Iron deficiency anaemia MedDRA version: 20.0 Level: LLT Classification code 10022974 Term: Iron deficiency anemia System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Product Name: Ferric maltol oral suspension
Product Code: ST10
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Ferric maltol
CAS Number: 33725-54-1
Current Sponsor code: ST10
Other descripti
Sponsors
Shield TX (UK) Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. 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. 2. Age =1 month and =17 years at the time of informed consent 3. 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 – =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject with anaemia due to any cause other than iron deficiency, including, but not limited to, a. Untreated or untreatable severe malabsorption syndrome 2. Subjects who have received prior to Screening: a. Within 28 days intramuscular or intravenous (IV) injection or administration of depot iron preparation. b. Within 7 days single agent iron preparations. c. Within 12 weeks of blood transfusion or is scheduled to have blood transfusion or donation during the study period d. Within 28 days erythropoiesis stimulating agents and during the study e. Within 14 days COVID-19 vaccination 3. 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. 4. Has concomitant disease that would significantly compromise iron absorption or absorbed iron utilization such as swallowing disorders and/or extensive small bowel resection. 5. History of active peptic ulcer. 6. Has chronic renal disease (eGFR 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 hospitalization and/or surgery during the course of the study 15. Participation in any other interventional clinical study within 28 days prior to Screening. 16. Diagnosed to be COVID-19 positive by (SARS-CoV-2-RT-PCR positive) within 28 days prior to screening. 17. 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. 18. Any other unspecified reason that, in the opinion of the Investigator or the Sponsor make the subject unsuitable for enrolment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: To assess the pharmacokinetics (PK) in children and adolescents aged 2 to 17 years after a single dose of ferric maltol oral suspension on Visit 2, and after twice daily administration for at least 6 days, on Visit 3 after a single morning dose, through measurement of serum iron, corrected serum iron, transferrin saturation (TSAT) and plasma maltol and maltol glucuronide. To assess the effect on iron markers in children and adolescents aged 1 month to 17 years after twice daily ferric maltol oral suspension administration for 12 weeks. To assess the PK in children aged 1 month to less than 2 years of age after a single dose of ferric maltol oral suspension (Pre-assignment PK visit) and after twice daily administration for at least 6 days, on Visit 3 after a single morning dose, through measurement of serum iron, corrected serum iron, transferrin saturation (TSAT), plasma and urine concentration of maltol and maltol glucuronide.;Primary end point(s): Safety and gastrointestinal tolerability: - Treatment emergent Adverse Events (TEAE) - Treatment-emergent Serious Adverse Events (TESAEs) - Treatment-emergent Adverse Events leading to premature discontinuation of study drug/PK assessments - Change in Hb concentration ;Timepoint(s) of evaluation of this end point: Week 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - PK analysis of serum iron, corrected serum iron, TSAT, transferrin, TIBC, UIBC, maltol and maltol glucuronide in children and adolescents aged 1 month to 17 years in the ferric maltol group - Changes in iron markers from baseline to Week 12 - Achieving Hb concentration within normal range at Week 12 - Qualitative assessments from subject questionnaires that allow evaluation of the acceptability, palatability and ease of use -Age 1 month to less than 2 years; maltol and maltol glucuronide in urine from both PK days in children aged 1 month to less than 2 years;Timepoint(s) of evaluation of this end point: Week 12 | — |
Countries
United Kingdom, United States
Contacts
Public ContactClinical Operations
Shield TX (UK) Ltd.
Outcome results
None listed