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.~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, metabolism of serum iron and elimination of maltol from the Pre-assignment PK samples by showing plasma maltol return to baseline, 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: 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.~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, summarised as the mean across all subjects, with 95% 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.~Population PK analysis will be conducted for maltol and maltol glucuronide in plasma and for serum iron and TSAT in children and adolescents aged 1 month - 17 years.~Full details of the statistical analysis, including the analysis of PK endpoints, will be specified in the statistical analysis plan (SAP).
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.