Multi-center Trial of Ferric Derisomaltose in Children 0 to <18 Years of Age With Iron Deficiency… (NCT05179226) | Clinical Trial Compass
RecruitingPhase 3
Multi-center Trial of Ferric Derisomaltose in Children 0 to <18 Years of Age With Iron Deficiency Anemia
United States200 participantsStarted 2022-11-28
Plain-language summary
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.
Who can participate
Age range
17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 t…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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.
Timeframe: From baseline at any time from week 1 to week 8
Trial details
NCT IDNCT05179226
SponsorPharmacosmos A/S
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2026-04-30
Contact for this trial
Pharmacosmos A/S Clinical and Non-clinical Research