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

Shield Therapeutics

Status and phase: Completed; Phase 1

Conditions: Iron-Deficiency; Iron Deficiency, Anaemia in Children

Treatments: Drug: Ferric Maltol

Study type: Interventional

Funder types: Industry

Identifiers

NCT03181451

ST10-01-103

Details and patient eligibility

About

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.

Full 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

Enrollment: 37 patients

Sex: All

Ages: 10 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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37 participants in 3 patient groups

30 mg Ferric Maltol

Active Comparator group

Description:

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.

Treatment:

Drug: Ferric Maltol

16.6 mg Ferric Maltol

Active Comparator group

Description:

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.

Treatment:

Drug: Ferric Maltol

7.8 mg Ferric Maltol

Active Comparator group

Description:

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.

Treatment:

Drug: Ferric Maltol