Iron Deficiency, Anaemia in ChildrenPhase 1NCT03181451

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

A Phase 1 study testing Ferric Maltol in people with Iron Deficiency, Anaemia in Children and Iron-Deficiency, run by Shield Therapeutics at 7 sites in United Kingdom.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

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. Read more about this study

Who is it for?: Ages 10 to 17 years
Conditions the study is for: Iron Deficiency, Anaemia in Children, Iron-Deficiency
What is being tested?: Ferric Maltol
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 37 participants
Where: 7 sitesUnited Kingdom
Study dates: 14 Mar 2017to 28 Mar 2018
Phase: Phase 1
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 10 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

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.
Willing and able to comply with study requirements.
Age ≥10 to ≤17 years at the time of informed consent and throughout duration of the study.
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).
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.

Who cannot join

Has untreated or untreatable severe malabsorption syndrome e.g., untreated coeliac disease
Has received within 28 days prior to Screening intramuscular or intravenous (IV) injection or administration of depot iron preparation.
Has received oral iron supplementation within 7 days prior to Screening
Has received blood transfusion within 12 weeks prior to Screening or is scheduled to have blood transfusion or donations during the study period.
Has concomitant disease that would significantly compromise iron absorption or absorbed iron utilisation such as swallowing disorders and/or extensive small bowel resection.
Has chronic renal disease (eGFR <30mL/min), as assessed at Screening based on serum creatinine.

Show all 17Show fewer

Known hypersensitivity or allergy to either the active substance or excipients of Ferric Maltol capsules.
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.
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

7 sites in 1 country.

City, postcode or hospital

7 sites in 1 country

United Kingdom7 sites

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

Other trials in United Kingdom

About this study

Official title: 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

Read the 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

Conditions: Iron Deficiency, Anaemia in Children, Iron-Deficiency
Being tested: Ferric Maltol
Medical subject headings: Iron Deficiencies, 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 ST10, Feraccru
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.

Study groups in NCT03181451
Study group	Role	What they receive
30 mg Ferric Maltol	Active comparator	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. Receives: Drug: Ferric Maltol
16.6 mg Ferric Maltol	Active comparator	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. Receives: Drug: Ferric Maltol
7.8 mg Ferric Maltol	Active comparator	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. Receives: Drug: Ferric Maltol

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: 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.
Measured: 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
Measured: 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
Measured: 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.
Measured: 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: 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: 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: 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: 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: 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: 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: 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: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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 outcomes (12)

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
Measured: 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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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
Measured: Screening and Day 10 (1-4 hours post-dose)
Concomitant Medications
Number of subjects with concomitant medications Taken by >5% of Subjects
Measured: 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
Measured: Day 10
Treatment-emergent Serious Adverse Event (TESAE)
Descriptive summary of TESAE according to MedDRA preferred Term
Measured: From first dose of ferric maltol Day 1 through study completions, on average 4 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

14 Mar 201728 Mar 2018

First posted8 Jun 2017
Study start14 Mar 2017
Primary completion28 Mar 2018
Study completion28 Mar 2018

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 Iron Deficiency, Anaemia in ChildrenStudies 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 1
Primary purpose: Other
Allocation: Randomised
Intervention model: Parallel groups
Model description: Subjects will be randomized in a 1:1:1 ratio and stratified by co-variates for age and sex. This will ensure that a minimum of 25% of each gender and at least 3 children per age group are enrolled in each Ferric Maltol dose group.
Masking: None (open label)
Enrolment: 37 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03181451
Sponsor's protocol ID: ST10-01-103
Status last verified: Sep 2021
First submitted: 23 May 2017
Last updated: 6 Oct 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruiting
Effect of Vitamin D Supplementation Added to Iron Therapy in Children With Iron Deficiency Anemia
Iron Deficiency Anemia
RecruitingObservational study
Maternal Iron Deficiency and Childhood Health
Iron Deficiency Anaemia in Childbirth, Iron Deficiency
RecruitingPhase 3
Multi-center Trial of Ferric Derisomaltose in Children 0 to <18 Years of Age With Iron Deficiency Anemia
Iron Deficiency, Anaemia in Children
Not yet recruitingPhase 2
COMPARISON OF EFFICACY OF ORAL VS IV IRON THERAPY IN CHILDREN AGED 12-59 MONTHS, WITH IRON DEFICIENCY ANEMIA
Iron Deficiency Anemia in Children
Recruiting
Efficacy of Oral Sucrosomial Iron Supplementation in Children With Celiac Disease and Iron Deficiency or Anemia
Celiac Disease in Children, Anemia

All Iron Deficiency, Anaemia in Children trials

Study at a glance

From the registry record, last updated by the sponsor on 6 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT03181451
Acronym / sponsor ID: ST10-01-103
Conditions: Iron Deficiency, Anaemia in Children, Iron-Deficiency
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 37 participants (actual)
Lead sponsor: Shield TherapeuticsIndustry (pharma, biotech, devices)
Collaborators: Medpace, Inc.
Study start: 14 Mar 2017 (actual)
Primary completion: 28 Mar 2018 (actual)
Study completion: 28 Mar 2018 (actual)
First posted: 8 Jun 2017 (actual)
Results: Posted 2 Jun 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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:

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

Study design

Primary purpose: Other
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 3
Model description: Subjects will be randomized in a 1:1:1 ratio and stratified by co-variates for age and sex. This will ensure that a minimum of 25% of each gender and at least 3 children per age group are enrolled in each Ferric Maltol dose group.

Arm	Type	Receives
30 mg Ferric Maltol	Active comparator	Drug: Ferric Maltol12 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.
16.6 mg Ferric Maltol	Active comparator	Drug: Ferric Maltol12 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.
7.8 mg Ferric Maltol	Active comparator	Drug: Ferric Maltol12 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.

Who may qualify

Ages: 10 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

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.
Willing and able to comply with study requirements.
Age ≥10 to ≤17 years at the time of informed consent and throughout duration of the study.
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).
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.

Key exclusion criteria (17 listed)

Has untreated or untreatable severe malabsorption syndrome e.g., untreated coeliac disease
Has received within 28 days prior to Screening intramuscular or intravenous (IV) injection or administration of depot iron preparation.
Has received oral iron supplementation within 7 days prior to Screening
Has received blood transfusion within 12 weeks prior to Screening or is scheduled to have blood transfusion or donations during the study period.
Has concomitant disease that would significantly compromise iron absorption or absorbed iron utilisation such as swallowing disorders and/or extensive small bowel resection.

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: ST10, FeraccruTo 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.	Drug	16.6 mg Ferric Maltol, 30 mg Ferric Maltol, 7.8 mg Ferric Maltol

Primary outcome

Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 1
Time frame: 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)
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.
Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 10
Time frame: 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)
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.
Area Under The Curve [AUC] of Maltol Glucuronide on Day 1
Time frame: 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)
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.
Area Under The Curve [AUC] of Maltol Glucuronide on Day 10
Time frame: 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)
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.
Average Plasma Concentration [Cave(0-6h)] of Maltol Glucuronide on Day 10
Time frame: 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)
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.
Time of Maximum Plasma Concentration [Tmax] of Maltol Glucuronide on Day 1
Time frame: 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)
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.
Time of Maximum Plasma Concentration [Tmax] of Maltol Glucuronide on Day 10
Time frame: 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)
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.
Half Life [t1/2] of Maltol Glucuronide on Day 1
Time frame: 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)
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.
Ratio of Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 10/Day 1
Time frame: 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)
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.
Ratio of Area Under The Curve [AUC] of Maltol Glucuronide on Day 10/Day 1
Time frame: 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)
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.
Average Serum Concentration [Cave(0-6h)] of Iron on Day 10
Time frame: 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)
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.
Change From Pre-Dose (Ctrough) to Maximum Post-Dose (Cmax) in Serum Iron on Day 1
Time frame: 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)
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.
Change From Pre-Dose (Ctrough) to Maximum Post-Dose (Cmax) in Serum Iron on Day 10
Time frame: 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)
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.
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Serum Iron on Day 1
Time frame: 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)
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.
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Serum Iron on Day 10
Time frame: 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)
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.
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Transferrin Saturation (TSAT) on Day 1
Time frame: 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)
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.
Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Transferrin Saturation (TSAT) on Day 10
Time frame: 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)
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.
Apparent Systemic Clearance (CL/F) of Iron on Day 1
Time frame: 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)
Descriptive statistics and population PK analysis of serum iron CL/F from PK samples collected on Day 1
Apparent Systemic Clearance (CL/F) of Iron on Day 10
Time frame: 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)
Descriptive statistics and population PK analysis of serum iron CL/F from PK samples collected on Day 10
Apparent Volume of Distribution (V/F) of Iron on Day 1
Time frame: 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)
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.
Apparent Volume of Distribution (V/F) of Transferrin Saturation (TSAT) on Day 1
Time frame: 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)
Descriptive statistics and population PK analysis of TSAT V/F from PK samples collected on Day 1
Apparent Volume of Distribution (V/F) of Transferrin Saturation (TSAT) on Day 10
Time frame: 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)
Descriptive statistics and population PK analysis of TSAT V/F from PK samples collected on Day 10
Ratio Auc(0-6) Maltol Glucuronide Day 10/Day 1
Time frame: Day 1 to Day 10 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Ratio AUC0-6h measured after last dose of Ferric Maltol on Day 10 vs first dose Day 1.
Serum Iron - RAUC(0-6h) D10/D1
Time frame: 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)
Serum Iron - RAUC(0-6h) Day 10/Day 1
Transferrin Saturation (%) Day 1, Baseline
Time frame: Measured after first dose of Ferric Maltol on Day 1 (0h)
Transferrin Saturation (TSAT%) Day 1, baseline
Transferrin Saturation (%) Day 1, Maximum Response (%)
Time frame: Measured after first dose of Ferric Maltol on Day 1 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Transferrin Saturation (TSAT%) Day 1, maximum response (%)
Transferrin Saturation Day 1, Time to Maximum Response Tmax
Time frame: Measured after first dose of Ferric Maltol on Day 1. (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
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.
Transferrin Saturation (%) Day 10, Maximum Response (%)
Time frame: Measured after last dose of Ferric Maltol on Day 10. (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
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.
Transferrin Saturation Day 10, Time to Maximum Response Tmax
Time frame: Measured after first dose of Ferric Maltol on Day 10. (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h).
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.
AUC0-inf Day 1 for Maltol Glucuronide
Time frame: Measured after first dose of Ferric Maltol on Day 1 (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
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.
AUC0-tau Day 10 for Maltol Glucuronide
Time frame: Measured after last dose of Ferric Maltol on Day 10. (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
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.
Cthrough for Maltol Glucuronide Day 10
Time frame: Day 10 pre-dose to last (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
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.
Serum Iron Cmax Day 1
Time frame: Day 1 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
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.
Serum Iron Cmax on Day 10
Time frame: Day 10 (0h, 0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Maximum serum concentration of serum iron on Day 10.
Plasma Maltol Glucuronide Cthrough D10/Day1
Time frame: Day 10 (0.5-1h, 1-2h, 2-3h, 3-4h, 4-6h)
Minimum concentration between dose time and dose time+TAU
Apparent Systemic Clearance (CL/F) of Transferrin Saturation (TSAT) on Day 1
Time frame: 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
Descriptive statistics and population PK analysis of serum TSAT CL/F from PK samples collected on Day 1
Apparent Systemic Clearance (CL/F) of Transferrin Saturation (TSAT) on Day 10
Time frame: 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)
Descriptive statistics and population PK analysis of serum TSAT CL/F from PK samples collected on Day 10
Apparent Systemic Clearance (CL/F) of Maltol Glucuronide on Day 1
Time frame: 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
Descriptive statistics and population PK analysis of plasma maltol glucuronide CL/F from PK samples collected on Day 1.
Apparent Systemic Clearance (CL/F) of Maltol Glucuronide on Day 10
Time frame: 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
Descriptive statistics and population PK analysis of plasma maltol glucuronide CL/F from PK samples collected on Day 10.

12 secondary outcomes

Transferrin - Change From Baseline to Day 10, Predose
Time frame: Day 1 pre-dose to Day 10 pre-dose (0h on each day)
Ferritin - Change From Baseline to Day 10, Predose
Time frame: Pre-dose on Day 1 to Day 10 (0h)
Total Iron Binding Capacity - Change From Day 1 to Day 10, Predose
Time frame: Predose from Day 1 to Day 10 (0h on each day)
UIBC - Change From Day 1 to Day 10, Predose
Time frame: Pre-dose on Day 1 to Day 10 (0h each day)
Negative and Positive NTBI Tests on Day 1
Time frame: Day 1 (0h)
Change From Baseline to Day 10 in Haemoglobin Concentration
Time frame: Screening and Day 10 (1-4 hours post-dose)
Change From Baseline to Day 10 in Absolute Reticulocyte Count
Time frame: 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
Time frame: 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
Time frame: Screening and Day 10 (1-4 hours post-dose)
Concomitant Medications
Time frame: Screening, Day 1, Day 10 and Post-Study Follow-up visit, on average 4 weeks
Negative and Positive NTBI Tests on Day 10, Predose
Time frame: Day 10
Treatment-emergent Serious Adverse Event (TESAE)
Time frame: From first dose of ferric maltol Day 1 through study completions, on average 4 weeks

Results and safety

Results posted to the registry on 2 Jun 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
30 mg Ferric Maltol	12	12
16.6 mg Ferric Maltol	13	11
7.8 mg Ferric Maltol	12	12

Primary outcome: Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 1

Time frame: 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). Mean (Standard Deviation). Unit: mg/L

Measure	30 mg Ferric Maltol n = 12	16.6 mg Ferric Maltol n = 13	7.8 mg Ferric Maltol n = 12
Value	2.4518 (1.29590)	1.1632 (0.18435)	0.5299 (0.02245)

Primary outcome: Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 10

Time frame: 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). Mean (Standard Deviation). Unit: mg/L

Measure	30 mg Ferric Maltol n = 12	16.6 mg Ferric Maltol n = 13	7.8 mg Ferric Maltol n = 12
Value	4.2355 (0.78110)	2.1028 (0.13342)	0.9926 (0.02349)

Primary outcome: Area Under The Curve [AUC] of Maltol Glucuronide on Day 1

Time frame: 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). Mean (Standard Deviation). Unit: h*mg/L

Measure	30 mg Ferric Maltol n = 12	16.6 mg Ferric Maltol n = 13	7.8 mg Ferric Maltol n = 12
Value	11.090 (3.9033)	5.613 (0.7589)	2.585 (0.0968)

Population analysed: 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.

Primary outcome: Area Under The Curve [AUC] of Maltol Glucuronide on Day 10

Time frame: 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). Mean (Standard Deviation). Unit: h*mg/L

Measure	30 mg Ferric Maltol n = 12	16.6 mg Ferric Maltol n = 13	7.8 mg Ferric Maltol n = 12
Value	20.511 (1.7997)	10.518 (0.5277)	5.016 (0.1133)

12 secondary outcomes are reported in the full results.

Adverse events

Time frame: 10 days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
30 mg Ferric Maltol	0 of 12 (0%)	7 of 12 (58.3%)	0 of 12
16.6 mg Ferric Maltol	0 of 13 (0%)	7 of 13 (53.8%)	0 of 13
7.8 mg Ferric Maltol	0 of 12 (0%)	7 of 12 (58.3%)	0 of 12

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Iron Deficiency, Anaemia in Children

Not yet recruiting
Effect of Vitamin D Supplementation Added to Iron Therapy in Children With Iron Deficiency Anemia
Iron Deficiency Anemia
RecruitingObservational study
Maternal Iron Deficiency and Childhood Health
Iron Deficiency Anaemia in Childbirth, Iron Deficiency
RecruitingPhase 3
Multi-center Trial of Ferric Derisomaltose in Children 0 to <18 Years of Age With Iron Deficiency Anemia
Iron Deficiency, Anaemia in Children
Not yet recruitingPhase 2
COMPARISON OF EFFICACY OF ORAL VS IV IRON THERAPY IN CHILDREN AGED 12-59 MONTHS, WITH IRON DEFICIENCY ANEMIA
Iron Deficiency Anemia in Children
Recruiting
Efficacy of Oral Sucrosomial Iron Supplementation in Children With Celiac Disease and Iron Deficiency or Anemia
Celiac Disease in Children, Anemia

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.

7 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Ferric Maltol in people with Iron Deficiency, Anaemia in Children and Iron-Deficiency, run by Shield Therapeutics at 7 sites in United Kingdom.

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 5 inclusion and 17 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 6 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United Kingdom. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 2 Jun 2020). 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?

Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 1 (time frame: 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)). The study lists 39 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03181451.

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 6 Oct 2021.