Ferumoxytol for Iron-Deficiency Anemia in Children

Recruiting at 16 trial locations

Overseen By Clinical Trial Interest

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: AMAG Pharmaceuticals, Inc.

Disqualifiers: CKD, Low BP, Severe anemia, 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 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 5 weeks

75 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests ferumoxytol as a treatment for children with iron-deficiency anemia (IDA), a condition where the body lacks enough iron to produce healthy red blood cells. The trial compares the effectiveness of ferumoxytol to another iron treatment, iron sucrose. Children who have had trouble with oral iron treatments or cannot take them are suitable candidates for this trial. It is open to children aged 2 to under 18 with low hemoglobin levels and other signs of IDA. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants early access to a potentially effective treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ferumoxytol has been studied for treating iron deficiency anemia (IDA) in various groups, including individuals with chronic kidney disease (CKD). In studies involving about 2,000 patients with IDA, ferumoxytol demonstrated a specific safety profile. Some patients experienced side effects, but serious reactions were rare, occurring in about 1% of patients. Common side effects included dizziness and nausea.

While the FDA has approved ferumoxytol for use in adults with certain conditions, limited information exists about its use in children. Consequently, less is known about how children might react to this treatment. Researchers are conducting ongoing trials to better understand its safety and effectiveness in children. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Ferumoxytol is unique because it delivers iron in a concentrated, stable form that's coated with polyglucose sorbitol carboxymethylether, which may enhance its absorption and reduce side effects compared to other iron treatments. Unlike standard treatments like iron sucrose or oral iron supplements, ferumoxytol's formulation allows it to be administered as a quick intravenous infusion, potentially increasing convenience and compliance. Researchers are excited because this could provide a faster, more efficient way to replenish iron levels in children with iron-deficiency anemia, especially for those who struggle with oral iron due to gastrointestinal side effects.

What evidence suggests that this trial's treatments could be effective for iron-deficiency anemia in children?

Research has shown that ferumoxytol, one of the treatments in this trial, effectively treats iron-deficiency anemia (IDA) in children. One study found that children treated with ferumoxytol experienced a 14.5% increase in transferrin saturation (TSAT), which measures iron in the blood, from the start of the study to week 5. Another study demonstrated that ferumoxytol significantly increased hemoglobin levels, with many children experiencing an improvement of 20 grams per liter or more. This treatment quickly replenishes iron levels and resolves anemia without the stomach issues often caused by oral iron supplements, making ferumoxytol a promising option for children with iron-deficiency anemia. Iron sucrose, another treatment option in this trial, serves as an active comparator. ^{3 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for children and teenagers aged 2 to less than 18 with Iron Deficiency Anemia (IDA). They must have low hemoglobin levels, iron saturation, or ferritin, or not tolerate oral iron well. Kids with severe allergies to IV iron, certain hypersensitivities, very low blood pressure, extremely low hemoglobin or chronic kidney disease are excluded.

Inclusion Criteria

I have iron deficiency anemia with specific blood test results and issues with oral iron.

I am between 2 and 17 years old.

Exclusion Criteria

I have chronic kidney disease or require regular dialysis.

History of ≥2 clinically significant drug allergies

Your hemoglobin level is less than 7.0 grams per deciliter.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either ferumoxytol or iron sucrose for the treatment of iron deficiency anemia

5 weeks

Multiple visits for drug administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ferumoxytol
• Iron sucrose

Trial Overview The study tests Ferumoxytol against Iron sucrose in treating IDA in young patients. It's a Phase 3 trial where participants are chosen randomly and the process is open for researchers and participants to know which treatment they receive.

How Is the Trial Designed?

Group I: Ferumoxytol Experimental Treatment 1 Intervention

Each 20 mL single-use vial contains 17 mL of ferumoxytol that consists of iron at a concentration of 30 mg Fe/mL, coated with polyglucose sorbitol carboxymethylether and formulated with mannitol, at a concentration of 44 mg/mL, in a black to reddish brown sterile, aqueous, colloidal, isotonic solution.

Group II: Iron sucrose Active Control 1 Intervention

Each mL contains 20 mg of elemental iron as iron sucrose in water for injection. The 5 mL single-use vial contains 100 mg of iron per 5 mL. The drug product contains approximately 30% sucrose (300 mg/mL)

ferumoxytol is already approved in United States for the following indications:

Approved in United States as Feraheme for:

• Anemia Due to Chronic Kidney Disease
• Iron Deficiency Anemia

Who Is Running the Clinical Trial?

AMAG Pharmaceuticals, Inc.

Lead Sponsor

Trials

44

Recruited

11,300+

Published Research Related to This Trial

Ferumoxytol, an intravenous treatment for iron deficiency anemia, can cause significant imaging artifacts in brain MRIs performed within 3 days of administration, affecting the diagnostic quality of the scans.

Recognizing these artifacts, such as susceptibility effects on various MRI sequences and altered vascular signals, is crucial for clinicians to avoid misinterpretations and unnecessary follow-up examinations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Incidental ferumoxytol artifacts in clinical brain MR imaging. Bowser, BA., Campeau, NG., Carr, CM., et al.[2018]

Ferumoxytol is a safe and effective iron formulation for treating iron deficiency, approved for use in various conditions beyond just chronic kidney disease, allowing for large doses to be administered quickly.

The risk of severe hypersensitivity reactions, including anaphylaxis, is extremely rare with ferumoxytol, making it a favorable option compared to other iron supplements like iron sucrose and iron dextran.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ferumoxytol for the treatment of iron deficiency anemia. Auerbach, M., Chertow, GM., Rosner, M.[2019]

Ferumoxytol is as effective and safe as other intravenous iron formulations, with moderate quality evidence showing little to no difference in treatment-emergent adverse events and serious adverse events across studies involving 5691 participants.

While ferumoxytol had fewer treatment-emergent adverse events compared to oral iron, it was associated with more adverse events compared to placebo, indicating a need for careful monitoring when used.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The safety and efficacy of ferumoxytol in the treatment of iron deficiency: a systematic review and meta-analysis. Abdulrehman, J., Tang, GH., Auerbach, M., et al.[2021]

Ferumoxytol is a newly approved and effective treatment for iron deficiency anemia, characterized by its superparamagnetic iron oxide core that affects MRI imaging.

The use of ferumoxytol can interfere with MRI interpretations, potentially masking important diagnostic information or mimicking disease states, which is crucial for both radiologists and prescribing physicians to consider.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parenteral ferumoxytol interaction with magnetic resonance imaging: a case report, review of the literature and advisory warning. Schieda, N.[2023]

In a preliminary study involving 6 pediatric patients with gastrointestinal diseases, ferumoxytol was administered as intravenous iron therapy and was well tolerated in 12 out of 13 infusions, with only one mild adverse effect reported.

The study suggests that ferumoxytol can be safely given at doses of 5.8-12 mg elemental iron/kg over 15-60 minutes, but further research is needed to evaluate long-term safety and the risk of iron overload.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ferumoxytol infusion in pediatric patients with gastrointestinal disorders: first case series. Hassan, N., Cahill, J., Rajasekaran, S., et al.[2013]

In a study of 54 children receiving ferumoxytol for iron deficiency anemia, significant increases in hemoglobin levels were observed at 1 week (11.5 g/dL) and 4 weeks (11.8 g/dL) post-infusion compared to baseline (9.2 g/dL), indicating the efficacy of the treatment.

The treatment was generally safe, with only a few mild adverse drug reactions reported, such as pruritus and urticaria, which were manageable with standard therapies, highlighting the importance of slow infusion rates and close monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravenous Ferumoxytol in Pediatric Patients With Iron Deficiency Anemia. Hassan, N., Boville, B., Reischmann, D., et al.[2018]

In a multicenter study involving 3215 patients who received 4240 ferumoxytol injections for MRI, the use of ferumoxytol was well tolerated with no severe or life-threatening adverse events reported.

Only 1.9% of patients experienced adverse reactions related to ferumoxytol, mostly mild, indicating a positive safety profile for its off-label diagnostic use as an alternative to gadolinium-based contrast agents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Multicenter Safety and Practice for Off-Label Diagnostic Use of Ferumoxytol in MRI. Nguyen, KL., Yoshida, T., Kathuria-Prakash, N., et al.[2023]

Ferumoxytol, an intravenous iron treatment approved by the FDA for iron deficiency anemia in patients with chronic kidney disease, significantly increased hemoglobin levels by about 1.0 g/dL over 35 days, outperforming oral iron treatments.

While ferumoxytol is effective, it carries safety concerns, including a 0.2% risk of serious hypersensitivity reactions and a 1.9% incidence of hypotension, highlighting the need for monitoring during administration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

FDA report: Ferumoxytol for intravenous iron therapy in adult patients with chronic kidney disease. Lu, M., Cohen, MH., Rieves, D., et al.[2013]

Citations

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

Intravenous iron therapy in pediatrics: who should get it and ... Benefits of intravenous iron therapy include a rapid repletion of iron stores in addition to resolution of anemia, less gastrointestinal side ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/28622742/

Intravenous Ferumoxytol in Pediatric Patients With Iron ... Ferumoxytol was effective in treating IDA in our small study. Slow infusion rate and close monitoring allowed early detection of the infrequent ADRs.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT01155388

A Trial of Ferumoxytol for the Treatment of Iron Deficiency ... Study evaluating the efficacy and safety of intravenous (IV) ferumoxytol compared with oral iron for the treatment of pediatric participants with chronic kidney ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4225478/

A Phase III, randomized, open-label trial of ferumoxytol ... Ferumoxytol-treated patients showed a statistically significant (P = 0.0048) greater increase in TSAT from Baseline to Week 5 (14.5%) compared with the increase ...

5. karger.com karger.com/aha/article/142/3/125/15878/The-Efficacy-of-Ferumoxytol-for-Iron-Deficiency

The Efficacy of Ferumoxytol for Iron Deficiency Anemia Our meta-analysis suggests that intravenous ferumoxytol is able to substantially improve the proportion of patients with a ≥20 g/L Hb increase ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2009/022180lbl.pdf

Feraheme (ferumoxytol) Injection Label - accessdata.fda.gov The safety and efficacy of Feraheme for the episodic treatment of iron deficiency anemia in patients with CKD were assessed in three randomized, open-label ...

7. mayoclinic.org mayoclinic.org/drugs-supplements/ferumoxytol-intravenous-route/description/drg-20073073

Ferumoxytol (intravenous route) - Side effects & uses Appropriate studies have not been performed on the relationship of age to the effects of ferumoxytol injection in the pediatric population.

8. feraheme.com feraheme.com/safety-side-effects/

Safety and side effects | FERAHEME® (ferumoxytol injection) The safety profile of FERAHEME was evaluated in ~2000 iron deficiency anemia (IDA) patients1. Adverse reactions to FERAHEME reported in ≥1% of IDA patients in ...

9. clinicaltrials.gov clinicaltrials.gov/study/NCT03619850

A Study to Evaluate the Safety (Compared to Iron Sucrose), ... Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia ...