Evaluate the Safety and Efficacy of Ferric Maltol Oral Suspension vs. Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, With a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years

NCT05126901 | Completed Phase 3 Results FDA Drug

• 1 other identifier: ST10-01-305
• Study Type: interventional
• Target: 65
• Locations: 3 countries
• Sites: 23

Brief Summary

The objective of the study is to compare the safety and gastrointestinal tolerability of ferric maltol oral suspension and ferrous sulfate oral liquid in children and adolescents aged 2 years to 17 years, and assess the safety and tolerability of ferric maltol oral suspension in children 1 month to less than 2 years, in the treatment of iron deficiency anaemia during the 12 weeks treatment period.

Conditions

Anemia; Iron-deficiency

Keywords

Anemia; Iron-Deficiency

Outcome Measures

Primary Outcomes (1)

• Change in Hemoglobin Concentration

  The change in Hb concentration from baseline to Week 12 is summarized based on the mITT Population for each treatment group using descriptive statistics summarized by mean, standard deviation, median, and range (minimum and maximum). The Randomized/ITT Population is defined as all patients who were randomized/assigned to treatment arms. The mITT Population is defined as all patients in the ITT Population who received at least 1 treatment dose.

  From baseline to week 12.

Secondary Outcomes (9)

• Changes in Ferritin Concentration

  From baseline to week 12.

• Change in Iron Concentration

  From baseline to week 12.

• Change in the Percentage of Transferrin Saturation

  From baseline to week 12.

• Cmax for Plasma Maltol Glucuronide

  PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).

• Tmax for Plasma Maltol Glucuronide

  PK parameters assessed at Day 1 (Visit 2) and Day 7-10 (Visit 3).

Study Arms (3)

1 month to 2 year old subjects (infants)

EXPERIMENTAL

Subjects aged 1 month to less than 2 years will enter a Pre-assignment phase: baseline urine samples are collected and subjects will take a single dose of 0.1 ml/kg ferric maltol suspension. Further 3 samples up to 12h will be taken. Subjects showing evidence of absorption, metabolism and elimination of maltol will enter the treatment phase and be assigned to the ferric maltol arm. The first 6 subjects screened will perform the pre-assignment PK phase. After review by the investigator, and medical monitors , if Maltol Glucuronide is shown to be adequately eliminated, timepoint 20-24 hrs (+ 4hrs) will not be performed on subsequent subjects. Subjects will be assigned to receive ferric maltol oral suspension and start the 0.1 ml/kg BID dose on V2 and continue for 7-10 days. On V3 they will perform the same PK assessments as on Pre-assignment PK visit.

Drug: Ferric Maltol

2 to 17 year old subjects - Ferric Maltol

EXPERIMENTAL

Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. The first 12 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs respectively) will enter a PK phase with 2 PK days. Following PK Day 2 subjects will continue until Week 12. Once the 18 subjects in each age subgroup have finished their PK visits, they will continue until week 12. Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose will be used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2 - 17 yrs will be dosed 6 mg/kg to the maximum of 4 ml BID. Subjects randomised to ferrous sulfate oral liquid will not need to complete the PK period.

Drug: Ferric Maltol

2 to 17 year old subjects - Ferrous Sulfate

ACTIVE COMPARATOR

Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. Ferrous sulfate 125 mg/ml (25 mg elemental iron) or equivalent dose will be used for all children/adolescents. To maximise the iron replenishment for subjects within this group as well; aged 2 - 17 yrs will be dosed 6 mg/kg to the maximum of 4 ml BID. Subjects randomised to ferrous sulfate oral liquid will not need to complete the PK period.

Drug: Ferrous sulfate

Interventions

Ferric Maltol DRUG

Ferric maltol oral suspension: 150 ml amber glass bottle with graduated syringe and adaptor. Oral suspension containing 30 mg elemental iron, in the form of 231.5 mg ferric maltol, in 5 ml suspension Study dosage: The dose of ferric maltol oral suspension that will be administered for children aged 1 month to \< 2 yrs: 0.1 ml/kg BID, 2 to - 11 yrs: 2.5 ml BID, 12-17 yrs: 5 ml BID.

Also known as: Feraccru, Ferric Trimaltol, ST10, ST10-01

1 month to 2 year old subjects (infants); 2 to 17 year old subjects - Ferric Maltol

Ferrous sulfate DRUG

Ferrous sulfate 125 mg/ml (25 mg/ml elemental iron) oral liquid : 15 ml glass bottle. Study dosage: For ferrous sulfate oral liquid, the dose administered will be for children and adolescents aged 2 years to 17 yrs: 6 mg/kg to the maximum of 4 ml BID.

2 to 17 year old subjects - Ferrous Sulfate

Eligibility Criteria

• Age: 1 Month - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patient is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.
• Age ≥1 month and ≤17 years at the time of informed consent
• Subjects must have iron deficiency anaemia defined by the following criteria, as measured by the central laboratory at the screening visit
• Haemoglobin thresholds define anaemia by age and gender:
• Children (1 m - \< 5 yrs) \<11.0 g/dl Children (5 yrs - \< 12 yrs) \<11.5 g/dl Children (12 yrs) \<12.0 g/dl Female child (≥13 yrs) \<12.0 g/dl Male child (≥13 yrs) \<13.0 g/dl and
• Ferritin thresholds define anaemia by:
• ferritin \<30 µg/L, or ferritin \<50 µg/L with transferrin saturation (TSAT) \<20%, 4. Female subjects of childbearing potential must agree to use a highly effective method of contraception (which includes complete abstinence) until study completion and for at least 4 weeks following their final study visit. Highly effective contraception is defined as a method which results in a low failure rate, i.e., less than 1% per year when used consistently and correctly, such as implants, injectables, some intrauterine contraceptive devices (IUDs), a vasectomised partner and oral contraceptive medications.
• The need for contraception and compliance with contraception requirements will be assessed at every visit for adolescent patients, and urine pregnancy testing will be performed at each visit for female subjects of childbearing potential.

You may not qualify if:

• Subject with anaemia due to any cause other than iron deficiency, including, but not limited to,
• a. Untreated or untreatable severe malabsorption syndrome
• Subjects who have received prior to Screening:
• Within 28 days intramuscular or intravenous (IV) injection or administration of depot iron preparation.
• Within 7 days single agent iron preparations and during the study.
• Within 12 weeks of blood transfusion or is scheduled to have blood transfusion or donation during the study period
• Within 28 days erythropoiesis stimulating agents and during the study period
• Within 14 days COVID-19 vaccination
• Subjects with vitamin B12 or folic acid deficiency as determined by the central laboratory screening results. Subjects may start vitamin B12 or folate replacement and rescreen after at least 2 weeks.
• Has concomitant disease that would significantly compromise iron absorption or absorbed iron utilization such as swallowing disorders and/or extensive small bowel resection.
• History of active peptic ulcer
• Has chronic renal disease (eGFR \<60 mL/min/m2), as assessed at Screening based on serum creatinine.
• Known hypersensitivity or allergy to either the active substance or excipients of ferric maltol or ferrous sulfate.
• Has a known contraindication for treatment with iron preparations, e.g. haemochromatosis, chronic haemolytic disease, sideroblastic anaemia, thalassemia, or lead intoxication induced anaemia.
• Impaired liver function as indicated by alanine aminotransferase (ALT) or aspartate transaminase (AST)\>2.0 times upper normal limit as measured at the Screening visit.

Sponsors & Collaborators

• Shield Therapeutics: lead

Study Sites (23)

The Center for Clinical Trials

Saraland, Alabama, 36571, United States

Location

Homestead Research Institute

Homestead, Florida, 33030, United States

Location

Kissimmee Clinical Research Corp

Kissimmee, Florida, 34743, United States

Location

Miami Clinical Research

Miami, Florida, 33155, United States

Location

Medical Research of Westcheste

Miami, Florida, 33165, United States

Location

Eminent Clinical Research and Associates

North Lauderdale, Florida, 33068, United States

Location

Clinical Research Prime

Idaho Falls, Idaho, 83404, United States

Location

Sierra Clinical Research

Las Vegas, Nevada, 89106, United States

Location

Levine Cancer Institute

Charlotte, North Carolina, 28204, United States

Location

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, 17033, United States

Location

Hasbro Children's Hospital

Providence, Rhode Island, 02903, United States

Location

BRCR Global Texas

Edinburg, Texas, 78539, United States

Location

Zion Research

Katy, Texas, 77494, United States

Location

MultiCare Health System Institute for Research and Innovation

Tacoma, Washington, 98405, United States

Location

BRCR Global Puerto

San Juan, 00907, Puerto Rico

Location

Noah's Ark Children's Hospital for Wales

Cardiff, CF14 4XW, United Kingdom

Location

Royal Hospital for Sick Children - Edinburgh

Edinburgh, EH16 4TJ, United Kingdom

Location

Leicester Royal Infirmary

Leicester, LE1 5WW, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, United Kingdom

Location

King's College Hospital

London, SE5 9RS, United Kingdom

Location

Newham University Hospital

London, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

Nottingham University Hospitals

Nottingham, NG7 2UH, United Kingdom

Location

MeSH Terms

Conditions

Anemia; Anemia, Iron-Deficiency

Interventions

ferric maltol; ferric trimaltol; ferrous sulfate

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia, Hypochromic; Iron Deficiencies; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Clinical Operations, Shield TX (UK) Ltd.
• Organization: Shield TX (UK) Ltd.

info@shieldtherapeutics.com

+44 (0) 191 511 8500

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: 12 weeks open label treatment
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The study will include 98 subjects in the 2 - 17 years age group, randomised 1:1 between ferric maltol and ferrous sulfate: 49 in each treatment group. The study will also include up to 12 subjects in the 1 month to less than 2 years age group. If less than 91 subjects in total have been randomized when 32 ferric maltol subjects have completed, then an interim analysis will be conducted. If significant, the study will stop recruitment. If not significant, the study will continue until 54 subjects have been recruited in the ferric maltol arm.

Study Record Dates

• First Submitted: October 27, 2021
• First Posted: November 19, 2021
• Study Start: November 3, 2021
• Primary Completion: June 9, 2024
• Study Completion: June 9, 2024
• Last Updated: November 10, 2025
• Results First Posted: November 10, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

GB (8); PR (1); US (14)