Iron Deficiency Anemia (IDA) and the Brain

NCT ID: NCT05929729

Last Updated: 2024-10-30

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 120 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-12-07
• Study Completion Date: 2028-09-01

Brief Summary

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.

Detailed 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.

Conditions

IDA - Iron Deficiency Anemia; Anemia; Iron Deficiency Anemia; Anemia, Iron Deficiency

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: BASIC_SCIENCE
• Blinding Strategy: NONE

Study Groups

Intravenous (IV) iron

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 Type: EXPERIMENTAL

Ferric derisomaltose

Intervention Type: DRUG

Refer to arm/group descriptions

NovaFerrum

Intervention Type: DRUG

Refer to arm/group descriptions

Standard of care iron

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

Group Type: NO_INTERVENTION

No interventions assigned to this group

Healthy Controls

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

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Ferric derisomaltose

Refer to arm/group descriptions

Intervention Type: DRUG

NovaFerrum

Refer to arm/group descriptions

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: FEMALE
• Accepts Healthy Volunteers: Yes

Sponsors

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

Children's Hospital Los Angeles

OTHER

Sponsor Role: lead

Responsible Party

John C. Wood

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

John Wood, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Los Angeles

Locations

City of Hope Blood Donor Center

Duarte, California, United States

Site Status: NOT_YET_RECRUITING

Cedar Sinai Blood Bank

Los Angeles, California, United States

Site Status: NOT_YET_RECRUITING

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status: RECRUITING

University of California, Los Angeles Blood Donor Center

Los Angeles, California, United States

Site Status: NOT_YET_RECRUITING

Countries

United States

Central Contacts

Silvie Suriany, MSc

Role: CONTACT

anemia@chla.usc.edu

323-361-4783

Facility Contacts

Silvie Suriany, MSc

Role: primary

anemia@chla.usc.edu

323-361-4783

Lefan Zhuang, MD

Role: backup

lzhuang@coh.org

Silvie Suriany, MSc

Role: primary

anemia@chla.usc.edu

323-361-4783

Ellen Klapper, MD

Role: backup

Ellen.Klapper@cshs.org

Silvie Suriany, MSc

Role: primary

anemia@chla.usc.edu

323-361-4783

John Wood, MD,PhD

Role: backup

Silvie Suriany, MSc

Role: primary

anemia@chla.usc.edu

323-361-4783

Dawn Ward, MD

Role: backup

DWard@mednet.ucla.edu

Other Identifiers

1R01NS117430-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

CHLA-23-00173

Identifier Type: -

Identifier Source: org_study_id