Iron Replacement Therapy for Iron-Deficiency Anemia

(IDA Trial)

Recruiting at 3 trial locations

Overseen By Silvie Suriany, MSc

Age: < 65

Sex: Female

Trial Phase: Phase 4

Sponsor: Children's Hospital Los Angeles

Disqualifiers: Diabetes, Hypertension, Morbid obesity, others

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

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

Compensation: Varies

Number of Visits: 1

120 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it excludes people who need medication for diabetes or hypertension. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the drug Ferric carboxymaltose for treating iron-deficiency anemia?

Ferric carboxymaltose is effective in treating iron-deficiency anemia, as it replenishes iron stores and corrects anemia in various populations, including those with chronic kidney disease, inflammatory bowel disease, and postpartum anemia. It is generally well tolerated and may require fewer doses compared to other iron treatments, with a lower incidence of gastrointestinal side effects. ^{1 2 3 4 5}

Is iron replacement therapy safe for humans?

Iron replacement therapy, such as ferric carboxymaltose and ferrous sulfate, is generally safe for humans. However, some people may experience mild side effects like nausea or low phosphate levels in the blood, which are usually temporary and not serious. Ferric carboxymaltose is often better tolerated than oral ferrous sulfate, with fewer stomach-related side effects. ^{1 2 6 7 8}

What makes ferric carboxymaltose unique for treating iron-deficiency anemia?

Ferric carboxymaltose is unique because it is administered intravenously, allowing for a high dose of iron to be delivered quickly, often in just 15 minutes, which means fewer doses are needed compared to other treatments. It is generally better tolerated than oral iron supplements like ferrous sulfate, with fewer gastrointestinal side effects. ^{1 2 3 5 6}

What is the purpose of this trial?

This is a trial with an observational and an interventional arm, in patients with moderate to severe anemia and control subjects. The main purposes of this study is to phenotype the scope of neurocognitive deficits from iron deficiency anemia (IDA) in adult women, determine derangements in cerebral perfusion, vascular reactivity, functional connectivity, and blood brain barrier permeability in adult-onset IDA and relate them to neurocognitive deficits, as well as determine the reversibility and durability of both the physiologic and neurocognitive derangements by iron replacement therapy. All eligible subjects will be asked to provide informed consent before participating in the study.

Research Team

John Wood, MD,PhD

Principal Investigator

Children's Hospital Los Angeles

Eligibility Criteria

This trial is for adult women aged 18-60 with moderate to severe iron deficiency anemia (IDA). Participants must have specific lab results indicating IDA and a hemoglobin level ≤10.5 g/dl or hematocrit <32%. Exclusions include MRI contraindications, systemic inflammatory diseases, severe asthma, eczema, atopy, diabetes on medication, hypertension on medication, prior reaction to IV iron, severe respiratory/cardiac disease or morbid obesity.

Inclusion Criteria

In the observational group, the control group should have hemoglobin levels above 13.2 g/dl or hematocrit levels above 39.6%.

My blood tests show I have low hemoglobin or hematocrit levels.

I have iron deficiency anemia as diagnosed by a hematologist.

Exclusion Criteria

I had a reaction to intravenous iron in the past.

I am taking medication for high blood pressure.

I have severe heart or lung disease.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observation

Comprehensive cerebrovascular MRI, bloodwork, patient-reported outcomes, and neurocognitive testing are collected from all subjects in the observational arm.

Baseline (Day 0)

1 visit (in-person)

Intervention

Eligible anemic subjects receive either intravenous iron therapy or standard oral iron therapy. Repeated tests are conducted to assess the effects and durability of iron therapy.

12 months

Multiple visits (in-person and virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments at various time points including Day 14, Day 90, Day 180, and Day 365 post iron therapy.

12 months

Multiple visits (in-person and virtual)

Treatment Details

Interventions

• Ferric carboxymaltose
• Ferrous sulfate

Trial Overview The study aims to understand the impact of IDA on brain function and structure in women and test if these effects can be reversed by iron replacement therapy. It involves two treatments: NovaFerrum and Ferric derisomaltose. The trial has both observational (examining existing conditions) and interventional (testing new treatments) components.

Participant Groups

Group I: Intravenous (IV) iron Experimental Treatment 2 Interventions

This group will receive intravenous iron (Ferric Derisomaltose) in a single dose of 20 mg/kg (max individual dose of 1000 mg). The drug is administered as an infusion over 30 minutes. 3 months after the infusion, they will receive a 9-month supply of Novaferrum pill to be taken once a day.

Group II: Standard of care iron Active Control 1 Intervention

This group will be referred to their primary care provider for oral iron therapy. If a participant cannot obtain care from a physician

Group III: Healthy Controls Active Control 1 Intervention

This group will only be participating in the observational part of the study and serve as our controls.

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

Approved in European Union as Ferinject for:

• Iron deficiency anemia in adult and pediatric patients when oral iron preparations are not tolerated or are ineffective

Approved in Canada as Ferinject for:

• Iron deficiency anemia in adult and pediatric patients one year of age and older when oral iron preparations are not tolerated or are ineffective
• Iron deficiency in adult patients with heart failure and New York Heart Association (NYHA) class II/III to improve exercise capacity

Approved in United States as Ferinject for:

• Iron deficiency anemia in adult patients who have intolerance to oral iron or have had unsatisfactory response to oral iron

Who Is Running the Clinical Trial?

Children's Hospital Los Angeles

Lead Sponsor

Trials

257

Recruited

5,075,000+

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Trials

1,403

Recruited

655,000+

Findings from Research

Ferric carboxymaltose is an effective intravenous treatment for iron-deficiency anemia, rapidly improving hemoglobin levels and replenishing iron stores in various patient populations, including those with chronic kidney disease and inflammatory bowel disease, with a single dose of up to 1000 mg administered in 15 minutes.

The treatment is well tolerated, with mild to moderate side effects similar to those of oral iron supplements, and it shows a faster improvement in hemoglobin levels compared to ferrous sulfate, making it a preferred option for patients who cannot take oral iron.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ferric carboxymaltose: a review of its use in iron-deficiency anaemia. Lyseng-Williamson, KA., Keating, GM.[2022]

Ferric carboxymaltose is an effective intravenous iron treatment for iron deficiency, allowing for a single high dose infusion (up to 1000 mg) that can improve patient outcomes in conditions like chronic heart failure, as shown in two randomized trials (FAIR-HF and CONFIRM-HF).

It is generally well tolerated with a low risk of hypersensitivity reactions and fewer gastrointestinal side effects compared to oral iron supplements, making it a preferable option for replenishing iron stores in various populations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ferric carboxymaltose: a review of its use in iron deficiency. Keating, GM.[2022]

Iron carboxymaltose is as effective as ferrous sulfate for treating postpartum iron deficiency anemia, achieving similar increases in hemoglobin levels in just 2 weeks compared to 12 weeks for ferrous sulfate.

Iron carboxymaltose is better tolerated than ferrous sulfate, with fewer gastrointestinal side effects and no safety concerns for breast-fed infants, making it a safer and more convenient treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative efficacy and safety of intravenous ferric carboxymaltose in the treatment of postpartum iron deficiency anemia. Breymann, C., Gliga, F., Bejenariu, C., et al.[2022]

In a study involving 40 patients with iron deficiency anemia, both controlled-release ferrous glycine sulfate and ferrous fumarate were equally effective in increasing hemoglobin and other blood parameters over 4 weeks.

Ferrous glycine sulfate was associated with fewer gastrointestinal side effects compared to ferrous fumarate, suggesting it may be a better-tolerated option for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A comparative trial of a controlled-release iron tablet preparation ('Ferrocontin' Continus) and ferrous fumarate tablets. Aronstam, A., Aston, DL.[2016]

In a study of 264 geriatric patients (average age 76.9 years), intravenous ferric carboxymaltose significantly improved hemoglobin levels, serum ferritin, and transferrin saturation, indicating its efficacy in treating functional iron deficiency anemia.

The treatment was well tolerated with no severe adverse reactions reported, making ferric carboxymaltose a safe and effective option for elderly patients with anemia related to chronic conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Efficacy and tolerability of ferric carboxymaltose in geriatric patients with anemia. Data from three non-interventional studies]. Röhrig, G., Steinmetz, T., Stein, J., et al.[2022]

Carboxymaltose iron (Ferinject®) is an effective intravenous treatment for iron deficiency anemia, especially when oral iron is ineffective or not tolerated.

While it has an excellent safety profile, hypophosphatemia can occur as a side effect, which may lead to mild to serious symptoms, highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hypophosphatemic osteomalacia, a side effect of iron carboxymaltose administration. Madero Velázquez, L., Muñoz Pérez, R., Rodríguez, A., et al.[2022]

In a study of 291 women with postpartum anemia, intravenous ferric carboxymaltose was found to be more effective than oral ferrous sulfate, leading to quicker and sustained increases in hemoglobin levels.

Ferric carboxymaltose was also associated with fewer drug-related adverse events, indicating it is a safer and better-tolerated option for treating postpartum iron deficiency anemia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ferric carboxymaltose injection in the treatment of postpartum iron deficiency anemia: a randomized controlled clinical trial. Seid, MH., Derman, RJ., Baker, JB., et al.[2022]

In a study involving 543 participants, the wax-matrix tablet of ferrous sulfate was associated with significantly fewer adverse effects compared to conventional ferrous sulfate tablets.

Eighty-one percent of participants using the wax-matrix preparation reported no severe or moderate side effects, while only 50% of those taking the conventional tablets had the same experience, indicating better tolerability of the wax-matrix formulation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse effects of iron supplementation: a comparative trial of a wax-matrix iron preparation and conventional ferrous sulfate tablets. Brock, C., Curry, H., Hanna, C., et al.[2014]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Ferric carboxymaltose: a review of its use in iron-deficiency anaemia. [2022]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Ferric carboxymaltose: a review of its use in iron deficiency. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Comparative efficacy and safety of intravenous ferric carboxymaltose in the treatment of postpartum iron deficiency anemia. [2022]

4. England pubmed.ncbi.nlm.nih.gov

A comparative trial of a controlled-release iron tablet preparation ('Ferrocontin' Continus) and ferrous fumarate tablets. [2016]

5. Germany pubmed.ncbi.nlm.nih.gov

[Efficacy and tolerability of ferric carboxymaltose in geriatric patients with anemia. Data from three non-interventional studies]. [2022]

6. Spain pubmed.ncbi.nlm.nih.gov

Hypophosphatemic osteomalacia, a side effect of iron carboxymaltose administration. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Ferric carboxymaltose injection in the treatment of postpartum iron deficiency anemia: a randomized controlled clinical trial. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Adverse effects of iron supplementation: a comparative trial of a wax-matrix iron preparation and conventional ferrous sulfate tablets. [2014]