Treatment of Iron Deficiency Anemia in Children with Ferric Derisomaltose
A Phase III, Prospective, Open-label, Multi-center Trial of Ferric Derisomaltose in Children 0 to <18 Years of Age With Iron Deficiency Anemia Due to NDD-CKD or With Iron Deficiency Anemia Who Are Intolerant or Unresponsive to Oral Iron
This study is testing if an intravenous iron treatment can help children with iron deficiency anemia who can't take regular iron pills feel better and improve their iron levels.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 200 (estimated) |
| Ages | N/A to 17 Years |
| Sex | All |
| Sponsor | Pharmacosmos A/S Industry-sponsored |
| Drugs / interventions | chemotherapy |
| Locations | 1 site (Miami, Florida) |
| Trial ID | NCT05179226 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the efficacy and safety of intravenous ferric derisomaltose in children aged 0 to less than 18 years who have iron deficiency anemia (IDA) due to non-dialysis dependent chronic kidney disease or who are intolerant or unresponsive to oral iron. The study is a prospective, open-label, multi-center trial involving 200 subjects, with specific pharmacokinetic assessments for a subset of 24 participants. Subjects will receive ferric derisomaltose at doses of either 10 mg/kg or 20 mg/kg, with a maximum dose of 1000 mg, and blood samples will be collected to analyze iron levels post-treatment.
Who should consider this trial
Good fit: Ideal candidates include children under 18 years with iron deficiency anemia related to non-dialysis dependent chronic kidney disease or those who cannot tolerate or respond to oral iron.
Not a fit: Patients who do not have iron deficiency anemia or those with conditions that contraindicate the use of intravenous iron may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide an effective intravenous option for managing iron deficiency anemia in children, improving their health outcomes.
How similar studies have performed: While ferric derisomaltose has been studied in adults, this trial represents a novel approach as it focuses on a pediatric population with iron deficiency anemia.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subjects \<18 years * Informed consent and child assent, as age-appropriate, obtained before any trial- related activities and willingness to participate. LAR of the subject must sign and date the ICF (according to local requirements). The child must sign and date the CAF or provide oral assent, if required according to local requirements * IDA caused by different etiologies such as gastrointestinal disease, NDD-CKD, or other conditions leading to IDA * Hb concentration less than the 5th percentile for age and sex-specific reference range (Appendix B) * Subjects with NDD-CKD (a) or who are intolerant or unresponsive to oral iron (b): a) Subjects with NDD-CKD: * TSAT ≤35 % or s-ferritin \<100 ng/mL * Estimated glomerular filtration rate (eGFR) \<60 mL/min/1.73m2 * If on ESA, receiving stable ESA regimen defined as dose adjustments no more than ± 20 % for ≥8 weeks prior to screening b) Subjects with documented history of intolerance or unresponsiveness to oral iron therapy for at least one month prior to trial enrolment. * TSAT ≤20 % or s-ferritin \<100 ng/mL Exclusion Criteria: * Anemia caused by factors other than IDA according to Investigator's judgment * S-ferritin \>600 ng/mL * Hb ≤5.0 g/dL * Iron overload or disturbances in utilization of iron (e.g. hemochromatosis and hemosiderosis) * ALAT and/or ASAT \>2 times upper limit of normal (e.g. decompensated liver cirrhosis or active hepatitis) * Pregnant or nursing female subjects. In order to avoid pregnancy, female subjects of childbearing potential have to use adequate contraception (e.g. intrauterine devices, hormonal contraceptives, or double barrier method) or be abstinent during the whole trial period and 7 days after the last dosing. Childbearing potential refers to all female subjects ≥12 years old or \<12 years old who have started menstruating * Previous serious hypersensitivity reactions to any IV iron compounds including ferric derisomaltose * Received an investigational drug within 30 days prior to screening * Treatment with IV iron within 10 days prior to screening * Treatment with blood transfusion, radiotherapy, chemotherapy or other drugs that suppress the bone marrow, and drugs which have anemia as side effect within 30 days prior to screening * Planned elective surgery (or planned surgery during the trial period) where significant blood loss is expected within the last 30 days prior to screening * Any non-viral infection (non-viral infection that has been fully treated before the baseline visit is accepted) * Any other laboratory abnormality, medical condition, or psychiatric disorders which, in the opinion of the Investigator, will put the subject's disease management at risk or may result in the subject being unable to comply with the trial requirements
Where this trial is running
Miami, Florida
- Pharmacosmos Investigational Site — Miami, Florida, United States (Recruiting)
Study contacts
- Study coordinator: Pharmacosmos A/S Clinical and Non-clinical Research
- Email: info@pharmacosmos.com
- Phone: +4559485959
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.