Ferric Derisomaltose for Iron Deficiency Anemia in Children

Overseen By Pharmacosmos A/S Clinical and Non-clinical Research

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Pharmacosmos A/S

Must not be taking: IV iron, Chemotherapy

Disqualifiers: Pregnancy, Iron overload, Liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 9

Duration: 8 weeks

200 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of an IV treatment called ferric derisomaltose for children with iron deficiency anemia (IDA), a condition where the body lacks enough iron to produce healthy red blood cells. It targets children with IDA due to chronic kidney issues or those who cannot tolerate or do not respond to oral iron supplements. Participants will receive one of two doses of the treatment, and the trial will last about 10 weeks with several visits. Suitable candidates are children under 18 who experience IDA due to specific conditions, such as kidney problems, or have a history of not responding to iron pills. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants early access to potentially effective treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot have received IV iron within 10 days before screening or certain treatments like blood transfusions or chemotherapy within 30 days before screening.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that ferric derisomaltose is generally safe for people with iron deficiency anemia (IDA). Studies have found it effective in boosting iron levels, and its safety has been tested in over 3,000 patients with IDA. While most research has focused on adults, studies with children have also shown promising results.

In children with digestive problems, ferric derisomaltose has improved iron levels without causing serious side effects. Some mild side effects, such as headaches or dizziness, have been reported, but they are rare.

This trial aims to confirm the treatment's safety for children, even though it is already approved for adults with IDA. The existing approval for adults suggests some level of safety, but this trial will help ensure its suitability for children. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for iron deficiency anemia?

Ferric Derisomaltose is unique because it offers a new way to treat iron deficiency anemia in children with potentially quicker results. Unlike traditional oral iron supplements, which often take weeks to improve symptoms and can cause gastrointestinal discomfort, Ferric Derisomaltose is administered intravenously, allowing for faster replenishment of iron stores in the body. Researchers are excited about this treatment because it can deliver a higher dose of iron directly into the bloodstream, which might lead to more rapid and efficient correction of anemia. This could be particularly beneficial for children who have trouble tolerating oral iron or need a swift improvement in their iron levels.

What evidence suggests that ferric derisomaltose might be an effective treatment for iron deficiency anemia in children?

Research has shown that ferric derisomaltose effectively treats iron deficiency anemia (IDA) in children. Studies have found that it quickly and consistently boosts iron levels in young patients. In past trials, ferric derisomaltose was well-tolerated and successfully improved iron levels. While most research has focused on adults, children with IDA, especially those who don't respond to or can't tolerate iron pills, can also benefit. This treatment delivers iron directly into the bloodstream, bypassing the digestive system where absorption problems might occur. Early findings suggest it could be a promising option for kids with low iron levels. In this trial, all participants will receive ferric derisomaltose, with some receiving a dosage of 10 mg/kg and others 20 mg/kg. ^{1 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children under 18 with Iron Deficiency Anemia (IDA) who haven't responded to or can't tolerate oral iron. It includes those with non-dialysis-dependent chronic kidney disease (NDD-CKD), provided they have specific blood test results and are not on certain treatments like IV iron, blood transfusions, or chemotherapy recently.

Inclusion Criteria

I am under 18 and have both my consent and child assent for trial participation.

I have iron deficiency anemia due to conditions like stomach issues or kidney disease.

I have a history of not responding well to oral iron treatments.

Exclusion Criteria

I am currently pregnant or nursing.

Your hemoglobin level is less than 5.0 grams per deciliter.

You had a bad allergic reaction to IV iron treatments in the past.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

1 visit (in-person)

Treatment

Participants receive intravenous ferric derisomaltose at single doses of 10 mg/kg or 20 mg/kg

8 weeks

6-9 visits (in-person)

Pharmacokinetic Assessment

24 subjects undergo additional blood sampling for PK analysis within the first week after treatment

1 week

2-3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ferric Derisomaltose

Trial Overview The trial tests the safety and effectiveness of Ferric Derisomaltose, an intravenous iron treatment for IDA in kids. Participants will receive a single dose based on weight, up to a maximum of 1000 mg. Some will have additional blood tests to measure how much iron stays in their system over a week.

How Is the Trial Designed?

Group I: Ferric Derisomaltose Experimental Treatment 1 Intervention

All subjects (a total of 200) will be treated with Ferric Derisomaltose. 12 subjects (half of the 24 subjects participating in the PK-part of the trial) will be treated with 10mg/kg while the remaining subjects will be treated with 20 mg/kg. .

Ferric Derisomaltose is already approved in United States, European Union for the following indications:

Approved in United States as Monoferric for:

• Iron deficiency anemia in adults with intolerance to oral iron or unsatisfactory response to oral iron or non-hemodialysis dependent chronic kidney disease (NDD-CKD)

Approved in European Union as Monofer for:

• Iron deficiency anemia in adults with intolerance to oral iron or unsatisfactory response to oral iron or non-hemodialysis dependent chronic kidney disease (NDD-CKD)

Who Is Running the Clinical Trial?

Pharmacosmos A/S

Lead Sponsor

Trials

45

Recruited

9,700+

Published Research Related to This Trial

In a trial involving 1512 patients with iron deficiency anemia, a single dose of 1000 mg iron isomaltoside (IIM) was found to be as safe as iron sucrose (IS), with very low rates of serious hypersensitivity reactions (0.3% for IIM vs 0.4% for IS).

IIM not only met the non-inferiority criteria for hemoglobin change but also resulted in a significantly faster hematological response within the first two weeks compared to IS, which requires multiple doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A prospective, multi-center, randomized comparison of iron isomaltoside 1000 versus iron sucrose in patients with iron deficiency anemia; the FERWON-IDA trial. Auerbach, M., Henry, D., Derman, RJ., et al.[2021]

In a study of 72 children with iron deficiency anemia (IDA) who did not respond to oral iron, intravenous ferric carboxymaltose (FCM) significantly increased hemoglobin levels from a median of 9.1 g/dL to 12.3 g/dL within 4-12 weeks after infusion.

The treatment was found to be safe, with 84% of patients experiencing no adverse effects, and only minor transient complications reported in 7 infusions, indicating a favorable risk profile for FCM in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravenous Ferric Carboxymaltose in Children with Iron Deficiency Anemia Who Respond Poorly to Oral Iron. Powers, JM., Shamoun, M., McCavit, TL., et al.[2018]

In a study involving 771 patients with heart failure and iron deficiency anemia, intravenous ferric derisomaltose (FDI) was associated with a reduction in recurrent hospitalizations for heart failure and cardiovascular death, suggesting it may improve patient outcomes.

Patients receiving FDI reported significant improvements in quality of life, particularly in physical health, indicating that FDI not only addresses iron deficiency but also enhances overall well-being in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The impact of ferric derisomaltose on cardiovascular and non-cardiovascular events in patients with anemia, iron deficiency and heart failure with reduced ejection fraction. Ray, R., Ford, I., Cleland, JGF., et al.[2023]

High-dose intravenous ferric derisomaltose (Monofer®) infusions of 500 and 1000 mg effectively corrected iron deficiency and iron deficiency anemia in elderly patients, leading to rapid improvements in laboratory parameters and symptoms like fatigue.

Intravenous iron administration is a viable treatment option for patients who cannot tolerate oral iron or have poor absorption, potentially reducing morbidity and mortality associated with untreated iron deficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of high-dose intravenous iron in middle-aged to elderly iron-deficient patients. Khiatani, D., Tay, HS., Cushway, TR.[2022]

Ferric derisomaltose (FDI) significantly increased hemoglobin levels in Japanese patients with iron-deficiency anemia (IDA) related to gastrointestinal diseases, with an average increase of 4.33 g/dL across all participants in a phase III study involving 40 patients.

The treatment was found to be safe, with 60% of patients experiencing treatment-emergent adverse events, but no serious or unexpected safety issues were reported, indicating a favorable safety profile for both intravenous bolus injection and drip infusion methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravenous ferric derisomaltose for iron-deficiency anemia associated with gastrointestinal diseases: a single-arm, randomized, uncontrolled, open-label study. Kawabata, H., Tamura, T., Tamai, S., et al.[2022]

Ferric derisomaltose (FDI) is a safe and effective treatment for iron deficiency anemia in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD), showing a lower rate of cardiovascular adverse events compared to iron sucrose (IS).

Patients receiving a single infusion of FDI experienced a faster hematological response, with significant improvements in hemoglobin and ferritin levels, compared to those receiving multiple doses of IS, regardless of their history of heart failure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of Intravenous Ferric Derisomaltose Compared to Iron Sucrose for Iron Deficiency Anemia in Patients with Chronic Kidney Disease With and Without Heart Failure. Ambrosy, AP., von Haehling, S., Kalra, PR., et al.[2021]

Ferric derisomaltose is an effective intravenous iron treatment that allows for complete iron repletion in a single high-dose infusion, making it a rapid and cost-effective option for patients with iron deficiency.

It has a good safety profile, with rare occurrences of serious reactions like anaphylaxis and lower rates of hypophosphatemia compared to older intravenous iron formulations, indicating its suitability for a wide range of patient groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evaluation of ferric derisomaltose as a treatment for anemia. Kassianides, X., Bodington, R., Bhandari, S.[2021]

Ferric derisomaltose (FDI) is effective for treating iron deficiency anemia in patients with non-dialysis-dependent chronic kidney disease (CKD) and those undergoing peritoneal dialysis, showing significant improvements in hemoglobin, transferrin saturation, and ferritin levels after administration.

The treatment was found to be safe, with only three adverse reactions reported among 52 infusions given to 35 patients, and a majority of Canadian renal pharmacists indicated that FDI is funded or available in their hospitals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ferric Derisomaltose Evaluation in Patients with Non-Dialysis-Dependent Chronic Kidney Disease or Peritoneal Dialysis. England, E., Sheffield, M., Poyah, P., et al.[2023]

In a large study of 7354 patients receiving ferric derisomaltose (FDI) for iron deficiency, the incidence of adverse reactions was very low, with only 1.7% experiencing any reactions and less than 0.1% having anaphylaxis, indicating that FDI is a safe treatment option.

Significant improvements in hemoglobin and ferritin levels were observed by Month 4 post-infusion, particularly in patients from surgical specialties, suggesting that FDI is effective in increasing iron levels in diverse patient populations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world experience of intravenous ferric derisomaltose evaluated through safety and efficacy reporting in the UK. Sinclair, RCF., Nadaraja, S., Kennedy, NA., et al.[2023]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40312072/

Initial effectiveness and safety data on intravenous ferric ... Our real-world data reveal FDI is rapidly and persistently effective in correcting IDA in paediatric gastroenterology.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05179226?term=AREA%5BConditionSearch%5D(Iron-refractory%20iron%20deficiency%20anemia)&rank=6

NCT05179226 | Multi-center Trial of Ferric Derisomaltose ... 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 ...

3. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1002/pbc.31847?af=R

A Retrospective Single‐Center Study of Ferric ... Our study describes a real-world retrospective cohort of adolescent patients receiving an off-label indication for an IV iron formulation with a favorable ...

4. ashpublications.org ashpublications.org/hematology/article/2023/1/630/506473/Intravenous-iron-therapy-in-pediatrics-who-should

Intravenous iron therapy in pediatrics: who should get it and ... Many patients with classic iron-deficiency anemia will improve with oral iron therapy. However, in children who have an incomplete response to ...

5. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2024/208171s003lbl.pdf

Reference ID: 5423294 - accessdata.fda.gov The safety and efficacy of Monoferric for treatment of iron deficiency anemia (IDA) were evaluated in two randomized, open-label, actively-controlled ...

6. adc.bmj.com adc.bmj.com/content/110/10/793

Initial effectiveness and safety data on intravenous ferric ... Indirect methods of comparison of the safety of ferric derisomaltose, iron sucrose and ferric carboxymaltose in the treatment of iron deficiency anemia.

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2020/208171s000lbl.pdf

MONOFERRIC (ferric derisomaltose) injection, for intravenous ... The safety of Monoferric was evaluated in 3008 patients with iron deficiency anemia enrolled in two randomized, actively-controlled trials. Trial 1 enrolled ...

8. mayoclinic.org mayoclinic.org/drugs-supplements/ferric-derisomaltose-intravenous-route/description/drg-20481648

Ferric derisomaltose (intravenous route) - Side effects & uses Ferric derisomaltose injection is an iron replacement product that is used to treat iron deficiency anemia (not enough iron in the blood)