Evaluate safety and efficacy of iron isomaltoside/ferric derisomaltose (Monofer®/Monoferric®) compared with iron sucrose (Venofer®), in subjects diagnosed with IDA.
IDA is highly prevalent condition in subjects with cancer and gastrointestinal diseases such as inflammatory bowel diseases, menstruating or pregnant women, and subjects who have undergone bariatric procedure or surgery. IDA can have a substantial medical and quality of life (QoL) burden. Treatment of subjects diagnosed with IDA includes controlling the bleeding and replenishing lost iron. This study was designed to evaluate the safety and efficacy of iron isomaltoside/ferric derisomaltose compared with iron sucrose in subjects diagnosed with IDA. In a subfraction of 35 subjects treated with iron isomaltoside/ferric derisomaltose, ECG and iron will be frequently measured. The study subjects received either a single intravenous (IV) dose of iron isomaltoside/ferric derisomaltose (1000 mg at baseline) or iron sucrose (200 mg IV injections at baseline and repeated according to standard practice or physician choice up to a maximum of five times within the first two weeks starting at baseline; a cumulative dose of 1000 mg was recommended). The study subjects were monitored for up to 8 weeks from baseline.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
1,512
Iron isomaltoside/ferric derisomaltose (Monofer®/Monoferric®; 100 mg/mL) was the test product in this trial. The dose of iron isomaltoside/ferric derisomaltose for the individual subject was set to 1000 mg. The dose was diluted in 100 mL 0.9 % sodium chloride (100 mL bags) and administered as a single IV infusion over approximately 20 minutes.
Iron sucrose (Venofer®; 20 mg elemental iron/mL) was the comparator in this trial. Iron sucrose was administered as 200 mg undiluted IV injections over approximately 2-5 minutes and repeated according to standard practice or physician choice up to a maximum of five times within the first two weeks starting at baseline. A cumulative dose of 1000 mg was recommended.
Change in Hemoglobin (Hb) From Baseline to Week 8
Efficacy Evaluate the effect on the hemoglobin (Hb) level following treatment with iron isomaltoside/ferric derisomaltose vs iron sucrose in subjects with 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 IDA, when oral iron preparations were ineffective or could not be used or in whom the screening Hb measurement in Investigators' opinion were sufficiently low to require rapid repletion of iron stores.
Time frame: Baseline to week 8
Incidence of Protocol-defined Serious or Severe Hypersensitivity Reactions
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.
Time frame: Baseline to week 8
Composite Cardiovascular Adverse Events (AEs)
Safety Results show the composite cardiovascular adverse events (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.
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Pharmacosmos Investigational Site
Birmingham, Alabama, United States
Pharmacosmos Investigational Site
Dothan, Alabama, United States
Pharmacosmos Investigational Site
Guntersville, Alabama, United States
Pharmacosmos Investigational Site
Phoenix, Arizona, United States
Pharmacosmos Investigational Site
Little Rock, Arkansas, United States
Pharmacosmos Investigational Site
Little Rock, Arkansas, United States
Pharmacosmos Investigational Site
Foothill Ranch, California, United States
Pharmacosmos Investigational Site 1
La Mesa, California, United States
Pharmacosmos Investigational Site 2
La Mesa, California, United States
Pharmacosmos Investigational Site
Long Beach, California, United States
...and 104 more locations
Time frame: Baseline, week 1, 2, and 8
Time to First Composite Cardiovascular Safety AE
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.
Time frame: Baseline, week 1, 2, 4, and 8
S-phosphate <2 mg/dL at Any Time From Baseline to Week 1, 2, 4, and 8
Safety Results show the number of subjects who had s-phosphate \<2 mg/dL at any time from baseline to week 1, 2, 4, or 8.
Time frame: Baseline, week 1, 2, 4, and 8
Hb Concentration Increase of ≥2 g/dL From Baseline to Week 1, 2, 4, and 8
Efficacy Results show responders to the treatment. A subject was considered a Hb responder to a certain week if an increase in Hb of at least 2 g/dL from baseline to the week in question was observed (week 1, 2, 4, and 8).
Time frame: Baseline, week 1, 2, 4, and 8
Time to Change in Hb Concentration ≥2 g/dL
Efficacy Time to change in Hb concentration ≥2 g/dL. Subjects who achieved Hb concentration increase of ≥2 g/dL (from baseline to week 1, 2, 4, or 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.
Time frame: Baseline, week 1, 2, 4, and 8
Hb Concentration of >12 g/dL at Any Time From Week 1 to Week 8
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.
Time frame: Week 1 to week 8
Hb Concentration Increase of ≥2 g/dL at Any Time From Week 1 to Week 8
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.
Time frame: Week 1 to week 8
S-Ferritin Concentration of ≥100 ng/mL and Transferrin Saturation (TSAT) of 20-50% at Any Time From Week 1 to Week 8
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.
Time frame: Week 1 to week 8
Change in Hb Concentration From Baseline to Week 1, 2, and 4
Efficacy Change in Hb concentration from baseline to week 1, 2, and 4.
Time frame: Baseline, week 1, 2, and 4
Change in S-ferritin Concentration From Baseline to Weeks 1, 2, 4, and 8
Efficacy Change in s-ferritin concentration from baseline to weeks 1, 2, 4, and 8.
Time frame: Baseline, week 1, 2, 4, and 8
Change in Transferrin Saturation (TSAT) From Baseline to Week 1, 2, 4, and 8
Efficacy Changes in transferrin saturation (TSAT) from baseline to week 1, 2, 4, and 8. TSAT is the value of serum iron divided by the total iron-binding capacity and the unit is %, which referrers to % of iron-binding sites of transferrin being occupied by iron.
Time frame: Baseline, week 1, 2, 4, and 8
Change in Concentrations of Serum Iron (S-iron) From Baseline to Week 1, 2, 4, and 8
Efficacy Changes in the concentrations of serum iron (s-iron) from baseline to week 1, 2, 4, and 8.
Time frame: Baseline, week 1, 2, 4, and 8
Change in Fatigue Symptoms From Baseline to Week 1, 2, and 8
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
Time frame: Baseline, week 1, 2, and 8
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Cost of Public Transport/Taxi And Parking
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).
Time frame: Baseline
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Return Journey by Car
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).
Time frame: Baseline
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Time Spent on Visit/Helping on Visit
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).
Time frame: Baseline
Intervals in Screening for Diabetic Retinopathy (ISDR) Questionnaire, Participants/Others Who Took Time Off Work to Attend Visits
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).
Time frame: Baseline
Health Care Resource Use Questionnaire
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 treatment groups is different (i.e. up to a factor 5 more frequent in the iron sucrose treatment group).
Time frame: Baseline