Evaluate the PK, Safety, Tolerability of Ferric Maltol at 3 Dosage Levels in Paediatric Subjects With Iron Deficiency

A Phase 1, Open-Label, Randomised, Repeat Dose, Parallel Group Study to Evaluate the PK, Safety, Tolerability of Ferric Maltol at 3 Dosage Levels in Paediatric Subjects Aged 10-17 Years of Age With Iron Deficiency

The study has been designed to establish the pharmacokinetics (PK) and iron uptake of Ferric Maltol in children and adolescents aged 10-17 years using two (2) lower dose strengths in comparison to the EU-approved 30mg BID dose in adults with IDA in IBD.

Study Overview

• Status: Completed
• Conditions: Iron Deficiency, Anaemia in Children; Iron-Deficiency
• Intervention / Treatment: Drug: Ferric Maltol

Detailed Description

Phase I, open label, randomized, repeat dose, multicentre, pharmacokinetic study to assess the Safety and Tolerability of Ferric Maltol in 3 different dosages.

36 eligible patients will be randomized in a 1:1:1 ratio to one of the following 3 dosages for 9 days BID and a single dose on Day 10:

• 30mg ferric maltol capsules
• 16.6 mg ferric maltol capsules
• 7.8 mg ferric maltol capsules

Subject participation in the study will consist of 3 stages:

Screening: up to 14 days Treatment period: 10 days treatment period with 2 visits on Day 1 and Day 10 for PK blood sampling. Patients will be randomly allocated to one of the three Ferric Maltol dose groups according to centralized treatment allocation scheme.

Post-treatment Safety Follow-up:3-10 days following completion of the treatment period or premature discontinuation of study medication

• Study Type: Interventional
• Enrollment (Actual): 37
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • Leicester, United Kingdom, LE1 5WW
    • Leicester Royal Infirmary
  • Liverpool, United Kingdom, L14 5AB
    • Alder Hey Children's NHS Foundation Trust
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital
  • London, United Kingdom, SE5 9RS
    • King's College Hospital NHS Foundation Trust
  • London, United Kingdom, NW1 2PG
    • University College London Hospitals NHS Foundation Trust
  • Manchester, United Kingdom, M13 9WL
    • Royal Manchester Children's Hospital
  • Nottingham, United Kingdom, NG7 2UH
    • Nottingham University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Ability to understand the information given in the Independent Ethics Committee (IEC) approved Information Sheet and Consent form. The parent or guardian of the study subject must sign and date the informed consent and authorisation to use protected health information (PHI) in accordance with national and local subject privacy regulations prior to any study mandated procedure. The study participant will be asked to provide their assent to participate in the study using the IEC approved Assent form.
2. Willing and able to comply with study requirements.
3. Age ≥10 to ≤17 years at the time of informed consent and throughout duration of the study.
4. A current diagnosis of iron deficiency (with or without anaemia); iron deficiency defined by ferritin <30 µg/L, or ferritin <50 µg/L with transferrin saturation (TSAT) <20%, as measured by the central laboratory at the Screening visit (subjects with or without anaemia may be enrolled providing Hb is ≥8.5 g/dL as measured at the Screening visit).
5. Where appropriate, female subjects of childbearing potential must agree to use a reliable method of contraception until study completion and for at least 4 weeks following their final study visit. Reliable 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), complete sexual abstinence, a vasectomized partner and oral contraceptive medications.

Exclusion Criteria:

1. Has untreated or untreatable severe malabsorption syndrome e.g., untreated coeliac disease
2. Has received within 28 days prior to Screening intramuscular or intravenous (IV) injection or administration of depot iron preparation.
3. Has received oral iron supplementation within 7 days prior to Screening
4. Has received blood transfusion within 12 weeks prior to Screening or is scheduled to have blood transfusion or donations during the study period.
5. Has concomitant disease that would significantly compromise iron absorption or absorbed iron utilisation such as swallowing disorders and/or extensive small bowel resection.
6. Has chronic renal disease (eGFR <30mL/min), as assessed at Screening based on serum creatinine.
7. Known hypersensitivity or allergy to either the active substance or excipients of Ferric Maltol capsules.
8. Has a known contraindication for treatment with iron preparations, e.g. haemochromatosis, chronic haemolytic disease, sideroblastic anaemia, thalassemia, or lead intoxication induced anaemia.
9. 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.
10. Active acute inflammatory disease, including IBD flare or disease exacerbation, which in the opinion of the Investigator, is clinically significant.
11. Active chronic or acute infectious diseases requiring antibiotic treatment.
12. Pregnant or breast feeding.
13. Concomitant medical conditions with extensive active bleeding, other than menstrual cycles; subjects who suffer from menorrhagia may be included at the Investigator's discretion.
14. Scheduled or expected hospitalisation and/or surgery during the course of the study
15. Participation in any other interventional clinical study within 28 days prior to Screening.
16. 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.
17. Any other unspecified reason that, in the opinion of the Investigator or the Sponsor make the subject unsuitable for enrolment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: OTHER
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
ACTIVE_COMPARATOR: 30 mg Ferric Maltol; 12 subjects will receive 30 mg Ferric Maltol twice daily for 9 days (Days 1-9) plus a final 30mg dose on the morning of Day 10. PK study Day 1 & Day 10.	Drug: Ferric Maltol; To assess the pharmacokinetics and iron uptake of Ferric Maltol through measurement of serum iron, transferrin saturation (TSAT) and plasma concentrations of maltol and maltol glucuronide.; Other Names: Feraccru; ST10
ACTIVE_COMPARATOR: 16.6 mg Ferric Maltol; 12 subjects will receive 16.6 mg Ferric Maltol twice daily for 9 days (Days 1-9) plus a final 16.6mg dose on the morning of Day 10. PK study Day 1 & Day 10.	Drug: Ferric Maltol; To assess the pharmacokinetics and iron uptake of Ferric Maltol through measurement of serum iron, transferrin saturation (TSAT) and plasma concentrations of maltol and maltol glucuronide.; Other Names: Feraccru; ST10
ACTIVE_COMPARATOR: 7.8 mg Ferric Maltol; 12 subjects will receive 7.8 mg Ferric Maltol twice daily for 9 days (Days 1-9) plus a final 7.8mg dose on the morning of Day 10. PK study Day 1 & Day 10.	Drug: Ferric Maltol; To assess the pharmacokinetics and iron uptake of Ferric Maltol through measurement of serum iron, transferrin saturation (TSAT) and plasma concentrations of maltol and maltol glucuronide.; Other Names: Feraccru; ST10

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 1	Descriptive statistics and population PK analysis of maltol glucuronide Cmax from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 10	Descriptive statistics and population PK analysis of maltol glucuronide Cmax from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Area Under The Curve [AUC] of Maltol Glucuronide on Day 1	Descriptive statistics and population PK analysis of maltol glucuronide AUC from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Area Under The Curve [AUC] of Maltol Glucuronide on Day 10	Descriptive statistics and population PK analysis of maltol AUC from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Average Plasma Concentration [Cave(0-6h)] of Maltol Glucuronide on Day 10	Descriptive statistics and population PK analysis of maltol glucuronide Cave(0-6h) from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Time of Maximum Plasma Concentration [Tmax] of Maltol Glucuronide on Day 1	Descriptive statistics and population PK analysis of maltol glucuronide Tmax from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Time of Maximum Plasma Concentration [Tmax] of Maltol Glucuronide on Day 10	Descriptive statistics and population PK analysis of maltol glucuronide Tmax from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Half Life [t1/2] of Maltol Glucuronide on Day 1	Descriptive statistics and population PK analysis of maltol glucuronide t1/2 from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Ratio of Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 10/Day 1	Descriptive statistics of ratio maltol glucuronide Cmax Day 10/Day 10 from PK samples collected on Day 1 and Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 and Day 10, pre-dose and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Ratio of Area Under The Curve [AUC] of Maltol Glucuronide on Day 10/Day 1	Descriptive statistics of ratio maltol glucuronide AUC Day 10/Day 10 from PK samples collected on Day 1 and Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 and Day 10, pre-dose and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Average Serum Concentration [Cave(0-6h)] of Iron on Day 10	Descriptive statistics and population PK analysis of iron Cave(0-6h) from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Change From Pre-Dose (Ctrough) to Maximum Post-Dose (Cmax) in Serum Iron on Day 1	Descriptive statistics and population PK analysis of change in serum iron [Ctrough to Cmax] from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Change From Pre-Dose (Ctrough) to Maximum Post-Dose (Cmax) in Serum Iron on Day 10	Descriptive statistics and population PK analysis of change in serum iron [Ctrough to Cmax] from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Serum Iron on Day 1	Descriptive statistics and population PK analysis of pre-dose adjusted incremental AUC(0-6h) of serum iron from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Serum Iron on Day 10	Descriptive statistics and population PK analysis of pre-dose adjusted incremental AUC(0-6h) of serum iron from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Transferrin Saturation (TSAT) on Day 1	Descriptive statistics and population PK analysis of pre-dose adjusted incremental AUC(0-6h) of TSAT from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Transferrin Saturation (TSAT) on Day 10	Descriptive statistics and population PK analysis of pre-dose adjusted incremental AUC(0-6h) of TSAT from PK samples collected on Day 10. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Apparent Systemic Clearance (CL/F) of Iron on Day 1	Descriptive statistics and population PK analysis of serum iron CL/F from PK samples collected on Day 1	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Apparent Systemic Clearance (CL/F) of Iron on Day 10	Descriptive statistics and population PK analysis of serum iron CL/F from PK samples collected on Day 10	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Apparent Volume of Distribution (V/F) of Iron on Day 1	Descriptive statistics and population PK analysis of iron V/F from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Apparent Volume of Distribution (V/F) of Transferrin Saturation (TSAT) on Day 1	Descriptive statistics and population PK analysis of TSAT V/F from PK samples collected on Day 1	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Apparent Volume of Distribution (V/F) of Transferrin Saturation (TSAT) on Day 10	Descriptive statistics and population PK analysis of TSAT V/F from PK samples collected on Day 10	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Ratio Auc(0-6) Maltol Glucuronide Day 10/Day 1	Ratio AUC0-6h measured after last dose of Ferric Maltol on Day 10 vs first dose Day 1.	Day 1 to Day 10 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Serum Iron - RAUC(0-6h) D10/D1	Serum Iron - RAUC(0-6h) Day 10/Day 1	Measured after first and last dose of Ferric Maltol on Day 1 & Day 10 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Transferrin Saturation (%) Day 1, Baseline	Transferrin Saturation (TSAT%) Day 1, baseline	Measured after first dose of Ferric Maltol on Day 1 (0h)
Transferrin Saturation (%) Day 1, Maximum Response (%)	Transferrin Saturation (TSAT%) Day 1, maximum response (%)	Measured after first dose of Ferric Maltol on Day 1 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Transferrin Saturation Day 1, Time to Maximum Response Tmax	Transferrin Saturation (TSAT%) Day 1, time to maximum response Tmax (h). Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Measured after first dose of Ferric Maltol on Day 1. (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Transferrin Saturation (%) Day 10, Maximum Response (%)	Transferrin Saturation (TSAT%) Day 10, maximum response (%). Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Measured after last dose of Ferric Maltol on Day 10. (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Transferrin Saturation Day 10, Time to Maximum Response Tmax	Transferrin Saturation (TSAT%) Day 1, time to maximum response Tmax (h). Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Measured after first dose of Ferric Maltol on Day 10. (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h).
AUC0-inf Day 1 for Maltol Glucuronide	AUC0-inf for Maltol Glucuronide from PK samples collected on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Measured after first dose of Ferric Maltol on Day 1 (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
AUC0-tau Day 10 for Maltol Glucuronide	AUC0-tau for Maltol Glucuronide from PK samples collected on Day 10. Area under the plasma concentration versus time curve from time 0 to tau.	Measured after last dose of Ferric Maltol on Day 10. (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Cthrough for Maltol Glucuronide Day 10	Change from pre-dose to last PK samples collected on Day 10 for maltol glucuronide.Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 10 pre-dose to last (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Serum Iron Cmax Day 1	Maximum serum concentration of serum iron on Day 1. Each subject had 1 pre-dose (0h) and 2 post dose PK blood sampling between (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h). Subjects were assigned to PK blood sampling schedule. For each individual subject the PK blood sampling schedule was the same on Day 1 and Day 10.	Day 1 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Serum Iron Cmax on Day 10	Maximum serum concentration of serum iron on Day 10.	Day 10 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Plasma Maltol Glucuronide Cthrough D10/Day1	Minimum concentration between dose time and dose time+TAU	Day 10 (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Apparent Systemic Clearance (CL/F) of Transferrin Saturation (TSAT) on Day 1	Descriptive statistics and population PK analysis of serum TSAT CL/F from PK samples collected on Day 1	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h
Apparent Systemic Clearance (CL/F) of Transferrin Saturation (TSAT) on Day 10	Descriptive statistics and population PK analysis of serum TSAT CL/F from PK samples collected on Day 10	Day 10 pre-final dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Apparent Systemic Clearance (CL/F) of Maltol Glucuronide on Day 1	Descriptive statistics and population PK analysis of plasma maltol glucuronide CL/F from PK samples collected on Day 1.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h
Apparent Systemic Clearance (CL/F) of Maltol Glucuronide on Day 10	Descriptive statistics and population PK analysis of plasma maltol glucuronide CL/F from PK samples collected on Day 10.	Day 1 pre-first dose of Ferric Maltol and up to 6 hours post-dose (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Transferrin - Change From Baseline to Day 10, Predose	Change calculated as difference in values measured at Day 1, predose (Baseline) and on Day 10, predose	Day 1 pre-dose to Day 10 pre-dose (0h on each day)
Ferritin - Change From Baseline to Day 10, Predose	Change calculated as difference in values measured at Day 1, predose (Baseline) and on Day 10, predose.	Pre-dose on Day 1 to Day 10 (0h)
Total Iron Binding Capacity - Change From Day 1 to Day 10, Predose	Change calculated as difference in values measured at Day 1, predose and on Day 10, predose	Predose from Day 1 to Day 10 (0h on each day)
UIBC - Change From Day 1 to Day 10, Predose	Change calculated as difference in values measured at Day 1, predose and on Day 10, predose	Pre-dose on Day 1 to Day 10 (0h each day)
Negative and Positive NTBI Tests on Day 1	Negative and Positive Non-Transferrin Bound Iron [NTBI] tests on Day 1, predose	Day 1 (0h)
Change From Baseline to Day 10 in Haemoglobin Concentration	Change calculated as difference in values measured at Screening (Baseline) and on Day 10	Screening and Day 10 (1-4 hours post-dose)
Change From Baseline to Day 10 in Absolute Reticulocyte Count	Change from Baseline to Day 10 in Absolute Reticulocyte Count collected from PK samples	Change calculated as difference in values measured at Screening (Baseline) and on Day 10.
Treatment-emergent Adverse Events (AEs) Leading to Premature Discontinuation of Study Drug/PK Assessments	Descriptive summary of incidence and causal relationship of treatment-emergent adverse events leading to discontinuation of study drug/PK assessments according to MedDRA preferred term (PT) and system organ class (SOC)	From first dose of Ferric Maltol on Day 1 through study completion, on average 4 weeks
Changes in 12-lead ECG Parameters From Screening to Day 10	Overall clinical interpretation of routine ECG parameters from Screening to Day 10	Screening and Day 10 (1-4 hours post-dose)
Concomitant Medications	Number of subjects with concomitant medications Taken by >5% of Subjects	Screening, Day 1, Day 10 and Post-Study Follow-up visit, on average 4 weeks
Negative and Positive NTBI Tests on Day 10, Predose	Negative and Positive Non-Transferrin Bound Iron [NTBI] tests on Day 10, predose	Day 10
Treatment-emergent Serious Adverse Event (TESAE)	Descriptive summary of TESAE according to MedDRA preferred Term	From first dose of ferric maltol Day 1 through study completions, on average 4 weeks

Collaborators and Investigators

• Sponsor: Shield Therapeutics
• Collaborators: Medpace, Inc.

Study record dates

Study Major Dates

• Study Start (ACTUAL): March 14, 2017
• Primary Completion (ACTUAL): March 28, 2018
• Study Completion (ACTUAL): March 28, 2018

Study Registration Dates

• First Submitted: May 23, 2017
• First Submitted That Met QC Criteria: June 7, 2017
• First Posted (ACTUAL): June 8, 2017

Study Record Updates

• Last Update Posted (ACTUAL): October 6, 2021
• Last Update Submitted That Met QC Criteria: September 13, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Anemia, Hypochromic; Anemia; Iron Metabolism Disorders; Anemia, Iron-Deficiency; Hematinics; Ferric maltol
• Other Study ID Numbers: ST10-01-103

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No