Trial Outcomes & Findings for Intravenous Iron Supplement for Iron Deficiency in Cardiac Transplant Recipients (NCT NCT03662789)

NCT ID: NCT03662789

Last Updated: 2021-05-25

Results Overview

The primary endpoint will be the baseline-adjusted between-group difference in peak oxygen consumption as measured on a treadmill exercise test

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 102 participants
• Primary outcome timeframe: 6 months after intervention
• Results posted on: 2021-05-25

Participant Flow

Participant milestones

Measure	Iron Isomaltoside 1000 The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Overall Study STARTED	52	50
Overall Study COMPLETED	47	43
Overall Study NOT COMPLETED	5	7

Reasons for withdrawal

Measure	Iron Isomaltoside 1000 The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Overall Study Lost to Follow-up	1	2
Overall Study Could not perform treadmill test	4	4
Overall Study Death	0	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Iron Isomaltoside 1000 n=52 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=50 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion	Total n=102 Participants Total of all reporting groups
Sex: Female, Male Female	18 Participants n=52 Participants	19 Participants n=50 Participants	37 Participants n=102 Participants
Age, Categorical <=18 years	0 Participants n=52 Participants	0 Participants n=50 Participants	0 Participants n=102 Participants
Age, Categorical Between 18 and 65 years	34 Participants n=52 Participants	39 Participants n=50 Participants	73 Participants n=102 Participants
Age, Categorical >=65 years	18 Participants n=52 Participants	11 Participants n=50 Participants	29 Participants n=102 Participants
Age, Continuous	55 years STANDARD_DEVIATION 15 • n=52 Participants	55 years STANDARD_DEVIATION 14 • n=50 Participants	55 years STANDARD_DEVIATION 14 • n=102 Participants
Sex: Female, Male Male	34 Participants n=52 Participants	31 Participants n=50 Participants	65 Participants n=102 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment Norway	52 participants n=52 Participants	50 participants n=50 Participants	102 participants n=102 Participants
Peak oxygen consumption	24.3 ml/kg/min STANDARD_DEVIATION 7.3 • n=52 Participants	22.3 ml/kg/min STANDARD_DEVIATION 6.0 • n=50 Participants	23.4 ml/kg/min STANDARD_DEVIATION 6.8 • n=102 Participants

PRIMARY outcome

Timeframe: 6 months after intervention

The primary endpoint will be the baseline-adjusted between-group difference in peak oxygen consumption as measured on a treadmill exercise test

Outcome measures

Measure	Iron Isomaltoside 1000 n=47 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=43 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Peak Oxygen Consumption	23.9 ml/kg/min Standard Deviation 6.6	22.0 ml/kg/min Standard Deviation 6.1

SECONDARY outcome

Timeframe: 6 months after intervention

The number of patients with absolute or functional iron deficiency

Outcome measures

Measure	Iron Isomaltoside 1000 n=47 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=43 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Iron Deficiency	7 Participants	40 Participants

SECONDARY outcome

Timeframe: 6 months after intervention

Baseline-adjusted muscle strength as measured by a hand-grip dynamometer

Outcome measures

Measure	Iron Isomaltoside 1000 n=47 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=43 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Muscle Strength	40 kg Standard Deviation 13	38 kg Standard Deviation 12

SECONDARY outcome

Timeframe: 6 months after intervention

Baseline-adjusted quality of life as assessed with the 36-item short form survey (SF-36), which measures each of the following 8 health domains: 1= general health, 2= physical function, 3= role physical, 4= bodily pain, 5= vitality, 6= social function, 7= role emotional, 8= mental health. Total score for each domain are scaled 0 (minimum) to 100 (maximum), where higher scores represented higher level of functioning. Two norm-based sum scores, the physical and the mental component summaries with a mean of 50±10, were generated from the eight scale scores using a T-score transformation. Higher scores represented higher level of functioning.

Outcome measures

Measure	Iron Isomaltoside 1000 n=47 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=43 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Health Related Quality of Life: SF-36, Physical Component Summary (PCS)	49 t-score Interval 42.0 to 55.0	45 t-score Interval 37.0 to 53.0

SECONDARY outcome

Timeframe: 6 months after intervention

The between-group difference in baseline-adjusted NT-proBNP

Outcome measures

Measure	Iron Isomaltoside 1000 n=47 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=43 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
N-terminal Pro-B-type Natriuretic Peptide (NT-proBNP)	421 ng/l Interval 134.0 to 807.0	349 ng/l Interval 237.0 to 706.0

SECONDARY outcome

Timeframe: 6 months after intervention

The between-group difference in baseline-adjusted TnT

Outcome measures

Measure	Iron Isomaltoside 1000 n=47 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=43 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Cardiac Troponin T (TnT)	13.0 ng/l Interval 8.0 to 25.8	15.0 ng/l Interval 8.0 to 24.3

SECONDARY outcome

Timeframe: 6 months after intervention

Baseline-adjusted quality of life as assessed with the 36-item short form survey (SF-36), which measures each of the following 8 health domains: 1= general health, 2= physical function, 3= role physical, 4= bodily pain, 5= vitality, 6= social function, 7= role emotional, 8= mental health. Total score for each domain are scaled 0 (minimum) to 100 (maximum), where higher scores represented higher level of functioning. Two norm-based sum scores, the physical and the mental component summaries with a mean of 50±10, were generated from the eight scale scores using a T-score transformation. Higher scores represented higher level of functioning.

Outcome measures

Measure	Iron Isomaltoside 1000 n=47 Participants The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=43 Participants Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Health Related Quality of Life: SF-36, Mental Component Summary (MCS)	56 t-score Interval 49.0 to 60.0	53 t-score Interval 48.0 to 58.0

Adverse Events

Iron Isomaltoside 1000

Serious events: 5 serious events

Other events: 22 other events

Deaths: 0 deaths

Placebo

Serious events: 12 serious events

Other events: 18 other events

Deaths: 1 deaths

Serious adverse events

Measure	Iron Isomaltoside 1000 n=52 participants at risk The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=50 participants at risk Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Infections and infestations Infection	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	16.0% 8/50 • Number of events 8 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Musculoskeletal and connective tissue disorders Fracture	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Hepatobiliary disorders Cholelitiasis	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Cardiac disorders ST-elevation myocardial infarction	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Respiratory, thoracic and mediastinal disorders Dyspnea	3.8% 2/52 • Number of events 2 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Endocrine disorders Hyperthyreosis	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Cardiac disorders Heart failure	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Cardiac disorders Deceased	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.

Other adverse events

Measure	Iron Isomaltoside 1000 n=52 participants at risk The active drug, iron isomaltoside 1000 will be administered as a single, intravenous infusion of 20 mg/kg body weight (rounded off to the nearest 100 mg) dissolved in 100 ml NaCl as recommended by the drug manufacturer ("on-label" treatment). Iron Isomaltoside 1000: Intravenous infusion	Placebo n=50 participants at risk Patients allocated to placebo will receive an intravenous infusion of 100 ml NaCl 0.9% Placebo: NaCl 0,9%: Intravenous infusion
Blood and lymphatic system disorders Bleeding	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	8.0% 4/50 • Number of events 4 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Infections and infestations Infection	23.1% 12/52 • Number of events 12 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	10.0% 5/50 • Number of events 5 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Musculoskeletal and connective tissue disorders Gout	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	4.0% 2/50 • Number of events 2 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Gastrointestinal disorders Hernia	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Skin and subcutaneous tissue disorders Hair loss	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	6.0% 3/50 • Number of events 3 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Endocrine disorders Diabetes	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Musculoskeletal and connective tissue disorders Fatigue	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Gastrointestinal disorders Reflux	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Skin and subcutaneous tissue disorders Spotty facial discoloration	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Vascular disorders Syncope	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Hepatobiliary disorders Elevated liver enzymes	1.9% 1/52 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Skin and subcutaneous tissue disorders Generalized Rash	3.8% 2/52 • Number of events 2 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	0.00% 0/50 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Skin and subcutaneous tissue disorders Local rash	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.
Gastrointestinal disorders Pancreatitis	0.00% 0/52 • 6 months Adverse events were registered at the follow up at 6 months for all participants.	2.0% 1/50 • Number of events 1 • 6 months Adverse events were registered at the follow up at 6 months for all participants.

Additional Information

Dr. Lars Gullestad

Oslo University Hospital

Phone: 0047 97644772

Email: lars.gullestad@medisin.uio.no

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place