Phase 1
N=37
Evaluate the PK, Safety, Tolerability of Ferric Maltol at 3 Dosage Levels in Paediatric Subjects With Iron Deficiency
Iron Deficiency, Anaemia in Children · Iron-Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT03181451 ↗Enrolled (actual)
37
Serious AEs
0.0%
Results posted
Jun 2020
Primary outcome: Primary: Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 1 — 2.4518; 1.1632; 0.5299 mg/L
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Ferric Maltol (Drug)
- Age
- Pediatric · 10+ yrs
- Sex
- All
- Sponsor
- Shield Therapeutics
- Primary completion
- Mar 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 1 |
2.4518; 1.1632; 0.5299 | — |
| PRIMARY Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 10 |
4.2355; 2.1028; 0.9926 | — |
| PRIMARY Area Under The Curve [AUC] of Maltol Glucuronide on Day 1 |
11.090; 5.613; 2.585 | — |
| PRIMARY Area Under The Curve [AUC] of Maltol Glucuronide on Day 10 |
20.511; 10.518; 5.016 | — |
| PRIMARY Average Plasma Concentration [Cave(0-6h)] of Maltol Glucuronide on Day 10 |
3.4186; 1.7531; 0.8360 | — |
| PRIMARY Time of Maximum Plasma Concentration [Tmax] of Maltol Glucuronide on Day 1 |
1.00; 1.00; 1.00 | — |
| PRIMARY Time of Maximum Plasma Concentration [Tmax] of Maltol Glucuronide on Day 10 |
0.75; 0.75; 0.75 | — |
| PRIMARY Half Life [t1/2] of Maltol Glucuronide on Day 1 |
11.384; 10.447; 10.806 | — |
| PRIMARY Ratio of Maximum Plasma Concentration [Cmax] of Maltol Glucuronide on Day 10/Day 1 |
1.956; 1.836; 1.875 | — |
| PRIMARY Ratio of Area Under The Curve [AUC] of Maltol Glucuronide on Day 10/Day 1 |
2.030; 1.899; 1.942 | — |
| PRIMARY Average Serum Concentration [Cave(0-6h)] of Iron on Day 10 |
1.1667; 0.9557; 0.9748 | — |
| PRIMARY Change From Pre-Dose (Ctrough) to Maximum Post-Dose (Cmax) in Serum Iron on Day 1 |
0.6159; 0.4102; 0.21715 | — |
| PRIMARY Change From Pre-Dose (Ctrough) to Maximum Post-Dose (Cmax) in Serum Iron on Day 10 |
0.5486; 0.3625; 0.2251 | — |
| PRIMARY Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Serum Iron on Day 1 |
6.711; 5.963; 3.928 | — |
| PRIMARY Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Serum Iron on Day 10 |
7.000; 5.734; 5.849 | — |
| PRIMARY Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Transferrin Saturation (TSAT) on Day 1 |
179.47; 159.21; 104.50 | — |
| PRIMARY Pre-dose Adjusted Incremental Area Under the Curve [AUC(0-6h)] of Transferrin Saturation (TSAT) on Day 10 |
180.14; 149.40; 157.43 | — |
| PRIMARY Apparent Systemic Clearance (CL/F) of Iron on Day 1 |
1.321; 0.654; 0.324 | — |
| PRIMARY Apparent Systemic Clearance (CL/F) of Iron on Day 10 |
3.264; 1.757; 0.808 | — |
| PRIMARY Apparent Volume of Distribution (V/F) of Iron on Day 1 |
22.114; 14.244; 10.462 | — |
| PRIMARY Apparent Volume of Distribution (V/F) of Transferrin Saturation (TSAT) on Day 1 |
— | — |
| PRIMARY Apparent Volume of Distribution (V/F) of Transferrin Saturation (TSAT) on Day 10 |
— | — |
| PRIMARY Ratio Auc(0-6) Maltol Glucuronide Day 10/Day 1 |
1.942; 1.899; 2.030 | — |
| PRIMARY Serum Iron - RAUC(0-6h) D10/D1 |
1.529; 1.020; 1.038 | — |
| PRIMARY Transferrin Saturation (%) Day 1, Baseline |
11.5; 16.8; 15.8 | — |
| PRIMARY Transferrin Saturation (%) Day 1, Maximum Response (%) |
18.68; 28.261; 32.845 | — |
| PRIMARY Transferrin Saturation Day 1, Time to Maximum Response Tmax |
4.00; 3.00; 3.00 | — |
| PRIMARY Transferrin Saturation (%) Day 10, Maximum Response (%) |
27.779; 27.214; 33.524 | — |
| PRIMARY Transferrin Saturation Day 10, Time to Maximum Response Tmax |
3.00; 3.00; 3.00 | — |
| PRIMARY AUC0-inf Day 1 for Maltol Glucuronide |
8.590; 17.862; 34.372 | — |
| PRIMARY AUC0-tau Day 10 for Maltol Glucuronide |
8.59; 17.862; 34.368 | — |
| PRIMARY Cthrough for Maltol Glucuronide Day 10 |
0.4804; 0.9762; 1.8496 | — |
| PRIMARY Serum Iron Cmax Day 1 |
0.7030; 1.681; 1.2328 | — |
| PRIMARY Serum Iron Cmax on Day 10 |
1.0338; 1.0462; 1.3034 | — |
| PRIMARY Plasma Maltol Glucuronide Cthrough D10/Day1 |
0.4804; 0.9762; 1.8496 | — |
| PRIMARY Apparent Systemic Clearance (CL/F) of Transferrin Saturation (TSAT) on Day 1 |
— | — |
| PRIMARY Apparent Systemic Clearance (CL/F) of Transferrin Saturation (TSAT) on Day 10 |
— | — |
| PRIMARY Apparent Systemic Clearance (CL/F) of Maltol Glucuronide on Day 1 |
— | — |
| PRIMARY Apparent Systemic Clearance (CL/F) of Maltol Glucuronide on Day 10 |
— | — |
| SECONDARY Transferrin - Change From Baseline to Day 10, Predose |
-0.085; -0.147; 0.032 | — |
| SECONDARY Ferritin - Change From Baseline to Day 10, Predose |
7.3; 4.1; 0.5 | — |
| SECONDARY Total Iron Binding Capacity - Change From Day 1 to Day 10, Predose |
-2.12; -3.11; 1.13 | — |
| SECONDARY UIBC - Change From Day 1 to Day 10, Predose |
-2.10; -2.33; -4.49 | — |
| SECONDARY Negative and Positive NTBI Tests on Day 1 |
0; 0; 0; 12; 13; 12 | — |
| SECONDARY Change From Baseline to Day 10 in Haemoglobin Concentration |
-0.03; -0.33; -0.45 | — |
| SECONDARY Change From Baseline to Day 10 in Absolute Reticulocyte Count |
0.036; -0.001; 0.016 | — |
| SECONDARY Treatment-emergent Adverse Events (AEs) Leading to Premature Discontinuation of Study Drug/PK Assessments |
0; 1; 0 | — |
| SECONDARY Changes in 12-lead ECG Parameters From Screening to Day 10 |
10; 11; 12; 0; 0; 0 | — |
| SECONDARY Concomitant Medications |
8; 13; 10 | — |
| SECONDARY Negative and Positive NTBI Tests on Day 10, Predose |
1; 0; 0; 11; 11; 10 | — |
| SECONDARY Treatment-emergent Serious Adverse Event (TESAE) |
0; 0; 0 | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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 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.
Data sourced from ClinicalTrials.gov (NCT03181451). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.