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 (FORTIS)
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| ClinicalTrials.gov Identifier: NCT05126901 |
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Recruitment Status :
Recruiting
First Posted : November 19, 2021
Last Update Posted : November 19, 2021
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Anemia Iron-deficiency | Drug: Ferric Maltol Drug: Ferrous sulfate | Phase 3 |
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| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 110 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Intervention Model Description: | 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. |
| Masking: | None (Open Label) |
| Masking Description: | 12 weeks open label treatment |
| Primary Purpose: | Treatment |
| Official Title: | Randomised, Open-label, Active-controlled, Multicentre, Comparative Study to Evaluate the Safety and Efficacy of Ferric Maltol (Iron (III)-Maltol Complex) (ST10) Oral Suspension Compared to Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, Incorporating a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years |
| Actual Study Start Date : | October 4, 2021 |
| Estimated Primary Completion Date : | May 2023 |
| Estimated Study Completion Date : | September 2023 |
| Arm | Intervention/treatment |
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Experimental: 1 month to 2 year old subjects (infants)
Subjects aged 1 month to less than 2 years will enter a Pre-assignment phase: baseline pre-dose blood and urine sample are collected and subjects will take a single dose of 0.1 ml/kg ferric maltol suspension under supervision. Further 3 PK blood samples up to 6 h and urine samples from 2 timepoints will be taken. Subjects showing evidence of absorption, metabolism of serum iron and elimination of maltol will enter the treatment phase and be assigned to the ferric maltol arm. 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
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. Other Names:
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Experimental: 2 to 17 year old subjects - Ferric Maltol
Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. The first 18 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs) 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
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. Other Names:
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Active Comparator: 2 to 17 year old subjects - Ferrous Sulfate
Subjects aged 2-17 will be randomised 1:1 to receive ferric maltol oral suspension or ferrous sulfate oral liquid. The first 18 subjects randomised to ferric maltol in each age sub-group (2 - 9 yrs, 10 - 17 yrs) 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: Ferrous sulfate
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. |
- Safety and gastrointestinal tolerability will be compared between ferric maltol oral suspension and ferrous sulfate oral liquid via summaries of treatment emergent adverse events (TEAEs), treatment emergent serious AEs (TESAEs) and treatment-emergent AEs [ Time Frame: 12 weeks ]
Safety and gastrointestinal tolerability will be compared between ferric maltol oral suspension and ferrous sulfate oral liquid via the incidence of treatment emergent adverse events (TEAEs), treatment emergent serious AEs (TESAEs) and treatment-emergent AEs (TEAEs) leading to premature discontinuation of study drug, estimated as the number of subjects with at least one event divided by the number of subjects in the safety population.
AEs will be categorised by primary system organ class and MedDRA preferred term as coded using the MedDRA dictionary. The number, intensity, relation to study medication and action taken will be described by incidence tables. SAEs will be discussed separately.
- Assess the PK in children and adolescents aged 2 to 17 years [ Time Frame: 12 weeks ]To assess the pharmacokinetics (PK) in children and adolescents aged 2 to 17 years after a single dose of ferric maltol oral suspension Visit 2 (PK Day 1), and after twice daily administration for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose, through measurement of serum iron, transferrin saturation (TSAT) and plasma maltol and maltol glucuronide
- Assess the effect on haemoglobin and iron markers in children and adolescents aged 1 month to 17 years after twice daily ferric maltol oral suspension administration for 12 weeks [ Time Frame: 12 weeks ]
- Assess the PK, in children aged 1 month to less than 2 years of age [ Time Frame: 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 (PK Day 2) after a single morning dose, through measurement of serum iron, transferrin saturation (TSAT), plasma and urine concentration of maltol and maltol glucuronide
- Assess the effect, in children aged 1 month to less than 2 years of age [ Time Frame: 12 weeks ]To assess the effect, in children aged 1 month to less than 2 years of age after a single dose of ferric maltol suspension (Pre-assignment PK visit), and after twice daily administration for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose, on serum transferrin, total and unsaturated iron binding capacity (TIBC, UIBC), ferritin
- Assess the effect, in children aged 2 to 17 [ Time Frame: 12 weeks ]To assess the effect, in children aged 2 to 17 after a single dose of ferric maltol suspension Visit 2 (PK Day 1), and after twice daily administration for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose, on serum transferrin, total and unsaturated iron binding capacity (TIBC, UIBC), ferritin.
- To compare the palatability from age-appropriate scoring system of ferric maltol oral suspension and ferrous sulfate oral liquid [ Time Frame: 12 weeks ]
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| Ages Eligible for Study: | 1 Month to 17 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
Exclusion Criteria:
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Subject with anaemia due to any cause other than iron deficiency, including, but not limited to,
a. Untreated or untreatable severe malabsorption syndrome
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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 7 days multivitamins that includes iron 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.
- 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.
- Diagnosed to be COVID-19 positive by (SARS-CoV-2-RT-PCR positive) 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05126901
| Contact: Houda Maaraf | +447562586332 | hmaaraf@shieldtx.com | |
| Contact: Jackie Mitchell | +440191 511 8515 | jmitchell@shieldtx.com |
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| Responsible Party: | Shield Therapeutics |
| ClinicalTrials.gov Identifier: | NCT05126901 |
| Other Study ID Numbers: |
ST10-01-305 |
| First Posted: | November 19, 2021 Key Record Dates |
| Last Update Posted: | November 19, 2021 |
| Last Verified: | November 2021 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Anemia Iron-Deficiency |
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Anemia Anemia, Iron-Deficiency Hematologic Diseases Anemia, Hypochromic |
Iron Metabolism Disorders Metabolic Diseases Ferric maltol Hematinics |