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The objective of the study is to compare the safety and gastrointestinal tolerability of ferric maltol oral suspension and ferrous sulfate oral liquid in children and adolescents aged 2 years to 17 years, and assess the safety and tolerability of ferric maltol oral suspension in children 1 month to less than 2 years, in the treatment of iron deficiency anaemia during the 12 weeks treatment period.
The study is a randomised, Open-label, Active-controlled, Multicentre, Comparative Study to Evaluate the Safety and Efficacy of Ferric Maltol (Iron (III)-Maltol Complex) (ST10) Oral Suspension Compared to Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, Incorporating a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years.
Approximately 110 male and female children from 1 month to 17 years of age, with iron deficiency anaemia. If less than 91 subjects in total have been randomized when 32 ferric maltol subjects have completed, then an interim analysis will be conducted.
Subjects aged 2 to 17 years will be 1:1 randomised to ferric maltol and ferrous sulfate, with 49 subjects in each arm. Subjects then will be further divided into 2 age groups: 2 yrs - 9 yrs and 10 yrs -17 yrs. A minimum of 18 subjects must be recruited into the 2 yrs - 9 yrs and 10 yrs - 17 yrs age groups and a minimum of 25% of either sex must be recruited.
A maximum of 12 subjects will be recruited in the 1 month to less than 2 years age group. They will only be assigned to the ferric maltol group, once there is evidence of absorption, of serum iron and elimination of maltol from the Pre-assignment PK samples by showing urine maltol return to baseline, or to a low level, confirming no accumulation of maltol or maltol glucuronide, they will continue on to the 12 weeks treatment phase.
Design: The study will comprise of the following stages:
Investigational Product Product: Ferric maltol oral suspension: oral suspension containing 30 mg elemental iron, in the form of 231.5 mg ferric maltol, in 5 ml suspension.
Ferric maltol oral suspension will be taken every morning and evening at least 30 minutes after a meal. Dosing will be supervised by the parent/legal guardian for children/adolescents throughout the treatment period and recorded on a dosing diary.
Ferric maltol bottles will be labelled for clinical trials use and each bottle will have a unique bottle number which will be utilised in the randomisation procedure.
A final eligibility evaluation must be conducted immediately prior to randomisation.
Reference safety information will be the Investigator Brochure.
Comparator therapy: Ferrous sulfate 125 mg/ml (25 mg/ml elemental iron) oral liquid or equivalent dose will be administered under this protocol. Dosing will be supervised by the parent/legal guardian for children/adolescents throughout the treatment period and recorded on a dosing diary.
Reference safety information will be the currently approved summary of product characteristics.
Statistical methods:
Safety and gastrointestinal tolerability will be compared between ferric maltol oral suspension and ferrous sulfate oral liquid via summaries of treatment emergent adverse events (TEAEs), treatment emergent serious AEs (TESAEs) and treatment-emergent AEs (TEAEs) leading to premature discontinuation of study drug.
Efficacy of ferric maltol will be assessed via the change in Hb concentration from baseline to week 12. If no interim analysis is conducted it will be based on a 95% two-sided confidence interval; If an interim analysis is conducted, a Pocock spending function will be used; the interim analysis will be based on a (100 - 3.45)% two sided confidence interval; if the study does not stop after the interim analysis, the final analysis will be based on a (100 - 2.57)% two sided confidence interval.
For the PK analysis, all analytes in serum will be summarised per PK day, for children and adolescents aged 1 month to 17 years receiving ferric maltol.
In addition, all analytes in urine will be summarised per PK day, for children aged 1 month to less than 2 years.
Full details of the statistical analysis, including the analysis of PK endpoints, will be specified in the statistical analysis plan (SAP).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 month to 2 year old subjects (infants) | Experimental | Subjects aged 1 month to less than 2 years will enter a Pre-assignment phase: baseline urine samples are collected and subjects will take a single dose of 0.1 ml/kg ferric maltol suspension. Further 3 samples up to 12h will be taken. Subjects showing evidence of absorption, metabolism and elimination of maltol will enter the treatment phase and be assigned to the ferric maltol arm. The first 6 subjects screened will perform the pre-assignment PK phase. After review by the investigator, and medical monitors , if Maltol Glucuronide is shown to be adequately eliminated, timepoint 20-24 hrs (+ 4hrs) will not be performed on subsequent subjects. Subjects will be assigned to receive ferric maltol oral suspension and start the 0.1 ml/kg BID dose on V2 and continue for 7-10 days. On V3 they will perform the same PK assessments as on Pre-assignment PK visit. |
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| 2 to 17 year old subjects - Ferric Maltol | Experimental | 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 will be used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2 - 17 yrs will be dosed 6 mg/kg to the maximum of 4 ml BID. Subjects randomised to ferrous sulfate oral liquid will not need to complete the PK period. |
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| 2 to 17 year old subjects - Ferrous Sulfate | Active Comparator | Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose will be used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2 - 17 yrs will be dosed 6 mg/kg to the maximum of 4 ml BID. Subjects randomised to ferrous sulfate oral liquid will not need to complete the PK period. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ferric Maltol | Drug | Ferric maltol oral suspension: 150 ml amber glass bottle with graduated syringe and adaptor. Oral suspension containing 30 mg elemental iron, in the form of 231.5 mg ferric maltol, in 5 ml suspension Study dosage: The dose of ferric maltol oral suspension that will be administered for children aged 1 month to < 2 yrs: 0.1 ml/kg BID, 2 to - 11 yrs: 2.5 ml BID, 12-17 yrs: 5 ml BID. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Hemoglobin Concentration | 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. | From baseline to week 12. |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in Ferritin Concentration | 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. | From baseline to week 12. |
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Inclusion Criteria:
Haemoglobin thresholds define anaemia by age and gender:
Children (1 m - < 5 yrs) <11.0 g/dl Children (5 yrs - < 12 yrs) <11.5 g/dl Children (12 yrs) <12.0 g/dl Female child (≥13 yrs) <12.0 g/dl Male child (≥13 yrs) <13.0 g/dl and
Ferritin thresholds define anaemia by:
ferritin <30 µg/L, or ferritin <50 µg/L with transferrin saturation (TSAT) <20%, 4. Female subjects of childbearing potential must agree to use a highly effective method of contraception (which includes complete abstinence) until study completion and for at least 4 weeks following their final study visit. Highly effective contraception is defined as a method which results in a low failure rate, i.e., less than 1% per year when used consistently and correctly, such as implants, injectables, some intrauterine contraceptive devices (IUDs), a vasectomised partner and oral contraceptive medications.
The need for contraception and compliance with contraception requirements will be assessed at every visit for adolescent patients, and urine pregnancy testing will be performed at each visit for female subjects of childbearing potential.
Exclusion Criteria:
Subject with anaemia due to any cause other than iron deficiency, including, but not limited to,
a. Untreated or untreatable severe malabsorption syndrome
Subjects who have received prior to Screening:
Subjects with vitamin B12 or folic acid deficiency as determined by the central laboratory screening results. Subjects may start vitamin B12 or folate replacement and rescreen after at least 2 weeks.
Has concomitant disease that would significantly compromise iron absorption or absorbed iron utilization such as swallowing disorders and/or extensive small bowel resection.
History of active peptic ulcer
Has chronic renal disease (eGFR <60 mL/min/m2), as assessed at Screening based on serum creatinine.
Known hypersensitivity or allergy to either the active substance or excipients of ferric maltol or ferrous sulfate.
Has a known contraindication for treatment with iron preparations, e.g. haemochromatosis, chronic haemolytic disease, sideroblastic anaemia, thalassemia, or lead intoxication induced anaemia.
Impaired liver function as indicated by alanine aminotransferase (ALT) or aspartate transaminase (AST)>2.0 times upper normal limit as measured at the Screening visit.
Active acute inflammatory disease, including IBD flare or disease exacerbation, which in the opinion of the Investigator, is clinically significant.
Active chronic or acute infectious diseases requiring antibiotic treatment.
Pregnant or breast feeding.
Concomitant medical conditions with extensive active bleeding, other than menstrual cycles; subjects who suffer from menorrhagia may be included at the Investigator's discretion.
Scheduled or expected hospitalisation and/or surgery during the course of the study
Participation in any other interventional clinical study within 28 days prior to Screening.
Diagnosed to be COVID-19 positive by (SARS-CoV-2-RT-PCR positive) within 28 days prior to screening.
Cardiovascular, liver, renal, hematologic, psychiatric, neurologic, gastrointestinal, immunologic, endocrine, metabolic, respiratory or central nervous system disease that, in the opinion of the Investigator, may adversely affect the safety of the subject and/or objectives of the study drug or severely limit the lifespan of the subject.
Any other unspecified reason that, in the opinion of the Investigator or the Sponsor make the subject unsuitable for enrolment.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Center for Clinical Trials | Saraland | Alabama | 36571 | United States | ||
| Homestead Research Institute |
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ferric Maltol Assigned | 12 weeks open-label Treatment Period for ferric maltol children aged 1 month to <2 years |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 6, 2023 | Jul 28, 2025 |
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The study will include 98 subjects in the 2 - 17 years age group, randomised 1:1 between ferric maltol and ferrous sulfate: 49 in each treatment group. The study will also include up to 12 subjects in the 1 month to less than 2 years age group. If less than 91 subjects in total have been randomized when 32 ferric maltol subjects have completed, then an interim analysis will be conducted. If significant, the study will stop recruitment. If not significant, the study will continue until 54 subjects have been recruited in the ferric maltol arm.
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12 weeks open label treatment
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| Ferrous sulfate | Drug | Ferrous sulfate 125 mg/ml (25 mg/ml elemental iron) oral liquid : 15 ml glass bottle. Study dosage: For ferrous sulfate oral liquid, the dose administered will be for children and adolescents aged 2 years to 17 yrs: 6 mg/kg to the maximum of 4 ml BID. |
|
| Change in Iron Concentration | 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. | From baseline to week 12. |
| Change in the Percentage of Transferrin Saturation | 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. | From baseline to week 12. |
| Cmax for Plasma Maltol Glucuronide | 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. | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
| Tmax for Plasma Maltol Glucuronide | 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. | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
| AUC0-t for Plasma Maltol Glucuronide | 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. | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
| Cmax for Baseline Corrected Serum Iron | 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. | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
| Tmax for Baseline Corrected Serum Iron | 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. | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
| AUC0-t for Baseline Corrected Serum Iron | 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. | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
| Homestead |
| Florida |
| 33030 |
| United States |
| Kissimmee Clinical Research Corp | Kissimmee | Florida | 34743 | United States |
| Miami Clinical Research | Miami | Florida | 33155 | United States |
| Medical Research of Westcheste | Miami | Florida | 33165 | United States |
| Eminent Clinical Research and Associates | North Lauderdale | Florida | 33068 | United States |
| Clinical Research Prime | Idaho Falls | Idaho | 83404 | United States |
| Sierra Clinical Research | Las Vegas | Nevada | 89106 | United States |
| Levine Cancer Institute | Charlotte | North Carolina | 28204 | United States |
| Penn State Hershey Children's Hospital | Hershey | Pennsylvania | 17033 | United States |
| Hasbro Children's Hospital | Providence | Rhode Island | 02903 | United States |
| BRCR Global Texas | Edinburg | Texas | 78539 | United States |
| Zion Research | Katy | Texas | 77494 | United States |
| MultiCare Health System Institute for Research and Innovation | Tacoma | Washington | 98405 | United States |
| BRCR Global Puerto | San Juan | 00907 | Puerto Rico |
| Noah's Ark Children's Hospital for Wales | Cardiff | CF14 4XW | United Kingdom |
| Royal Hospital for Sick Children - Edinburgh | Edinburgh | EH16 4TJ | United Kingdom |
| Leicester Royal Infirmary | Leicester | LE1 5WW | United Kingdom |
| Alder Hey Children's NHS Foundation Trust | Liverpool | United Kingdom |
| King's College Hospital | London | SE5 9RS | United Kingdom |
| Newham University Hospital | London | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | M13 9WL | United Kingdom |
| Nottingham University Hospitals | Nottingham | NG7 2UH | United Kingdom |
| FG002 | Ferrous Sulfate Randomized | Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Ferric Maltol Assigned | 12 weeks open-label Treatment Period for ferric maltol children aged 1 month to <2 years |
| BG001 | 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. |
| BG002 | Ferrous Sulfate Randomized | Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Hemoglobin Concentration | 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. | 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). | Posted | Mean | Standard Deviation | g/L | From baseline to week 12. |
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| Secondary | Changes in Ferritin Concentration | 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. | 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). | Posted | Mean | Standard Deviation | μg/L | From baseline to week 12. |
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| Secondary | Change in Iron Concentration | 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. | 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). | Posted | Mean | Standard Deviation | μmol/L | From baseline to week 12. |
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| Secondary | Change in the Percentage of Transferrin Saturation | 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. | 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). | Posted | Mean | Standard Deviation | Percentage of Transferrin Saturation | From baseline to week 12. |
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| Secondary | Cmax for Plasma Maltol Glucuronide | 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. | 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. | Posted | Number | ng/mL | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
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| Secondary | Tmax for Plasma Maltol Glucuronide | 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. | 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. | Posted | Number | h | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
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| Secondary | AUC0-t for Plasma Maltol Glucuronide | 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. | 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. | Posted | Number | hxng/mL | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
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| Secondary | Cmax for Baseline Corrected Serum Iron | 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. | 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. | Posted | Number | μg/dL | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
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| Secondary | Tmax for Baseline Corrected Serum Iron | 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. | 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. | Posted | Number | h | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
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| Secondary | AUC0-t for Baseline Corrected Serum Iron | 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. | 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. | Posted | Number | hxμg/dL | PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3). |
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During the 12-week treatment period and within 2 weeks after receiving the last dose of the study drug.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ferric Maltol Assigned | 12 weeks open-label Treatment Period for ferric maltol children aged 1 month to <2 years | 0 | 3 | 1 | 3 | 2 | 3 |
| EG001 | 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. | 0 | 31 | 0 | 31 | 4 | 31 |
| EG002 | Ferrous Sulfate Randomized | Patients aged 2 to 17 years were randomized 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. | 0 | 30 | 0 | 30 | 3 | 30 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Wheezing | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | Systematic Assessment |
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| Pyrexia | General disorders | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
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| Teething | Gastrointestinal disorders | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | Systematic Assessment |
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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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Operations, Shield TX (UK) Ltd. | Shield TX (UK) Ltd. | +44 (0) 191 511 8500 | info@shieldtherapeutics.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 10, 2024 | Jul 28, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D000740 | Anemia |
| D018798 | Anemia, Iron-Deficiency |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000747 | Anemia, Hypochromic |
| D000090463 | Iron Deficiencies |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C062088 | ferric maltol |
| C115835 | ferric trimaltol |
| C020748 | ferrous sulfate |
Not provided
Not provided
Not provided
| Newborns (0-27 days) |
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| Infants and toddlers (28 days-23 months) |
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| Children (2-11 years) |
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| Adolescents (12-17 years) |
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| Adults (18-64 years) |
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| From 65-84 years |
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| 85 years and over |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| OG001 | PK Population Ferric Maltol 15mg (10-17 y) Day 1 | 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. |
| OG002 | PK Population Ferric Maltol 30mg (10-17 y) Day 1 | 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. |
| OG003 | 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. |
| OG004 | 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. |
| OG005 | 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. |
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| OG001 |
| PK Population Ferric Maltol 15mg (10-17 y) Day 1 |
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. |
| OG002 | PK Population Ferric Maltol 30mg (10-17 y) Day 1 | 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. |
| OG003 | 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. |
| OG004 | 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. |
| OG005 | 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. |
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| OG001 | PK Population Ferric Maltol 15mg (10-17 y) Day 1 | 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. |
| OG002 | PK Population Ferric Maltol 30mg (10-17 y) Day 1 | 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. |
| OG003 | 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. |
| OG004 | 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. |
| OG005 | 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. |
|
|
| OG001 | PK Population Ferric Maltol 15mg (10-17 y) Day 1 | 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. |
| OG002 | PK Population Ferric Maltol 30mg (10-17 y) Day 1 | 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. |
| OG003 | 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. |
| OG004 | 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. |
| OG005 | 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. |
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| OG001 | PK Population Ferric Maltol 15mg (10-17 y) Day 1 | 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. |
| OG002 | PK Population Ferric Maltol 30mg (10-17 y) Day 1 | 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. |
| OG003 | 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. |
| OG004 | 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. |
| OG005 | 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 (applicable only for the Ferric Maltol group). 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. |
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| OG001 | PK Population Ferric Maltol 15mg (10-17 y) Day 1 | 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. |
| OG002 | PK Population Ferric Maltol 30mg (10-17 y) Day 1 | 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. |
| OG003 | 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. |
| OG004 | 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. |
| OG005 | 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. |
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