Several clinical trials have been reported for ferric derisomaltose where it has been shown to be well tolerated and to improve markers of IDA. All clinical trials with ferric derisomaltose have been performed in adults, however, IDA is not specific to the adult population. In fact, children are likely to develop IDA due to their rapid growth.
The aim in this trial is to evaluate the efficacy and safety of intravenous (IV) ferric derisomaltose in children 0 to \<18 years of age with IDA due to NDD-CKD or with IDA who are intolerant or unresponsive to oral iron .
The subjects will receive ferric derisomaltose/iron isomaltoside 1000 (Monoferric®/Monofer®), at single doses of 10 mg/kg or 20 mg/kg with a maximal dose of 1000 mg.
24 subjects will be part of a PK assessment, meaning that more blood samples will be drawn within the first week after treatment. The blood samples will be used for analysis of the amount of total iron in the blood from treatment is given to day 7.
For the individual subject, duration of the trial will be approximately 10 weeks (including a 14-day screening period) and each subject will attend 6-9 visits. Subjects who will be included in the PK assessments will attend 8 (subjects age 6 to \<12 years old and 0 to \<6 years old) or 9 (subjects age 12 to \<18 years old) visits, while the other subjects will attend 6 visits.
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)
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)
Single group
Ferric Derisomaltose
DrugAll subjects will be treated with Ferric derisomaltose 20 mg/kg at baseline visit except for 12 subjects included in the PK-group, who will be treated with 10 mg/kg
Incidence of subjects with a Hb increase of ≥1 g/dL (NDD-CKD) or 2 g/dL (intolerant or unresponsive to oral iron). Measurement by bloodsample.
Hb (g/dL), measurement by bloodsample analysis
Time to increase Hb ≥1 g/dL (NDD-CKD) or 2 g/dL (intolerant or unresponsive to oral iron). Measurement by bloodsample.
Hb (g/dL), measurement by bloodsample analysis
Incidence of subjects who achieve a serum (s-) ferritin of ≥100 ng/mL. Measurement by bloodsample.
s-ferritin (ng/mL), measurement by bloodsample analysis
Incidence of subjects who achieve a TSAT of ≥35 % (NDD-CKD) or ≥20 % (intolerant or unresponsive to oral iron). Measurement by bloodsample.
TSAT (%), measurement by bloodsample analysis
Total iron PK parameters: AUC0-∞
Total iron (µg/dL), measurement by bloodsample analysis
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