Trial Outcomes & Findings for Evaluate the Safety and Efficacy of Ferric Maltol Oral Suspension vs. Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, With a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years (NCT NCT05126901)
NCT ID: NCT05126901
Last Updated: 2025-11-10
Results Overview
The change in Hb concentration from baseline to Week 12 is summarized based on the mITT Population for each treatment group using descriptive statistics summarized by mean, standard deviation, median, and range (minimum and maximum). The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.
COMPLETED
PHASE3
65 participants
From baseline to week 12.
2025-11-10
Participant Flow
This was a multicenter study with a total of 23 clinical sites located in the United States(including Puerto Rico) and United Kingdom. A total of 65 patients were enrolled in the study.
All eligible subjects aged 1 month to less than 2 years entered a Pre-assignment phase, 1-day Pharmacokinetic assessment day following a single dose of ferric maltol oral suspension.
Participant milestones
| Measure |
Ferric Maltol Assigned
12 weeks open-label Treatment Period for ferric maltol children aged 1 month to \<2 years
|
Ferric Maltol Randomized
Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days.
Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12.
Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose was used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2
\- 17 yrs were dosed 6 mg/kg to the maximum of 4 ml BID.
|
Ferrous Sulfate Randomized
Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
|
|---|---|---|---|
|
Overall Study
STARTED
|
4
|
31
|
30
|
|
Overall Study
COMPLETED
|
3
|
28
|
25
|
|
Overall Study
NOT COMPLETED
|
1
|
3
|
5
|
Reasons for withdrawal
| Measure |
Ferric Maltol Assigned
12 weeks open-label Treatment Period for ferric maltol children aged 1 month to \<2 years
|
Ferric Maltol Randomized
Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days.
Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12.
Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose was used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2
\- 17 yrs were dosed 6 mg/kg to the maximum of 4 ml BID.
|
Ferrous Sulfate Randomized
Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
|
|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
4
|
|
Overall Study
Adverse Event
|
0
|
0
|
1
|
|
Overall Study
Lost to Follow-up
|
0
|
2
|
0
|
Baseline Characteristics
Evaluate the Safety and Efficacy of Ferric Maltol Oral Suspension vs. Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, With a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years
Baseline characteristics by cohort
| Measure |
Ferric Maltol Assigned
n=4 Participants
12 weeks open-label Treatment Period for ferric maltol children aged 1 month to \<2 years
|
Ferric Maltol Randomized
n=31 Participants
Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days.
Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12.
Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose was used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2
\- 17 yrs were dosed 6 mg/kg to the maximum of 4 ml BID.
|
Ferrous Sulfate Randomized
n=30 Participants
Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
|
Total
n=65 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Customized
Preterm newborn infants (gestational age < 37 wks)
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Age, Customized
Newborns (0-27 days)
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Age, Customized
Infants and toddlers (28 days-23 months)
|
4 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
4 Participants
n=28 Participants
|
|
Age, Customized
Children (2-11 years)
|
0 Participants
n=5 Participants
|
10 Participants
n=20 Participants
|
11 Participants
n=40 Participants
|
21 Participants
n=28 Participants
|
|
Age, Customized
Adolescents (12-17 years)
|
0 Participants
n=5 Participants
|
21 Participants
n=20 Participants
|
19 Participants
n=40 Participants
|
40 Participants
n=28 Participants
|
|
Age, Customized
Adults (18-64 years)
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Age, Customized
From 65-84 years
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Age, Customized
85 years and over
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
23 Participants
n=20 Participants
|
22 Participants
n=40 Participants
|
48 Participants
n=28 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
8 Participants
n=20 Participants
|
8 Participants
n=40 Participants
|
17 Participants
n=28 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
13 Participants
n=20 Participants
|
12 Participants
n=40 Participants
|
27 Participants
n=28 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
18 Participants
n=20 Participants
|
18 Participants
n=40 Participants
|
37 Participants
n=28 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
1 Participants
n=28 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
3 Participants
n=20 Participants
|
3 Participants
n=40 Participants
|
7 Participants
n=28 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
9 Participants
n=20 Participants
|
11 Participants
n=40 Participants
|
20 Participants
n=28 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
18 Participants
n=20 Participants
|
16 Participants
n=40 Participants
|
35 Participants
n=28 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
1 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
3 Participants
n=28 Participants
|
PRIMARY outcome
Timeframe: From baseline to week 12.Population: All patients that finalized Day 84 measurement were reported and analyzed. All patients with a missing Day 84 measurement were imputed using a last observation carried forward approach in which the last available post-baseline measurement was used. Post-baseline measurement was missing in 5 patients in the 2. group (mITT Ferric Maltol) , and 6 patients in the 3. group (mITT Ferrous Sulfate).
The change in Hb concentration from baseline to Week 12 is summarized based on the mITT Population for each treatment group using descriptive statistics summarized by mean, standard deviation, median, and range (minimum and maximum). The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=24 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=26 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Change in Hemoglobin Concentration
|
11.5 g/L
Standard Deviation 13.97
|
—
|
—
|
—
|
17.7 g/L
Standard Deviation 13.61
|
12.5 g/L
Standard Deviation 13.89
|
SECONDARY outcome
Timeframe: From baseline to week 12.Population: All patients that finalized Day 84 measurement were reported and analyzed. All patients with a missing Day 84 measurement were imputed using a last observation carried forward approach in which the last available post-baseline measurement was used. Post-baseline measurement was missing in 2 patients in the 3. group (mITT Ferrous Sulfate).
Changes in ferritin concentration from baseline to week 12 summarised based on the mITT Population for each treatment group. The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=28 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=31 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Changes in Ferritin Concentration
|
20.6 μg/L
Standard Deviation 30.97
|
—
|
—
|
—
|
6.3 μg/L
Standard Deviation 8.74
|
8.1 μg/L
Standard Deviation 11.06
|
SECONDARY outcome
Timeframe: From baseline to week 12.Population: All patients that finalized Day 84 measurement were reported and analyzed. All patients with a missing Day 84 measurement were imputed using a last observation carried forward approach in which the last available post-baseline measurement was used. Post-baseline measurement was missing in 2 patients in the 3. group (mITT Ferrous Sulfate).
Change in iron concentration from baseline to week 12 summarized based on the mITT Population for each treatment group. The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=28 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=31 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Change in Iron Concentration
|
3.64 μmol/L
Standard Deviation 8.911
|
—
|
—
|
—
|
1.07 μmol/L
Standard Deviation 0.577
|
5.77 μmol/L
Standard Deviation 8.518
|
SECONDARY outcome
Timeframe: From baseline to week 12.Population: All patients that finalized Day 84 measurement were reported and analyzed. All patients with a missing Day 84 measurement were imputed using a last observation carried forward approach in which the last available post-baseline measurement was used. Post-baseline measurement was missing in 1 patient in the 1. group (mITT Ferric Maltol Assigned) and 2 patients in the 3. group (mITT Ferrous Sulfate).
Change from baseline to Day 84 summarized based on the mITT Population for each treatment group. The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=28 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=2 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=31 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Change in the Percentage of Transferrin Saturation
|
6.5 Percentage of Transferrin Saturation
Standard Deviation 12.39
|
—
|
—
|
—
|
2.0 Percentage of Transferrin Saturation
Standard Deviation 0.00
|
7.7 Percentage of Transferrin Saturation
Standard Deviation 10.14
|
SECONDARY outcome
Timeframe: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).Population: A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.
Measured maximum plasma concentration (Cmax) (ng/mL) for plasma maltol glucuronide after a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose. Because of the limitations of PK sampling in the pediatric patient population, data from multiple patients in the same treatment and age group had to be combined, using the naïve pooled approach, to achieve a complete set of observations over time for PK analysis. The data did not support PK parameters generation using noncompartmental analysis (NCA) without combining patients. Using validated PK software WinNonlin, the 'sparse sampling' option was utilized to treat the data as one virtual subject and thus the resulting data did not support reliable error estimates of the PK parameters.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Cmax for Plasma Maltol Glucuronide
|
6420 ng/mL
NA
|
4250 ng/mL
NA
|
5940 ng/mL
NA
|
8160 ng/mL
NA
|
4350 ng/mL
NA
|
6810 ng/mL
NA
|
SECONDARY outcome
Timeframe: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).Population: A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.
Time to maximum plasma concentration (Tmax) (h) for plasma maltol glucuronide after a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose. Because of the limitations of PK sampling in the pediatric patient population, data from multiple patients in the same treatment and age group had to be combined, using the naïve pooled approach, to achieve a complete set of observations over time for PK analysis. The data did not support PK parameters generation using noncompartmental analysis (NCA) without combining patients. Using validated PK software WinNonlin, the 'sparse sampling' option was utilized to treat the data as one virtual subject and thus the resulting data did not support reliable error estimates of the PK parameters.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Tmax for Plasma Maltol Glucuronide
|
0.52 h
NA
|
0.98 h
NA
|
2.78 h
NA
|
3.63 h
NA
|
1.00 h
NA
|
2.13 h
NA
|
SECONDARY outcome
Timeframe: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).Population: A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.
Area under the plasma concentration-time curve from pre-dose (time 0) to the time of last quantifiable plasma concentration for plasma maltol glucuronide (AUC0-t) (hxng/mL) after a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose. Because of the limitations of PK sampling in the pediatric patient population, data from multiple patients in the same treatment and age group had to be combined, using the naïve pooled approach, to achieve a complete set of observations over time for PK analysis. The data did not support PK parameters generation using noncompartmental analysis (NCA) without combining patients. Using validated PK software WinNonlin, the 'sparse sampling' option was utilized to treat the data as one virtual subject and thus the resulting data did not support reliable error estimates of the PK parameters.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
AUC0-t for Plasma Maltol Glucuronide
|
14600 hxng/mL
NA
|
5900 hxng/mL
NA
|
8070 hxng/mL
NA
|
16700 hxng/mL
NA
|
7670 hxng/mL
NA
|
12000 hxng/mL
NA
|
SECONDARY outcome
Timeframe: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).Population: A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.
Measured maximum plasma concentration (Cmax) (μg/dL) for baseline corrected serum iron after a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose. Because of the limitations of PK sampling in the pediatric patient population, data from multiple patients in the same treatment and age group had to be combined, using the naïve pooled approach, to achieve a complete set of observations over time for PK analysis. The data did not support PK parameters generation using noncompartmental analysis (NCA) without combining patients. Using validated PK software WinNonlin, the 'sparse sampling' option was utilized to treat the data as one virtual subject and thus the resulting data did not support reliable error estimates of the PK parameters.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Cmax for Baseline Corrected Serum Iron
|
119 μg/dL
NA
|
96.5 μg/dL
NA
|
21.0 μg/dL
NA
|
364 μg/dL
NA
|
87.0 μg/dL
NA
|
40.0 μg/dL
NA
|
SECONDARY outcome
Timeframe: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).Population: A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.
Time to maximum plasma concentration (Tmax) (h) for baseline corrected serum iron after a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose. Because of the limitations of PK sampling in the pediatric patient population, data from multiple patients in the same treatment and age group had to be combined, using the naïve pooled approach, to achieve a complete set of observations over time for PK analysis. The data did not support PK parameters generation using noncompartmental analysis (NCA) without combining patients. Using validated PK software WinNonlin, the 'sparse sampling' option was utilized to treat the data as one virtual subject and thus the resulting data did not support reliable error estimates of the PK parameters.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
Tmax for Baseline Corrected Serum Iron
|
2.00 h
NA
|
2.10 h
NA
|
2.78 h
NA
|
6.63 h
NA
|
2.37 h
NA
|
2.13 h
NA
|
SECONDARY outcome
Timeframe: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).Population: A total of 20 patients in the ferric maltol group were included in the PK Population. Of these patients, there were 7 patients (3 female patients and 4 male patients) who were 2 to 9 years of age, 3 patients (all female patients) who were 10 to 17 years of age and who received 15 mg BID of ferric maltol, and 10 patients (9 female patients and 1 male patient) who were 10 to 17 years of age and who received 30 mg BID of ferric maltol.
Area under the plasma concentration-time curve from pre-dose (time 0) to the time of last quantifiable plasma concentration for baseline corrected serum iron (AUC0-t) (hxng/mL) after a single dose of ferric maltol oral suspension on Visit 2 (PK Day 1) and after twice daily administrations for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose. Because of the limitations of PK sampling in the pediatric patient population, data from multiple patients in the same treatment and age group had to be combined, using the naïve pooled approach, to achieve a complete set of observations over time for PK analysis. The data did not support PK parameters generation using noncompartmental analysis (NCA) without combining patients. Using validated PK software WinNonlin, the 'sparse sampling' option was utilized to treat the data as one virtual subject and thus the resulting data did not support reliable error estimates of the PK parameters.
Outcome measures
| Measure |
PK Population Ferric Maltol 30mg (10-17 y) Day 1
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 7-10
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 7-10
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 30mg (10-17 y) Day 7-10
n=10 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (2-9 y) Day 1
n=7 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
PK Population Ferric Maltol 15mg (10-17 y) Day 1
n=3 Participants
The PK Population is defined as all randomized/assigned patients who had at least 1 dose of study drug and who had at least 1 evaluable post-dose PK sample.
|
|---|---|---|---|---|---|---|
|
AUC0-t for Baseline Corrected Serum Iron
|
374 hxμg/dL
NA
|
100 hxμg/dL
NA
|
1.93 hxμg/dL
NA
|
1180 hxμg/dL
NA
|
64.4 hxμg/dL
NA
|
60.1 hxμg/dL
NA
|
Adverse Events
Ferric Maltol Assigned
Ferric Maltol Randomized
Ferrous Sulfate Randomized
Serious adverse events
| Measure |
Ferric Maltol Assigned
n=3 participants at risk
12 weeks open-label Treatment Period for ferric maltol children aged 1 month to \<2 years
|
Ferric Maltol Randomized
n=31 participants at risk
Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days.
Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12.
Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose was used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2
\- 17 yrs were dosed 6 mg/kg to the maximum of 4 ml BID.
|
Ferrous Sulfate Randomized
n=30 participants at risk
Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
33.3%
1/3 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/31 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/30 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
Other adverse events
| Measure |
Ferric Maltol Assigned
n=3 participants at risk
12 weeks open-label Treatment Period for ferric maltol children aged 1 month to \<2 years
|
Ferric Maltol Randomized
n=31 participants at risk
Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days.
Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12.
Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose was used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2
\- 17 yrs were dosed 6 mg/kg to the maximum of 4 ml BID.
|
Ferrous Sulfate Randomized
n=30 participants at risk
Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid.
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
1/3 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
3.2%
1/31 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/30 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
|
Gastrointestinal disorders
Teething
|
33.3%
1/3 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/31 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/30 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/3 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
6.5%
2/31 • Number of events 3 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
10.0%
3/30 • Number of events 3 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
1/3 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
3.2%
1/31 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/30 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
|
Infections and infestations
Conjunctivitis
|
33.3%
1/3 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/31 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/30 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
33.3%
1/3 • Number of events 1 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/31 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/30 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
|
General disorders
Pyrexia
|
0.00%
0/3 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
6.5%
2/31 • Number of events 2 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
0.00%
0/30 • During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
|
Additional Information
Clinical Operations, Shield TX (UK) Ltd.
Shield TX (UK) Ltd.
Results disclosure agreements
- Principal investigator is a sponsor employee Study is part of a multicenter trial, and Institution may publish the results of its part of the Study in collaboration with the other investigators, but in complete compliance with this section and with the Confidential Information section. After the multicenter publication or twelve (12) months after completion of the Study, whichever occurs first. Sponsor should be informed at least sixty (60) days prior to the planned date of submission or presentation.
- Publication restrictions are in place
Restriction type: OTHER