AnemiaPhase 3FORTISNCT05126901
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
A Phase 3 study testing Ferric Maltol and Ferrous sulfate in people with Anemia and Iron-deficiency, run by Shield Therapeutics at 23 sites in 3 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 1 month to 17 years
- Conditions the study is for
- Anemia, Iron-deficiency
- What is being tested?
- Ferric Maltol and Ferrous sulfate
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 23 sitesin 3 countries
- Study dates
- 3 Nov 2021to 9 Jun 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patient is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.
- Age ≥1 month and ≤17 years at the time of informed consent
- Subjects must have iron deficiency anaemia defined by the following criteria, as measured by the central laboratory at the screening visit
- 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.
Who cannot join
- Subject with anaemia due to any cause other than iron deficiency, including, but not limited to,
- Untreated or untreatable severe malabsorption syndrome
- Subjects who have received prior to Screening:
- Within 28 days intramuscular or intravenous (IV) injection or administration of depot iron preparation.
- Within 7 days single agent iron preparations and during the study.
- Within 12 weeks of blood transfusion or is scheduled to have blood transfusion or donation during the study period
Show all 24Show fewer
- Within 28 days erythropoiesis stimulating agents and during the study period
- Within 14 days COVID-19 vaccination
- 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.
Trial locations and contacts
23 sites in 3 countries.
23 sites in 3 countries
United States14 sites
Levine Cancer Institute
Charlotte, North Carolina, 28204
BRCR Global Texas
Edinburg, Texas, 78539
Penn State Hershey Children's Hospital
Hershey, Pennsylvania, 17033
Homestead Research Institute
Homestead, Florida, 33030
Clinical Research Prime
Idaho Falls, Idaho, 83404
Zion Research
Katy, Texas, 77494
Kissimmee Clinical Research Corp
Kissimmee, Florida, 34743
Sierra Clinical Research
Las Vegas, Nevada, 89106
Miami Clinical Research
Miami, Florida, 33155
Medical Research of Westcheste
Miami, Florida, 33165
Eminent Clinical Research and Associates
North Lauderdale, Florida, 33068
Hasbro Children's Hospital
Providence, Rhode Island, 02903
The Center for Clinical Trials
Saraland, Alabama, 36571
MultiCare Health System Institute for Research and Innovation
Tacoma, Washington, 98405
United Kingdom8 sites
Noah's Ark Children's Hospital for Wales
Cardiff, CF14 4XW
Royal Hospital for Sick Children - Edinburgh
Edinburgh, EH16 4TJ
Leicester Royal Infirmary
Leicester, LE1 5WW
Alder Hey Children's NHS Foundation Trust
Liverpool
King's College Hospital
London, SE5 9RS
Newham University Hospital
London
Royal Manchester Children's Hospital
Manchester, M13 9WL
Nottingham University Hospitals
Nottingham, NG7 2UH
About this study
Official title: 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
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.
Read the detailed description
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:
- Screening: within 14 days prior to randomisation for each subject
- Pre-assignment PK phase: only applicable for subjects aged 1 month to less than 2 years. Up to 21 days from Screening.
- Randomised treatment: 12 weeks open label treatment
- Assigned treatment phase 12 weeks open label treatment for ferric maltol children aged 1 month to less than 2 years
- End of study: Week 12 visit
- Post-treatment safety follow-up: 10-14 days following study completion of the treatment period or premature discontinuation
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).
- Conditions
- Anemia, Iron-deficiency
- Being tested
- Ferric Maltol, Ferrous sulfate
- Keywords
- Anemia, Iron-Deficiency
- Medical subject headings
- Anemia, Anemia, Iron-Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ferric MaltolDrug
Also called Feraccru, Ferric Trimaltol, ST10, ST10-01
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.
Ferrous sulfateDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ferric Maltol |
| 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. Receives: Drug: Ferric Maltol |
| 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. Receives: Drug: Ferrous sulfate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to week 12.
Secondary outcomes (9)
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.
Measured: From baseline to week 12.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2021
- Study start3 Nov 2021
- Primary completion9 Jun 2024
- Study completion9 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shield Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05126901
- Sponsor's protocol ID
- ST10-01-305
- Acronym
- FORTIS
- Status last verified
- Sep 2025
- First submitted
- 27 Oct 2021
- Last updated
- 10 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05126901
- Acronym / sponsor ID
- FORTIS / ST10-01-305
- Conditions
- Anemia, Iron-deficiency
- Keywords
- Anemia, Iron-Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- Shield TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 3 Nov 2021 (actual)
- Primary completion
- 9 Jun 2024 (actual)
- Study completion
- 9 Jun 2024 (actual)
- First posted
- 19 Nov 2021 (actual)
- Results
- Posted 10 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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:
- Screening: within 14 days prior to randomisation for each subject
- Pre-assignment PK phase: only applicable for subjects aged 1 month to less than 2 years. Up to 21 days from Screening.
- Randomised treatment: 12 weeks open label treatment
- Assigned treatment phase 12 weeks open label treatment for ferric maltol children aged 1 month to less than 2 years
- End of study: Week 12 visit
- Post-treatment safety follow-up: 10-14 days following study completion of the treatment period or premature discontinuation
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| 1 month to 2 year old subjects (infants) | Experimental | Drug: Ferric MaltolSubjects 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. |
| 2 to 17 year old subjects - Ferric Maltol | Experimental | Drug: Ferric MaltolSubjects 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. |
| 2 to 17 year old subjects - Ferrous Sulfate | Active comparator | Drug: Ferrous sulfateSubjects 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. |
Who may qualify
- Ages
- 1 month to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Patient is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.
- Age ≥1 month and ≤17 years at the time of informed consent
- Subjects must have iron deficiency anaemia defined by the following criteria, as measured by the central laboratory at the screening visit
- 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 <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.
Key exclusion criteria (24 listed)
- Subject with anaemia due to any cause other than iron deficiency, including, but not limited to,
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ferric MaltolAlso: Feraccru, Ferric Trimaltol, ST10, ST10-01Ferric 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. | Drug | 1 month to 2 year old subjects (infants), 2 to 17 year old subjects - Ferric Maltol |
| Ferrous sulfateFerrous 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. | Drug | 2 to 17 year old subjects - Ferrous Sulfate |
Primary outcome
Change in Hemoglobin Concentration
Time frame: From baseline to week 12.
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.
9 secondary outcomes
Changes in Ferritin Concentration
Time frame: From baseline to week 12.
Change in Iron Concentration
Time frame: From baseline to week 12.
Change in the Percentage of Transferrin Saturation
Time frame: From baseline to week 12.
Cmax for Plasma Maltol Glucuronide
Time frame: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).
Tmax for Plasma Maltol Glucuronide
Time frame: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).
AUC0-t for Plasma Maltol Glucuronide
Time frame: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).
Cmax for Baseline Corrected Serum Iron
Time frame: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).
Tmax for Baseline Corrected Serum Iron
Time frame: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).
AUC0-t for Baseline Corrected Serum Iron
Time frame: PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ferric Maltol and Ferrous sulfate in people with Anemia and Iron-deficiency, run by Shield Therapeutics at 23 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, United Kingdom, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Nov 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Hemoglobin Concentration (time frame: From baseline to week 12.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Nov 2025.