Trial Outcomes & Findings for Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA (NCT NCT03523117)
NCT ID: NCT03523117
Last Updated: 2022-06-22
Results Overview
Change in hemoglobin g/dL from baseline to day 35 will be analyzed using parametric analysis of covariance (ANCOVA). The model will include terms for the randomization strata (hemoglobin and age categories), baseline hemoglobin, as well as treatment group. Baseline hemoglobin will be defined as the last hemoglobin obtained before randomization.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
79 participants
Primary outcome timeframe
Baseline to day 35
Results posted on
2022-06-22
Participant Flow
Phase 3, multicenter, multinational, randomized, active-controlled study that compared the efficacy and safety of FCM to oral iron in pediatric participants with IDA and a documented history of an inadequate response to oral iron at least 8 weeks (56 days) prior to randomization. Participants who satisfied the inclusion requirements and no exclusionary criteria were eligible to participate in this study and enter into a screening phase to confirm eligibility.
Participant milestones
| Measure |
Ferric Caroboxymaltose
Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
Ferric carboxymaltose: Intravenous iron
|
Oral Ferrous Sulfate
Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants \<12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to \<4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to \<12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.
Ferrous Sulfate: oral iron therapy
|
|---|---|---|
|
Overall Study
STARTED
|
40
|
39
|
|
Overall Study
COMPLETED
|
39
|
37
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA
Baseline characteristics by cohort
| Measure |
Ferric Caroboxymaltose
n=40 Participants
Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
Ferric carboxymaltose: Intravenous iron
|
Oral Ferrous Sulfate
n=39 Participants
Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants \<12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to \<4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to \<12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.
Ferrous Sulfate: oral iron therapy
|
Total
n=79 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
|
12.5 years
STANDARD_DEVIATION .5 • n=99 Participants
|
12.8 years
STANDARD_DEVIATION .4 • n=107 Participants
|
12.6 years
STANDARD_DEVIATION .5 • n=206 Participants
|
|
Sex: Female, Male
Female
|
33 Participants
n=99 Participants
|
30 Participants
n=107 Participants
|
63 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
35 Participants
n=99 Participants
|
34 Participants
n=107 Participants
|
69 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline to day 35Population: Intent to Treat (ITT)
Change in hemoglobin g/dL from baseline to day 35 will be analyzed using parametric analysis of covariance (ANCOVA). The model will include terms for the randomization strata (hemoglobin and age categories), baseline hemoglobin, as well as treatment group. Baseline hemoglobin will be defined as the last hemoglobin obtained before randomization.
Outcome measures
| Measure |
Ferric Caroboxymaltose
n=40 Participants
Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
Ferric carboxymaltose: Intravenous iron
|
Oral Ferrous Sulfate
n=39 Participants
Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants \<12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to \<4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to \<12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.
Ferrous Sulfate: oral iron therapy
|
|---|---|---|
|
Change in Hemoglobin g/dL
|
2.22 g/dL
Interval 1.69 to 2.75
|
1.92 g/dL
Interval 1.43 to 2.41
|
SECONDARY outcome
Timeframe: Baseline to day 35Population: intent to treat (ITT) all randomized
Change in ferritin µg/L from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate.
Outcome measures
| Measure |
Ferric Caroboxymaltose
n=40 Participants
Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
Ferric carboxymaltose: Intravenous iron
|
Oral Ferrous Sulfate
n=39 Participants
Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants \<12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to \<4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to \<12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.
Ferrous Sulfate: oral iron therapy
|
|---|---|---|
|
Change in Ferritin µg/L From Baseline to Day 35
|
2.22 µg/L
Interval 1.69 to 2.75
|
1.92 µg/L
Interval 1.43 to 2.41
|
SECONDARY outcome
Timeframe: Baseline to day 35Population: Intent to treatment
Change in TSAT (%) from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate
Outcome measures
| Measure |
Ferric Caroboxymaltose
n=40 Participants
Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
Ferric carboxymaltose: Intravenous iron
|
Oral Ferrous Sulfate
n=39 Participants
Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants \<12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to \<4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to \<12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.
Ferrous Sulfate: oral iron therapy
|
|---|---|---|
|
Change in TSAT (%) From Baseline to Day 35
|
24.30 percentage
Interval 19.19 to 29.41
|
8.66 percentage
Interval 3.7 to 13.63
|
SECONDARY outcome
Timeframe: Baseline to day 35Population: Intent to treat
Change in reticulocyte hemoglobin (picograms) content from baseline to day 35 was analyzed using a mixed model repeated. The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a reticulocyte hemoglobin content.
Outcome measures
| Measure |
Ferric Caroboxymaltose
n=40 Participants
Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
Ferric carboxymaltose: Intravenous iron
|
Oral Ferrous Sulfate
n=39 Participants
Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants \<12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to \<4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to \<12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.
Ferrous Sulfate: oral iron therapy
|
|---|---|---|
|
Change in Reticulocyte Hemoglobin (Picograms) Content From Baseline to Day 35
|
6.95 picograms
Interval 6.21 to 7.7
|
4.90 picograms
Interval 4.15 to 5.65
|
Adverse Events
Ferric Caroboxymaltose
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Oral Ferrous Sulfate
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place