/mL) as a single IV infusion of 1000 mg at the baseline visit.	Iron Sucrose Iron sucrose (Venofer®) was the comparator in this trial. Subjects received iron sucrose (Venofer®), 200 mg IV injection up to a maximum of 5 IV injections within the first 2 weeks, starting at baseline (a cumulative dose of 1000 mg was recommended).
Overall Study STARTED	1027	511
Overall Study COMPLETED	971	476
Overall Study NOT COMPLETED	56	35

Reasons for withdrawal

Reasons for withdrawal
Measure	Iron Isomaltoside/Ferric Derisomaltose Iron isomaltoside/ferric derisomaltose (Monofer®/Monoferric®; 100 mg/mL) was the test product in this trial. Subjects received iron isomaltoside/ferric derisomaltose (Monofer®/Monoferric®; 100 mg/mL) as a single IV infusion of 1000 mg at the baseline visit.	Iron Sucrose Iron sucrose (Venofer®) was the comparator in this trial. Subjects received iron sucrose (Venofer®), 200 mg IV injection up to a maximum of 5 IV injections within the first 2 weeks, starting at baseline (a cumulative dose of 1000 mg was recommended).
Overall Study Non-serious AE	1	2
Overall Study Serious AE	7	3
Overall Study Withdrawal by Subject	19	9
Overall Study Lost to Follow-up	13	7
Overall Study Physician Decision	5	3
Overall Study Sponsor Decision	0	1
Overall Study Protocol Violation	0	2
Overall Study Death	3	2
Overall Study Moved, poor venous access, transport	6	4
Overall Study Not treated	2	2

Baseline Characteristics

Iron Isomaltoside/Ferric Derisomaltose vs Iron Sucrose for Treatment of Iron Deficiency Anemia in Non-Dialysis-Dependent Chronic Kidney Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV	Total n=1538 Participants Total of all reporting groups
Age, Continuous	68.3 years STANDARD_DEVIATION 12.3 • n=5 Participants	69.3 years STANDARD_DEVIATION 12.3 • n=7 Participants	68.6 years STANDARD_DEVIATION 12.3 • n=5 Participants
Age, Customized Between 18 and 65 years	337 participants n=5 Participants	151 participants n=7 Participants	488 participants n=5 Participants
Age, Customized 65-84 years	608 participants n=5 Participants	316 participants n=7 Participants	924 participants n=5 Participants
Age, Customized > 84 years	82 participants n=5 Participants	44 participants n=7 Participants	126 participants n=5 Participants
Sex: Female, Male Female	633 Participants n=5 Participants	329 Participants n=7 Participants	962 Participants n=5 Participants
Sex: Female, Male Male	394 Participants n=5 Participants	182 Participants n=7 Participants	576 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	476 Participants n=5 Participants	248 Participants n=7 Participants	724 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	551 Participants n=5 Participants	263 Participants n=7 Participants	814 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) Asian	16 Participants n=5 Participants	11 Participants n=7 Participants	27 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	264 Participants n=5 Participants	117 Participants n=7 Participants	381 Participants n=5 Participants
Race (NIH/OMB) White	731 Participants n=5 Participants	375 Participants n=7 Participants	1106 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	11 Participants n=5 Participants	6 Participants n=7 Participants	17 Participants n=5 Participants
Race/Ethnicity, Customized Mexican	4 participants n=5 Participants	3 participants n=7 Participants	7 participants n=5 Participants
Race/Ethnicity, Customized Indigenous Mexican	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Mixed (Caucasian and Native american)	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Hispanic	4 participants n=5 Participants	3 participants n=7 Participants	7 participants n=5 Participants
Race/Ethnicity, Customized Central Indian	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Guyanese	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Other (Mixed: not declared)	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment United States	1027 participants n=5 Participants	511 participants n=7 Participants	1538 participants n=5 Participants
Current smoker YES	118 participants n=5 Participants	53 participants n=7 Participants	171 participants n=5 Participants
Current smoker NO	909 participants n=5 Participants	458 participants n=7 Participants	1367 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to week 8

Population: Intention to treat (ITT). All randomised subjects.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Change in Hemoglobin (Hb) From Baseline to Week 8	1.22 g/dL Interval 1.14 to 1.31	1.14 g/dL Interval 1.03 to 1.26

PRIMARY outcome

Timeframe: Baseline to week 8

Population: Safety analysis set. All randomised subjects who received at least one dose of the investigational product.

Safety For this endpoint, the number of participants with serious or severe hypersensitivity reactions were evaluated. The hypersensitivity reactions that were included in the analysis were those that started on or after the first dose of randomised treatment (i.e. treatment emergent). The terms used to define hypersensitivity were those specified by the Standardised MedDRA Queries (SMQ) for hypersensitivity, plus four additional terms: loss of consciousness, seizure, syncope, unresponsiveness. The potential hypersensitivity AEs were adjudicated in a blinded fashion by an independent Clinical Endpoint Adjudication Committee (CEAC). Results show only those participants that had adjudicated and confirmed serious or severe hypersensitivity reactions.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1019 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=506 Participants Iron sucrose, administered IV
Incidence of Protocol-defined Serious or Severe Hypersensitivity Reactions	3 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, week 1, 2, and 8

Population: Safety analysis set. All randomised subjects who received at least one dose of the investigational product.

Safety Results show the composite cardiovascular AEs, that started on or after the first dose of randomised treatment (i.e. treatment emergent) up to week 8. The reported potential cardiovascular AEs were adjudicated in a blinded fashion by an independent Clinical Endpoint Adjudication Committee (CEAC). The potential cardiovascular AEs included the following: * Death due to any cause * Non-fatal myocardial infarction * Non-fatal stroke * Unstable angina requiring hospitalisation * Congestive heart failure requiring hospitalisation or medical intervention * Arrhythmias * Hypertension * Hypotension Results show only those participants that had adjudicated and confirmed treatment-emergent composite cardiovascular AEs.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1019 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=506 Participants Iron sucrose, administered IV
Composite Cardiovascular Adverse Events (AEs)	42 Participants	35 Participants

SECONDARY outcome

Timeframe: Baseline, week 1, 2, 4, and 8

Population: Safety analysis set. All randomised subjects who received at least one dose of the investigational product.

Safety Time to first composite cardiovascular AE was defined as the actual time in days from first dose of treatment until the date of the composite cardiovascular AE. For subjects not reporting a composite cardiovascular AE, the time was censored at the date of the last attended visit. Only the adjudicated and confirmed composite cardiovascular safety AEs, as judged by the CEAC, were considered for this endpoint. Time to first composite cardiovascular AE was defined as the actual time in days from first dose of treatment until the date of the composite cardiovascular AE. For subjects not reporting a composite cardiovascular AE, the time was censored at the date of the last attended visit.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1019 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=506 Participants Iron sucrose, administered IV
Time to First Composite Cardiovascular Safety AE	NA Week The number of adjudicated and confirmed composite cardiovascular safety AEs was too low (N=42) for estimation of median and 95% CI.	NA Week The number of adjudicated and confirmed composite cardiovascular safety AEs was too low (N=35) for estimation of median and 95% CI.

SECONDARY outcome

Timeframe: Baseline, week 1, 2, 4, and 8

Population: Safety analysis set. All randomised subjects who received at least one dose of the investigational product.

Safety Results show the number of participants who had s-phosphate \<2 mg/dL at any time from baseline to week 1, 2, 4, or 8.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1011 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=500 Participants Iron sucrose, administered IV
S-phosphate <2 mg/dL at Any Time From Baseline to Week 1, 2, 4, and 8	32 Participants	4 Participants

SECONDARY outcome

Timeframe: Baseline, week 1, 2, 4, and 8

Population: ITT. All randomised subjects.

Efficacy Results show Hb responders to the treatment. A subject was considered a Hb responder to a certain week if an increase in Hb of at least 1 g/dL from baseline to the week in question was observed (from baseline to week 1, 2, 4, and 8).

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Hb Concentration Increase of ≥1 g/dL From Baseline to Week 1, 2, 4, and 8 Responder YES week 1	200 Participants	78 Participants
Hb Concentration Increase of ≥1 g/dL From Baseline to Week 1, 2, 4, and 8 Responder YES week 2	339 Participants	112 Participants
Hb Concentration Increase of ≥1 g/dL From Baseline to Week 1, 2, 4, and 8 Responder YES week 4	430 Participants	174 Participants
Hb Concentration Increase of ≥1 g/dL From Baseline to Week 1, 2, 4, and 8 Responder YES week 8	474 Participants	226 Participants

SECONDARY outcome

Timeframe: Baseline, week 1, 2, 4, and 8

Population: ITT. All randomised subjects.

Efficacy Time to change in Hb concentration ≥1 g/dL. Subjects who showed Hb concentration increase of ≥1 g/dL (from baseline to week 1, 2, 4, and 8). For responders, time to Hb response was defined as the scheduled time from baseline until the visit where the first Hb response was measured.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Time to Change in Hb Concentration ≥1 g/dL	56 Days Interval 28.0 to 56.0	56 Days Interval 28.0 to The 95% Confidence Interval value for this treatment group could not be calculated.

SECONDARY outcome

Timeframe: Week 1 to week 8

Population: ITT. All randomised subjects.

Efficacy Hb concentration of \>12 g/dL at any time from week 1 to week 8. Results show the number of participants who achieved Hb concentration of \>12 g/dL at any time from week 1 to week 8.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=510 Participants Iron sucrose, administered IV
Hb Concentration of >12 g/dL at Any Time From Week 1 to Week 8	259 Participants	121 Participants

SECONDARY outcome

Timeframe: Week 1 to week 8

Population: ITT. All randomised subjects.

Efficacy Results show the number of participants who achieved Hb concentration increase of ≥2 g/dL at any time from week 1 to week 8.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=510 Participants Iron sucrose, administered IV
Hb Concentration Increase of ≥2 g/dL at Any Time From Week 1 to Week 8	307 Participants	133 Participants

SECONDARY outcome

Timeframe: Week 1 to week 8

Population: ITT. All randomised subjects.

Efficacy Proportion of subjects reaching the composite endpoint of s-ferritin concentration ≥100 ng/mL and TSAT of 20-50% at any time from week 1 to 8.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1012 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=500 Participants Iron sucrose, administered IV
S-Ferritin Concentration of ≥100 ng/mL and Transferrin Saturation (TSAT) of 20-50% at Any Time From Week 1 to Week 8	873 Participants	388 Participants

SECONDARY outcome

Timeframe: Baseline, week 1, 2, and 4

Population: ITT. All randomised subjects.

Efficacy Change in Hb concentration from baseline to week 1, 2, and 4.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Change in Hb Concentration From Baseline to Week 1, 2, and 4 Week 1	0.44 g/dL Standard Deviation 0.94	0.21 g/dL Standard Deviation 1.02
Change in Hb Concentration From Baseline to Week 1, 2, and 4 Week 2	0.77 g/dL Standard Deviation 1.16	0.50 g/dL Standard Deviation 1.15
Change in Hb Concentration From Baseline to Week 1, 2, and 4 Week 4	1.08 g/dL Standard Deviation 1.32	0.90 g/dL Standard Deviation 1.33

SECONDARY outcome

Timeframe: Baseline, week 1, 2, 4, and 8

Population: ITT. All randomised subjects.

Efficacy Changes in s-ferritin from baseline to weeks 1, 2, 4, and 8.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Change in S-ferritin From Baseline to Weeks 1, 2, 4, and 8 Week 1	492.4 ng/mL Standard Deviation 309.7	183.9 ng/mL Standard Deviation 129.4
Change in S-ferritin From Baseline to Weeks 1, 2, 4, and 8 Week 2	381.2 ng/mL Standard Deviation 283.8	292.4 ng/mL Standard Deviation 210.4
Change in S-ferritin From Baseline to Weeks 1, 2, 4, and 8 Week 4	258.4 ng/mL Standard Deviation 214.5	255.4 ng/mL Standard Deviation 265.6
Change in S-ferritin From Baseline to Weeks 1, 2, 4, and 8 Week 8	191.3 ng/mL Standard Deviation 196.1	187.9 ng/mL Standard Deviation 210.6

SECONDARY outcome

Timeframe: Baseline, week 1, 2, 4, and 8

Population: ITT. All randomised subjects.

Efficacy Changes in transferrin saturation (TSAT) from baseline to week 1, 2, 4, and 8.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Change in Transferrin Saturation (TSAT) From Baseline to Week 1, 2, 4, and 8 Week 1	12.10 percent Standard Deviation 31.99	4.31 percent Standard Deviation 23.10
Change in Transferrin Saturation (TSAT) From Baseline to Week 1, 2, 4, and 8 Week 2	5.84 percent Standard Deviation 29.79	5.64 percent Standard Deviation 14.35
Change in Transferrin Saturation (TSAT) From Baseline to Week 1, 2, 4, and 8 Week 4	4.99 percent Standard Deviation 30.00	5.59 percent Standard Deviation 12.31
Change in Transferrin Saturation (TSAT) From Baseline to Week 1, 2, 4, and 8 Week 8	5.10 percent Standard Deviation 30.17	5.93 percent Standard Deviation 12.86

SECONDARY outcome

Timeframe: Baseline, week 1, 2, 4, and 8

Population: ITT. All randomised subjects.

Efficacy Changes in the concentrations of serum iron (s-iron) from baseline to week 1, 2, 4, and 8.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Change in Concentration of S-iron From Baseline to Week 1, 2, 4, and 8 Week 1	34.8 μg/dL Standard Deviation 99.7	11.1 μg/dL Standard Deviation 71.1
Change in Concentration of S-iron From Baseline to Week 1, 2, 4, and 8 Week 2	11.1 μg/dL Standard Deviation 90.6	12.4 μg/dL Standard Deviation 44.1
Change in Concentration of S-iron From Baseline to Week 1, 2, 4, and 8 Week 4	6.5 μg/dL Standard Deviation 91.2	10.2 μg/dL Standard Deviation 38.3
Change in Concentration of S-iron From Baseline to Week 1, 2, 4, and 8 Week 8	7.1 μg/dL Standard Deviation 91.3	12.4 μg/dL Standard Deviation 41.9

SECONDARY outcome

Timeframe: Baseline, week 1, 2, and 8

Population: ITT. All randomised subjects.

Efficacy Change in fatigue symptoms from baseline to week 1, 2, and 8 was measured by the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale. The Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale consisted of 13 items ranging from 0 (not at all) to 4 (very much), except items #7 and #8 which are reversed scored. The total score range is 0-52. A score of less than 30 indicated severe fatigue, and the higher the score, the better outcome/quality of life (QoL). If more than 50% of the items for a subject at a given visit were missing, the total score was not calculated. Total score was calculated as shown below: Total score= Sum of individual scores x 13 / Number of items answered

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Change in Fatigue Symptoms From Baseline to Week 1, 2, and 8 Week 1	5.04 score on a scale Standard Deviation 8.85	5.01 score on a scale Standard Deviation 7.93
Change in Fatigue Symptoms From Baseline to Week 1, 2, and 8 Week 2	7.29 score on a scale Standard Deviation 9.84	7.63 score on a scale Standard Deviation 9.74
Change in Fatigue Symptoms From Baseline to Week 1, 2, and 8 Week 8	9.13 score on a scale Standard Deviation 11.09	9.07 score on a scale Standard Deviation 11.40

SECONDARY outcome

Timeframe: Baseline

Population: ITT. All randomised subjects.

Pharmacoeconomics The Intervals in Screening for Diabetic Retinopathy (ISDR) questionnaire at the baseline visit assessed the resources used by subjects to receive treatment. Resources used on other trial activities were not included. ISDR responses were summarised using descriptive statistics. The data for this endpoint show the responses at baseline for both treatment groups. The frequency of drug administration between the 2 treatment groups is different, however, (i.e. up to a factor 5 in the iron sucrose treatment group).

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Cost of Public Transport/Taxi And Parking Cost of public transport/taxi	0.0 US dollars ($) Interval 0.0 to 87.0	0.0 US dollars ($) Interval 0.0 to 46.0
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Cost of Public Transport/Taxi And Parking Cost of parking	0.0 US dollars ($) Interval 0.0 to 45.0	0.0 US dollars ($) Interval 0.0 to 25.0

SECONDARY outcome

Timeframe: Baseline

Population: ITT. All randomised subjects. The number of participants analyzed were less the number of participants starting the study since not all participants had data collected for this outcome measure.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=878 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=446 Participants Iron sucrose, administered IV
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Return Journey by Car	19.0 miles Interval 0.0 to 232.0	18.0 miles Interval 0.0 to 240.0

SECONDARY outcome

Timeframe: Baseline

Population: ITT. All randomised subjects.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Time Spent on Visit/Helping on Visit Time spent on visit	2.00 Hours Interval 0.0 to 6.3	2.00 Hours Interval 0.0 to 6.0
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Time Spent on Visit/Helping on Visit Total time spent helping on visit	2.00 Hours Interval 0.0 to 6.3	2.00 Hours Interval 0.0 to 10.5

SECONDARY outcome

Timeframe: Baseline

Population: ITT. All randomised subjects.

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1027 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=511 Participants Iron sucrose, administered IV
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Participants/Others Who Took Time Off Work to Attend Visits In employment, YES	152 Participants	57 Participants
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Participants/Others Who Took Time Off Work to Attend Visits Took time off work to attend, YES	70 Participants	26 Participants
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Participants/Others Who Took Time Off Work to Attend Visits Assistance by others to attend visit, YES	410 Participants	197 Participants
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Participants/Others Who Took Time Off Work to Attend Visits Others took time off work to attend, YES	82 Participants	40 Participants

SECONDARY outcome

Timeframe: Baseline

Population: ITT. All randomised subjects.

Pharmacoeconomics Resources used by the health care staff (per administration), measured by the health care resource use questionnaire. The questionnaire assessed the time used by the health care staff during administration of the investigational product and the administration time (including the observational time). The health care participants included in the evaluation were: investigator, pharmacist, physician, study coordinator, study nurse. The data for this endpoint show the responses at baseline for both treatment groups. The frequency of drug administration between the 2 treatment groups is different (i.e. up to a factor 5 more frequent in the iron sucrose treatment group).

Outcome measures

Outcome measures
Measure	Iron Isomaltoside/Ferric Derisomaltose n=2345 Participants Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=1166 Participants Iron sucrose, administered IV
Health Care Resource Use Questionnaire Time spent per site staff	1.17 hours Interval 0.0 to 20.8	1.00 hours Interval 0.0 to 20.1
Health Care Resource Use Questionnaire Time spent per subject	2.58 hours Interval 0.5 to 20.8	2.33 hours Interval 0.2 to 20.3

Adverse Events

Iron Isomaltoside/Ferric Derisomaltose

Serious events: 83 serious events

Other events: 56 other events

Deaths: 3 deaths

Iron Sucrose

Serious events: 50 serious events

Other events: 34 other events

Deaths: 3 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Iron Isomaltoside/Ferric Derisomaltose n=1019 participants at risk Iron isomaltoside/ferric derisomaltose, administered IV	Iron Sucrose n=506 participants at risk Iron sucrose, administered IV
Infections and infestations Urinary tract infection	2.2% 22/1019 • Number of events 22 • From the time a subjects had signed the informed consent form (CRF) and until they had completed the study, all adverse events (AEs) or serious adverse events (SAEs) were reported in the CRF. The actual study duration was 8 weeks (or shorted if the trial was discontinued). Overall, eligible subjects participated in the trial for approximately 10 weeks (including a 14-day screening period). An AE was described as follows: the nature of the event was described in precise, standard medical terminology (i.e. not necessarily the exact words used by the subject). If known, a specific diagnosis was stated. Furthermore, the Investigator described an AE regarding seriousness, severity, relatedness, and outcome.	2.8% 14/506 • Number of events 14 • From the time a subjects had signed the informed consent form (CRF) and until they had completed the study, all adverse events (AEs) or serious adverse events (SAEs) were reported in the CRF. The actual study duration was 8 weeks (or shorted if the trial was discontinued). Overall, eligible subjects participated in the trial for approximately 10 weeks (including a 14-day screening period). An AE was described as follows: the nature of the event was described in precise, standard medical terminology (i.e. not necessarily the exact words used by the subject). If known, a specific diagnosis was stated. Furthermore, the Investigator described an AE regarding seriousness, severity, relatedness, and outcome.
Vascular disorders Hypertension	1.4% 14/1019 • Number of events 15 • From the time a subjects had signed the informed consent form (CRF) and until they had completed the study, all adverse events (AEs) or serious adverse events (SAEs) were reported in the CRF. The actual study duration was 8 weeks (or shorted if the trial was discontinued). Overall, eligible subjects participated in the trial for approximately 10 weeks (including a 14-day screening period). An AE was described as follows: the nature of the event was described in precise, standard medical terminology (i.e. not necessarily the exact words used by the subject). If known, a specific diagnosis was stated. Furthermore, the Investigator described an AE regarding seriousness, severity, relatedness, and outcome.	2.6% 13/506 • Number of events 18 • From the time a subjects had signed the informed consent form (CRF) and until they had completed the study, all adverse events (AEs) or serious adverse events (SAEs) were reported in the CRF. The actual study duration was 8 weeks (or shorted if the trial was discontinued). Overall, eligible subjects participated in the trial for approximately 10 weeks (including a 14-day screening period). An AE was described as follows: the nature of the event was described in precise, standard medical terminology (i.e. not necessarily the exact words used by the subject). If known, a specific diagnosis was stated. Furthermore, the Investigator described an AE regarding seriousness, severity, relatedness, and outcome.
Metabolism and nutrition disorders Hyperkalaemia	2.2% 22/1019 • Number of events 22 • From the time a subjects had signed the informed consent form (CRF) and until they had completed the study, all adverse events (AEs) or serious adverse events (SAEs) were reported in the CRF. The actual study duration was 8 weeks (or shorted if the trial was discontinued). Overall, eligible subjects participated in the trial for approximately 10 weeks (including a 14-day screening period). An AE was described as follows: the nature of the event was described in precise, standard medical terminology (i.e. not necessarily the exact words used by the subject). If known, a specific diagnosis was stated. Furthermore, the Investigator described an AE regarding seriousness, severity, relatedness, and outcome.	1.8% 9/506 • Number of events 10 • From the time a subjects had signed the informed consent form (CRF) and until they had completed the study, all adverse events (AEs) or serious adverse events (SAEs) were reported in the CRF. The actual study duration was 8 weeks (or shorted if the trial was discontinued). Overall, eligible subjects participated in the trial for approximately 10 weeks (including a 14-day screening period). An AE was described as follows: the nature of the event was described in precise, standard medical terminology (i.e. not necessarily the exact words used by the subject). If known, a specific diagnosis was stated. Furthermore, the Investigator described an AE regarding seriousness, severity, relatedness, and outcome.

Additional Information

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Results disclosure agreements

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