Trial Outcomes & Findings for Ferric Carboxymaltose (FCM) Assessment in Subjects With Iron Deficiency Anaemia and Non-dialysis-dependent Chronic Kidney Disease (NDD-CKD) (NCT NCT00994318)

Last Updated: 2014-05-20

Results Overview

Endpoint reported number of participants with/without events and was reached: * First time of initiation of additional or alternative anaemia management, * First time the subject reached the Hb trigger. 3 primary comparisons using a hierarchical step-down procedure on the log-rank test to preserve an alpha level of 0.05, performed in the following order: 1. FCM (high ferritin target) compared with oral iron. 2. FCM (high ferritin target) compared with FCM (low ferritin target). 3. FCM (low ferritin target) compared with oral iron. Sensitivity analyses of the primary endpoint were performed using the following alternative definitions of time to initiation of additional or alternative anaemia management: 1. Without taking into account the Hb trigger. 2. Taking into account the Hb trigger based on local laboratory data, instead of central laboratory data. 3. Taking into account the Hb trigger based on subjects with a complete set of Hb values from the central laboratory.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

626 participants

Primary outcome timeframe

Up to 1 year after baseline

Results posted on

2014-05-20

Participant Flow

Participant milestones

Participant milestones
Measure	FCM (High Ferritin Target) Ferric carboxymaltose (FCM) (Ferinject / Injectafer) targeting ferritin level of 400 - 600 mcg/L	FCM (Low Ferritin Target) Ferric carboxymaltose (FCM) (Ferinject / Injectafer) targeting ferritin level of 100 - 200 mcg/L	Oral Iron Ferrous sulphate 100 mg iron twice daily, continuous
Overall Study STARTED	155	154	317
Overall Study COMPLETED	133	136	250
Overall Study NOT COMPLETED	22	18	67

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ferric Carboxymaltose (FCM) Assessment in Subjects With Iron Deficiency Anaemia and Non-dialysis-dependent Chronic Kidney Disease (NDD-CKD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	FCM (High Ferritin Target n=153 Participants Ferric carboxymaltose (FCM) (Ferinject / Injectafer) targeting ferritin level of 400 - 600 mcg/L	FCM (Low Ferritin Target) n=152 Participants Ferric carboxymaltose (FCM) (Ferinject / Injectafer) targeting ferritin level of 100 - 200 mcg/L	Oral Iron n=308 Participants Ferrous sulphate 100 mg iron twice daily, continuous	Total n=613 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	41 Participants n=5 Participants	49 Participants n=7 Participants	92 Participants n=5 Participants	182 Participants n=4 Participants
Age, Categorical >=65 years	112 Participants n=5 Participants	103 Participants n=7 Participants	216 Participants n=5 Participants	431 Participants n=4 Participants
Age, Continuous	69.46 years STANDARD_DEVIATION 12.643 • n=5 Participants	68.19 years STANDARD_DEVIATION 13.264 • n=7 Participants	69.31 years STANDARD_DEVIATION 13.404 • n=5 Participants	69.07 years STANDARD_DEVIATION 13.172 • n=4 Participants
Sex: Female, Male Female	91 Participants n=5 Participants	98 Participants n=7 Participants	192 Participants n=5 Participants	381 Participants n=4 Participants
Sex: Female, Male Male	62 Participants n=5 Participants	54 Participants n=7 Participants	116 Participants n=5 Participants	232 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	3 Participants n=7 Participants	9 Participants n=5 Participants	14 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants	14 Participants n=4 Participants
Race (NIH/OMB) White	149 Participants n=5 Participants	144 Participants n=7 Participants	291 Participants n=5 Participants	584 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Region of Enrollment United States	4 participants n=5 Participants	3 participants n=7 Participants	7 participants n=5 Participants	14 participants n=4 Participants
Region of Enrollment Portugal	2 participants n=5 Participants	2 participants n=7 Participants	5 participants n=5 Participants	9 participants n=4 Participants
Region of Enrollment Greece	28 participants n=5 Participants	28 participants n=7 Participants	55 participants n=5 Participants	111 participants n=4 Participants
Region of Enrollment Turkey	7 participants n=5 Participants	7 participants n=7 Participants	15 participants n=5 Participants	29 participants n=4 Participants
Region of Enrollment Austria	2 participants n=5 Participants	2 participants n=7 Participants	4 participants n=5 Participants	8 participants n=4 Participants
Region of Enrollment United Kingdom	17 participants n=5 Participants	18 participants n=7 Participants	36 participants n=5 Participants	71 participants n=4 Participants
Region of Enrollment Italy	10 participants n=5 Participants	10 participants n=7 Participants	19 participants n=5 Participants	39 participants n=4 Participants
Region of Enrollment France	1 participants n=5 Participants	0 participants n=7 Participants	3 participants n=5 Participants	4 participants n=4 Participants
Region of Enrollment Czech Republic	16 participants n=5 Participants	15 participants n=7 Participants	31 participants n=5 Participants	62 participants n=4 Participants
Region of Enrollment Belgium	3 participants n=5 Participants	3 participants n=7 Participants	4 participants n=5 Participants	10 participants n=4 Participants
Region of Enrollment Poland	11 participants n=5 Participants	11 participants n=7 Participants	20 participants n=5 Participants	42 participants n=4 Participants
Region of Enrollment Romania	2 participants n=5 Participants	2 participants n=7 Participants	4 participants n=5 Participants	8 participants n=4 Participants
Region of Enrollment Australia	15 participants n=5 Participants	16 participants n=7 Participants	31 participants n=5 Participants	62 participants n=4 Participants
Region of Enrollment Denmark	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants	4 participants n=4 Participants
Region of Enrollment Germany	26 participants n=5 Participants	25 participants n=7 Participants	49 participants n=5 Participants	100 participants n=4 Participants
Region of Enrollment Netherlands	4 participants n=5 Participants	4 participants n=7 Participants	8 participants n=5 Participants	16 participants n=4 Participants
Region of Enrollment Norway	0 participants n=5 Participants	1 participants n=7 Participants	4 participants n=5 Participants	5 participants n=4 Participants
Region of Enrollment Sweden	0 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants	1 participants n=4 Participants
Region of Enrollment Spain	4 participants n=5 Participants	4 participants n=7 Participants	10 participants n=5 Participants	18 participants n=4 Participants

PRIMARY outcome

Timeframe: Up to 1 year after baseline

Population: The Full Analysis Set (FAS) was used for the primary endpoint analysis, which consisted of all subjects randomised to treatment, received at least 1 dose of study treatment or, according to the protocol, were not treated due to ferritin value \<100 mcg/L, and attended at least 1 post-baseline visit with at least 1 non-missing assessment available.

Outcome measures

Outcome measures
Measure	FCM (High Ferritin Target) n=153 Participants Ferric carboxymaltose (FCM) (Ferinject / Injectafer), targeting ferritin level of 400-600 mcg/L	FCM (Low Ferritin Level) n=152 Participants Ferric carboxymaltose (FCM) (Ferinject / Injectafer), targeting ferritin level of 100-200 mcg/L	Oral Iron n=308 Participants Ferrous sulphate 100 mg iron twice daily, continuous
Kaplan-Meier Survival Analysis for Time to Other Anemia Therapy or Hb Trigger Number of patients with events	36 participants 7.65	49 participants 9.47 • Interval 0.45 to 1.05	98 participants 7.71 • Interval 0.44 to 0.95
Kaplan-Meier Survival Analysis for Time to Other Anemia Therapy or Hb Trigger Number of patients without events	117 participants	103 participants	210 participants

Adverse Events

FCM (High Ferritin Target)

Serious events: 39 serious events

Other events: 126 other events

Deaths: 0 deaths

FCM (Low Ferritin Target)

Serious events: 36 serious events

Other events: 129 other events

Deaths: 0 deaths

Oral Iron

Serious events: 59 serious events

Other events: 255 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Medical Information

Vifor Pharma

Phone: 41 58 851 8222

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Investigators may not present or publish partial or complete study results individually. Any manuscript or abstract proposed by the Investigators must be reviewed and approved in writing by Vifor Pharma before submission for publication. Names of all Investigators participating in the study will be included in the publication.
Publication restrictions are in place

Restriction type: OTHER

Measure	FCM (High Ferritin Target) n=154 participants at risk Ferric carboxymaltose (FCM) (Ferinject / Injectafer) targeting ferritin level of 400 - 600 mcg/L	FCM (Low Ferritin Target) n=150 participants at risk Ferric carboxymaltose (FCM) (Ferinject / Injectafer) targeting ferritin level of 100 - 200 mcg/L	Oral Iron n=312 participants at risk Ferrous sulphate 100 mg iron twice daily, continuous
Gastrointestinal disorders Diarrhoea	9.7% 15/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	7.3% 11/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	14.4% 45/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Gastrointestinal disorders Constipation	1.3% 2/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	3.3% 5/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	11.9% 37/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Gastrointestinal disorders Nausea	5.8% 9/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	4.7% 7/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	4.8% 15/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Gastrointestinal disorders Dyspepsia	1.3% 2/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	2.0% 3/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	5.4% 17/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Infections and infestations Urinary Tract Infection	11.7% 18/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	6.7% 10/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	5.4% 17/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Infections and infestations Nasopharyngitis	8.4% 13/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	6.7% 10/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	5.1% 16/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Infections and infestations Influenza	2.6% 4/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	5.3% 8/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	2.2% 7/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
General disorders Oedema Peripheral	13.6% 21/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	14.0% 21/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	9.3% 29/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Back Pain	9.7% 15/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	8.0% 12/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	3.5% 11/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Arthralgia	6.5% 10/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	4.7% 7/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	4.8% 15/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Pain in Extremity	1.3% 2/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	5.3% 8/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	4.8% 15/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Vascular disorders Hypertension	13.6% 21/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	9.3% 14/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	10.3% 32/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Vascular disorders Hypotension	5.2% 8/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	2.7% 4/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	1.6% 5/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Dyspnoea	4.5% 7/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	7.3% 11/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	3.5% 11/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Nervous system disorders Dizziness	5.8% 9/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	5.3% 8/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	2.2% 7/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Nervous system disorders Headache	3.9% 6/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	6.7% 10/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	2.2% 7/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.
Blood and lymphatic system disorders Anaemia	4.5% 7/154 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	5.3% 8/150 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.	3.2% 10/312 • Adverse Event data was collected for 56 weeks after baseline. The Number of Patients at Risk is the Safety Set which consists of all randomised subjects who received at least 1 dose of study drug.