Iron Deficiency Anemia (IDA) and the Brain

Children's Hospital Los Angeles

Status and phase: Enrolling; Phase 4

Conditions: Iron Deficiency Anemia; Anemia; IDA - Iron Deficiency Anemia; Anemia, Iron Deficiency

Treatments: Drug: Ferric derisomaltose; Drug: NovaFerrum

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT05929729

CHLA-23-00173

1R01NS117430-01 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Full description

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.

Comprehensive cerebrovascular magnetic resonance imaging (MRI), bloodwork, patient reported outcomes, and neurocognitive testing will be collected from all subjects in the observational arm. 136 subjects total will be enrolled, 40 controls and 96 anemic subjects. The 96 eligible anemic subjects will then be invited to enroll in the interventional arm of the study. Anticipating a drop-out rate of 20%, 40 will be randomized into intravenous iron therapy, and 40 will be randomized into standard of care (oral iron via primary care). Repeated tests will be done at different timepoints to assess the effects and durability of iron therapy.

Enrollment: 120 estimated patients

Sex: Female

Ages: 16 to 60 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Observational arm:

  1. Age between 16 and 60 years of age.
  2. Any ethnicity.
  3. Female
  4. Anemic group: hemoglobin ≤10.5 g/dl or hematocrit <32% from finger prick or plethysmography test, or <11 g/dl from venipuncture blood draw
  5. Control group: hemoglobin >13.2 g/dl or hematocrit >39.6%

• Interventional arm:

  1. Criteria for observational component, plus
  2. Iron deficiency anemia based upon attending hematologist interpretation of transferrin saturation, ferritin, and other ancillary labs including hs-CRP, MMA, hemoglobin electrophoresis

Exclusion criteria

• Observational arm:

  1. Diabetes requiring medication.
  2. Hypertension requiring medication.
  3. Sleep disordered breathing requiring intervention.
  4. Body mass index >40 (morbid obesity)
  5. Contraindications to MRI, including pacemaker, severe claustrophobia, pregnancy.
  6. Known systemic inflammatory disease such as inflammatory bowel disease, systemic lupus erythematosus, or scleroderma.
  7. Known HIV.

• Interventional arm:

  1. Criteria for observational component, plus
  2. Prior reaction to intravenous iron.
  3. History of multiple drug allergies.
  4. History of severe asthma, eczema, or atopy.
  5. Systemic mastocytosis.
  6. Severe respiratory or cardiac disease.
  7. Having no access to a physician who can manage the iron deficiency anemia.

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

120 participants in 3 patient groups

Intravenous (IV) iron

Experimental group

Description:

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.

Treatment:

Drug: NovaFerrum

Drug: Ferric derisomaltose

Standard of care iron

No Intervention group

Description:

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

Healthy Controls

No Intervention group

Description:

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