IDA - Iron Deficiency AnemiaPhase 4IDANCT05929729
Iron Deficiency Anemia (IDA) and the Brain
A Phase 4 study testing Ferric derisomaltose and NovaFerrum in people with IDA - Iron Deficiency Anemia and Anemia, run by Children's Hospital Los Angeles at 4 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 16 to 60 years, women onlyHealthy volunteers can take part
- Conditions the study is for
- IDA - Iron Deficiency Anemia, Anemia, Iron Deficiency Anemia
- What is being tested?
- Ferric derisomaltose and NovaFerrum
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 4 sitesUnited States
- Study dates
- 7 Dec 2023to 1 Sep 2028
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 60 years
- Women only
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Observational arm:
- Age between 16 and 60 years of age.
- Any ethnicity.
- Female
- Anemic group: hemoglobin ≤10.5 g/dl or hematocrit <32% from finger prick or plethysmography test, or <11 g/dl from venipuncture blood draw
- Control group: hemoglobin >13.2 g/dl or hematocrit >39.6%
Show all 9Show fewer
- Interventional arm:
- Criteria for observational component, plus
- Iron deficiency anemia based upon attending hematologist interpretation of transferrin saturation, ferritin, and other ancillary labs including hs-CRP, MMA, hemoglobin electrophoresis
Who cannot join
- Observational arm:
- Diabetes requiring medication.
- Hypertension requiring medication.
- Sleep disordered breathing requiring intervention.
- Body mass index >40 (morbid obesity)
- Contraindications to MRI, including pacemaker, severe claustrophobia, pregnancy.
Show all 16Show fewer
- Known systemic inflammatory disease such as inflammatory bowel disease, systemic lupus erythematosus, or scleroderma.
- Known HIV.
- Interventional arm:
- Criteria for observational component, plus
- Prior reaction to intravenous iron.
- History of multiple drug allergies.
- History of severe asthma, eczema, or atopy.
- Systemic mastocytosis.
- Severe respiratory or cardiac disease.
- Having no access to a physician who can manage the iron deficiency anemia.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Not yet recruiting
City of Hope Blood Donor Center
Duarte, California, 91010
Silvie Suriany, MSc, 323-361-4783, anemia@chla.usc.edu
Lefan Zhuang, MD, lzhuang@coh.org
- Not yet recruiting
Cedar Sinai Blood Bank
Los Angeles, California, 90027-6062
Silvie Suriany, MSc, 323-361-4783, anemia@chla.usc.edu
Ellen Klapper, MD, Ellen.Klapper@cshs.org
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Silvie Suriany, MSc, 323-361-4783, anemia@chla.usc.edu
John Wood, MD,PhD
- Not yet recruiting
University of California, Los Angeles Blood Donor Center
Los Angeles, California, 90095
Silvie Suriany, MSc, 323-361-4783, anemia@chla.usc.edu
Dawn Ward, MD, DWard@mednet.ucla.edu
About this study
Official title: Neurovascular and Neurocognitive Consequences of Iron Deficiency Anemia
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.
Read the 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.
- Being tested
- Ferric derisomaltose, NovaFerrum
- Medical subject headings
- Anemia, Iron-Deficiency, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ferric derisomaltoseDrug
Refer to arm/group descriptions
NovaFerrumDrug
Refer to arm/group descriptions
| Study group | Role | What they receive |
|---|---|---|
| Intravenous (IV) iron | Experimental | 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. Receives: Drug: Ferric derisomaltose; Drug: NovaFerrum |
| Standard of care iron | No intervention | 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 | This group will only be participating in the observational part of the study and serve as our controls. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Impact of iron deficiency anemia on regional cerebrovascular oxygen delivery (ml O2/100g/min).
Baseline impact of iron deficiency anemia on cerebrovascular oxygen delivery will be assessed by measuring cerebral blood flow and oxygen content through MRI (time-encoded arterial spin labelling) and peripheral blood sample
Measured: Day 0 (observation arm)
Impact of iron therapy on regional cerebrovascular oxygen delivery (ml O2/100g/min) in iron deficiency anemia at day 90 post therapy
Impact of iron therapy on cerebrovascular oxygen delivery will be assessed at day 90 by measuring cerebral blood flow and oxygen content through MRI (time-encoded arterial spin labelling) and peripheral blood sample in people with iron deficiency anemia.
Measured: Day 90 post-iron-therapy
Impact of iron therapy on regional cerebrovascular oxygen delivery (ml O2/100g/min) in iron deficiency anemia at day 365 post therapy.
Impact of iron therapy on cerebrovascular oxygen delivery will be assessed at day 365 by measuring cerebral blood flow and oxygen content through MRI (time-encoded arterial spin labelling) and peripheral blood sample in people with iron deficiency anemia
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on cerebrovascular flow reactivity (%SI change/%ETCO2)
baseline MRI with blood oxygenation level dependent (BOLD) acquisition will be assessed in response to carbon dioxide exposure to determine whether iron deficiency anemia affects cerebrovascular reserve
Measured: Day 0 (observation arm)
Impact of iron therapy on cerebrovascular flow reactivity (%SI change/%ETCO2) in people with iron deficiency anemia at 90 days post iron therapy.
Impact of iron therapy on cardiovascular reserve in iron deficiency anemia will be assessed using MRI with blood oxygenation level dependent (BOLD) acquisition at 90 days post iron therapy.
Measured: Day 90 post iron therapy
Impact of iron therapy on cerebrovascular flow reactivity (%SI change/%ETCO2) in people with iron deficiency anemia at day 365.
Impact of iron therapy on cardiovascular reserve in iron deficiency anemia will be assessed using MRI with blood oxygenation level dependent (BOLD) acquisition at day 365 post iron therapy.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on blood brain barrier permeability surface area product (ml H20/100g/min)
baseline PSA product using water-extraction-with phase- contrast-arterial-spin-tagging (WEPCAST) MRI will be assessed to determine whether iron deficiency anemia affects blood brain barrier permeability to water
Measured: Day 0 (observation arm)
Impact of iron therapy on blood brain barrier permeability surface area product (ml H20/100g/min) in iron deficiency anemia will be assessed at 90 days.
PSA product using water-extraction-with phase- contrast-arterial-spin-tagging (WEPCAST) MRI will be assessed at 90 days post iron therapy to determine the impact of iron therapy on blood brain barrier permeability to water in patients with iron deficiency anemia.
Measured: Day 90 post iron therapy
Impact of iron therapy on blood brain barrier permeability surface area product (ml H20/100g/min) in iron deficiency anemia will be assessed again at day 365.
PSA product using water-extraction-with phase- contrast-arterial-spin-tagging (WEPCAST) MRI will be assessed at 365 days post iron therapy to determine the impact of iron therapy on blood brain barrier permeability to water in patients with iron deficiency anemia.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on cerebral metabolic rate of oxygen (ml O2/100g/min).
Baseline T2 relaxation under spin tagging (TRUST) acquisition via MRI will be used to assess any impact of iron deficiency anemia on cerebral metabolic rate of oxygen
Measured: Day 0 (observation arm)
Impact of iron therapy on cerebral metabolic rate of oxygen (ml O2/100g/min) in people with iron deficiency anemia at day 90 post iron therapy.
T2 relaxation under spin tagging (TRUST) acquisition via MRI will be used to assess any impact of iron therapy on cerebral metabolic rate of oxygen in anemic subjects at day 90.
Measured: Day 90 post iron therapy
Impact of iron therapy on cerebral metabolic rate of oxygen (ml O2/100g/min) in people with iron deficiency anemia at day 365 post iron therapy.
T2 relaxation under spin tagging (TRUST) acquisition via MRI will be used to assess any impact of iron therapy on cerebral metabolic rate of oxygen in anemic subjects at day 365.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on total brain blood flow (ml blood/100g/min).
Phase contrast MRI will be assessed to determine whether iron deficiency anemia affects total brain blood flow at baseline
Measured: Day 0 (observation arm)
Impact of iron therapy on total brain blood flow (ml blood/100g/min) in people with iron deficiency anemia at day 90
Phase contrast MRI will be assessed at day 90 post iron therapy to determine whether iron therapy affects total brain blood flow in subjects with iron deficiency anemia
Measured: Day 90 post iron therapy
Impact of iron therapy on total brain blood flow (ml blood/100g/min) in people with iron deficiency anemia at day 365
Phase contrast MRI will be assessed at day 365 post iron therapy to determine whether iron therapy affects total brain blood flow in subjects with iron deficiency anemia
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on visual-motor integration.
Visual-motor integration at baseline (day 0) will be assessed using Beery Buktenica Developmental Test of Visual-Motor Integration (6th Edition). Standardized scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on visual-motor integration in people with iron deficiency anemia.
Visual-motor integration will be assessed using Beery Buktenica Developmental Test of Visual-Motor Integration (6th Edition) at day 365 post iron-therapy. Standardized scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on sustained attention.
Sustained attention at baseline (day 0) will be assessed using Conners' Continuous Performance Test (3rd Edition) at day 90 post iron-therapy. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean worse performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on sustained attention in people with iron deficiency anemia.
Sustained attention will be assessed using Conners' Continuous Performance Test (3rd Edition) at day 365 post iron-therapy. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean worse performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on working memory function.
Working memory function at baseline (day 0) will be assessed using Digit Span, Coding, and Symbol Search Subtests from Wechsler Adult Intelligence Scale-Fourth Edition (WAIS-IV). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on working memory function in people with iron deficiency anemia.
Working memory function will be assessed at day 365 post iron therapy using Digit Span, Coding, and Symbol Search Subtests from Wechsler Adult Intelligence Scale-Fourth Edition (WAIS-IV). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on the ability to inhibit cognitive interference
The ability to inhibit cognitive interference at baseline (day 0) will be assessed using Color-Word Interference Subtest from the Delis-Kaplan Executive Function System (D-KEFS). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on the ability to inhibit cognitive interference in people with iron deficiency anemia.
The ability to inhibit cognitive interference at day 365 post iron therapy will be assessed using Color-Word Interference Subtest from the Delis-Kaplan Executive Function System (D-KEFS). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on fine motor control.
Fine motor control will be assessed at baseline (day 0) using Reitan Finger Tapping. Z scores with a mean of zero and a standard deviation of one are used. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on fine motor control in people with iron deficiency anemia.
Fine motor control will be assessed at day 365 post iron therapy using Reitan Finger Tapping. Z scores with a mean of zero and a standard deviation of one are used. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron therapy on list learning and recall task in people with iron deficiency anemia.
List learning and recall task will be assessed at day 365 post iron therapy using California Verbal Learning Test-Third Edition (CVLT-3). Z scores with a mean of zero and a standard deviation of 1 are used. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on visuospatial memory
Visuospatial memory will be assessed at baseline (day 0) using Brief Visuospatial Memory Test-Revised (BVMT-R). T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on visuospatial memory in people with iron deficiency anemia.
Visuospatial memory will be assessed at day 365 post iron therapy using Brief Visuospatial Memory Test-Revised (BVMT-R). T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on general intellectual functioning, verbal and nonverbal abilities.
General intellectual functioning, verbal and nonverbal abilities will be assessed at baseline (day 0) using Wechsler Abbreviated Scale of Intelligence-Second Edition (WASI-2). T scores with a mean of 50 and a standard deviation of 10 are used for the subtests, with standard scores (mean of 100 and standard deviation of 15) used for composite scores. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on general intellectual functioning, verbal and nonverbal abilities in people with iron deficiency anemia.
General intellectual functioning, verbal and nonverbal abilities will be assessed at day 365 post iron therapy using Wechsler Abbreviated Scale of Intelligence-Second Edition (WASI-2). T scores with a mean of 50 and a standard deviation of 10 are used for the subtests, with standard scores (mean of 100 and standard deviation of 15) used for composite scores. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on cognitive flexibility and processing speed.
Cognitive flexibility and processing speed will be assessed at baseline (day 0) using NIH Toolbox: Dimensional Change Card Sort and Pattern Comparison Processing Speed. Standard scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron therapy on cognitive flexibility and processing speed in people with iron deficiency anemia.
Cognitive flexibility and processing speed will be assessed at day 365 post iron therapy using NIH Toolbox: Dimensional Change Card Sort and Pattern Comparison Processing Speed. Standard scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on list learning and recall task
List learning and recall task will be assessed at baseline (day 0) using California Verbal Learning Test-Third Edition (CVLT-3). Z scores with a mean of zero and a standard deviation of 1 are used. Higher scores mean better performance.
Measured: Day 0 (observation arm)
Impact of iron deficiency anemia on emotional health
Emotional health will be assessed by using NIH toolbox emotion battery at baseline. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean higher number/frequency of symptoms.
Measured: Day 0 (observation arm)
Impact of iron therapy on emotional health in people with iron deficiency anemia.
Emotional health will be assessed at day 365 post iron therapy by using NIH toolbox emotion battery. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean higher number/frequency of symptoms.
Measured: Day 365 post iron therapy
Impact of iron deficiency anemia on executive functions in day-to-day life.
Executive functions in day-to-day life will be assessed at baseline using the Behavior Rating Inventory of Executive Function 2 (BRIEF-2). T-scores with a mean of 50 and a standard deviation of 10 are used. Scores above T=65 may indicated problems.
Measured: Day 0 (observation arm)
Impact of iron therapy on executive functions in day-to-day life in people with iron deficiency anemia.
Executive functions in day-to-day life will be assessed at day 365 using the Behavior Rating Inventory of Executive Function 2 (BRIEF-2). T-scores with a mean of 50 and a standard deviation of 10 are used. Scores above T=65 may indicated problems.
Measured: Day 365 post iron therapy
Secondary outcomes (11)
Patient reported outcomes of health and quality of life will be assessed at baseline.
Rand Short Form Health Survey will be obtained at baseline. Score range is 0-100. Higher score means better quality of life.
Measured: Day 0 (observation arm)
Changes in patient reported outcomes of health and quality of life will be assessed over a period of 1 year post iron therapy.
Any effects and the changes in the effects of iron therapy will be assessed by obtaining the Rand Short Form Health Survey again post therapy at day 14 (for intravenous (IV) iron group only), day 90, day 180 and day 365 post iron therapy. Score range is 0-100. Higher score means better quality of life.
Measured: Day 14 (for IV iron group only), Day 90, Day 180, Day 365
Patient reported outcomes of fatigue will be assessed at baseline.
Functional Assessment of Chronic Illness Therapy will be obtained at baseline. Score range is 0-52. Higher score means less fatigue.
Measured: Day 0 (observation arm)
Changes in patient reported outcomes of fatigue will be assessed over a period of 1 year post iron therapy.
Any effects and the changes in the effects of iron therapy will be assessed by obtaining the Functional Assessment of Chronic Illness Therapy again post therapy at day 14 (for intravenous (IV) iron group only), day 90, day 180 and day 365 post iron therapy. Score range is 0-52. Higher score means less fatigue.
Measured: Day 14 (for IV iron group only), Day 90, Day 180, Day 365
Impact of iron deficiency anemia on MRI measured brain iron in deep brain nuclei, hippocampus, whole brain grey and white matter.
Quantitative susceptibility mapping will be assessed at baseline.
Measured: Day 0 (observation arm)
Changes in the impact of iron therapy on MRI measured brain iron in deep brain nuclei, hippocampus, whole brain grey and white matter.
Quantitative susceptibility mapping will be assessed at day 14 (for intravenous (IV) iron group only), day 90 and day 365 post iron therapy.
Measured: Day 14 (for IV iron group only), Day 90, Day 365
Changes in hemoglobin and ferritin will be assessed to determine durability of hemoglobin and ferritin response to therapy from baseline to 1 year post iron therapy.
Determine effect sizes of the durability of iron replacement over a period of one year following treatment with oral or intravenous iron.
Measured: Day 0 (observation arm), Day 14 (for IV iron treated), Day 90, Day 180, Day 365
Screening for Restless Legs Syndrome (RLS) at baseline
RLS will be assessed at baseline using the Henning Telephone Diagnostic Interview will be done at baseline. Scores are as follows: 0 = no symptoms; 1 = bedtime symptoms after or within an hour of going to bed; 2 = evening and bedtime symptoms starting at or after 6:00 pm; and 3 = day and night symptoms starting before 6:00 pm. Higher score indicates more severe RLS.
Measured: Day 0 (observation arm)
Changes in Restless Legs Syndrome (RLS) as an effect of iron therapy in people with iron deficiency anemia will be assessed post iron therapy.
Changes in RLS will be assessed by using John Hopkins Telephone Diagnostic Interview at time points over one year post iron therapy. Scores are as follows: 0 = no symptoms; 1 = bedtime symptoms after or within an hour of going to bed; 2 = evening and bedtime symptoms starting at or after 6:00 pm; and 3 = day and night symptoms starting before 6:00 pm. Higher score indicates more severe RLS.
Measured: Day 14 (for intravenous iron group only), Day, 90, Day 180, Day 365
Acute impact of intravenous (IV) iron therapy on visuospatial memory in people with iron deficiency anemia.
Visuospatial memory will be assessed at day 14 post IV iron using Brief Visuospatial Memory Test-Revised (BVMT-R). T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean better performance.
Measured: Day 14 post IV iron therapy
Acute impact of intravenous (IV) iron therapy on list learning and recall task in people with iron deficiency anemia.
List learning and recall task will be assessed at day 14 post IV iron using California Verbal Learning Test-Third Edition (CVLT-3) alternate form. Z scores with a mean of zero and a standard deviation of 1 are used. Higher scores mean better performance.
Measured: Day 14 post IV iron therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2023
- Study start7 Dec 2023
- Primary completion1 Sep 2028 (estimated)
- Study completion1 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for IDA - Iron Deficiency AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Principal investigator, John C. Wood, Children's Hospital Los Angeles
- Investigators
- John Wood, MD, PhD, Principal investigator, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05929729
- Sponsor's protocol ID
- CHLA-23-00173
- Other IDs
- 1R01NS117430-01
- Acronym
- IDA
- Status last verified
- Jul 2024
- First submitted
- 3 May 2023
- Last updated
- 30 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neurovascular and Neurocognitive Consequences of Iron Deficiency Anemia
- ClinicalTrials.gov ID
- NCT05929729
- Acronym / sponsor ID
- IDA / CHLA-23-00173
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Children's Hospital Los AngelesUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- 7 Dec 2023 (actual)
- Primary completion
- 1 Sep 2028 (estimated)
- Study completion
- 1 Sep 2028 (estimated)
- First posted
- 3 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Intravenous (IV) iron | Experimental | Drug: Ferric derisomaltose, Drug: NovaFerrumThis 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. |
| Standard of care iron | No intervention | –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 | –This group will only be participating in the observational part of the study and serve as our controls. |
Who may qualify
- Ages
- 16 to 60 years
- Sex
- Female only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Observational arm:
- Interventional arm:
Key exclusion criteria (16 listed)
- Observational arm:
- Interventional arm:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ferric derisomaltoseRefer to arm/group descriptions | Drug | Intravenous (IV) iron |
| NovaFerrumRefer to arm/group descriptions | Drug | Intravenous (IV) iron |
Primary outcome
Impact of iron deficiency anemia on regional cerebrovascular oxygen delivery (ml O2/100g/min).
Time frame: Day 0 (observation arm)
Baseline impact of iron deficiency anemia on cerebrovascular oxygen delivery will be assessed by measuring cerebral blood flow and oxygen content through MRI (time-encoded arterial spin labelling) and peripheral blood sample
Impact of iron therapy on regional cerebrovascular oxygen delivery (ml O2/100g/min) in iron deficiency anemia at day 90 post therapy
Time frame: Day 90 post-iron-therapy
Impact of iron therapy on cerebrovascular oxygen delivery will be assessed at day 90 by measuring cerebral blood flow and oxygen content through MRI (time-encoded arterial spin labelling) and peripheral blood sample in people with iron deficiency anemia.
Impact of iron therapy on regional cerebrovascular oxygen delivery (ml O2/100g/min) in iron deficiency anemia at day 365 post therapy.
Time frame: Day 365 post iron therapy
Impact of iron therapy on cerebrovascular oxygen delivery will be assessed at day 365 by measuring cerebral blood flow and oxygen content through MRI (time-encoded arterial spin labelling) and peripheral blood sample in people with iron deficiency anemia
Impact of iron deficiency anemia on cerebrovascular flow reactivity (%SI change/%ETCO2)
Time frame: Day 0 (observation arm)
baseline MRI with blood oxygenation level dependent (BOLD) acquisition will be assessed in response to carbon dioxide exposure to determine whether iron deficiency anemia affects cerebrovascular reserve
Impact of iron therapy on cerebrovascular flow reactivity (%SI change/%ETCO2) in people with iron deficiency anemia at 90 days post iron therapy.
Time frame: Day 90 post iron therapy
Impact of iron therapy on cardiovascular reserve in iron deficiency anemia will be assessed using MRI with blood oxygenation level dependent (BOLD) acquisition at 90 days post iron therapy.
Impact of iron therapy on cerebrovascular flow reactivity (%SI change/%ETCO2) in people with iron deficiency anemia at day 365.
Time frame: Day 365 post iron therapy
Impact of iron therapy on cardiovascular reserve in iron deficiency anemia will be assessed using MRI with blood oxygenation level dependent (BOLD) acquisition at day 365 post iron therapy.
Impact of iron deficiency anemia on blood brain barrier permeability surface area product (ml H20/100g/min)
Time frame: Day 0 (observation arm)
baseline PSA product using water-extraction-with phase- contrast-arterial-spin-tagging (WEPCAST) MRI will be assessed to determine whether iron deficiency anemia affects blood brain barrier permeability to water
Impact of iron therapy on blood brain barrier permeability surface area product (ml H20/100g/min) in iron deficiency anemia will be assessed at 90 days.
Time frame: Day 90 post iron therapy
PSA product using water-extraction-with phase- contrast-arterial-spin-tagging (WEPCAST) MRI will be assessed at 90 days post iron therapy to determine the impact of iron therapy on blood brain barrier permeability to water in patients with iron deficiency anemia.
Impact of iron therapy on blood brain barrier permeability surface area product (ml H20/100g/min) in iron deficiency anemia will be assessed again at day 365.
Time frame: Day 365 post iron therapy
PSA product using water-extraction-with phase- contrast-arterial-spin-tagging (WEPCAST) MRI will be assessed at 365 days post iron therapy to determine the impact of iron therapy on blood brain barrier permeability to water in patients with iron deficiency anemia.
Impact of iron deficiency anemia on cerebral metabolic rate of oxygen (ml O2/100g/min).
Time frame: Day 0 (observation arm)
Baseline T2 relaxation under spin tagging (TRUST) acquisition via MRI will be used to assess any impact of iron deficiency anemia on cerebral metabolic rate of oxygen
Impact of iron therapy on cerebral metabolic rate of oxygen (ml O2/100g/min) in people with iron deficiency anemia at day 90 post iron therapy.
Time frame: Day 90 post iron therapy
T2 relaxation under spin tagging (TRUST) acquisition via MRI will be used to assess any impact of iron therapy on cerebral metabolic rate of oxygen in anemic subjects at day 90.
Impact of iron therapy on cerebral metabolic rate of oxygen (ml O2/100g/min) in people with iron deficiency anemia at day 365 post iron therapy.
Time frame: Day 365 post iron therapy
T2 relaxation under spin tagging (TRUST) acquisition via MRI will be used to assess any impact of iron therapy on cerebral metabolic rate of oxygen in anemic subjects at day 365.
Impact of iron deficiency anemia on total brain blood flow (ml blood/100g/min).
Time frame: Day 0 (observation arm)
Phase contrast MRI will be assessed to determine whether iron deficiency anemia affects total brain blood flow at baseline
Impact of iron therapy on total brain blood flow (ml blood/100g/min) in people with iron deficiency anemia at day 90
Time frame: Day 90 post iron therapy
Phase contrast MRI will be assessed at day 90 post iron therapy to determine whether iron therapy affects total brain blood flow in subjects with iron deficiency anemia
Impact of iron therapy on total brain blood flow (ml blood/100g/min) in people with iron deficiency anemia at day 365
Time frame: Day 365 post iron therapy
Phase contrast MRI will be assessed at day 365 post iron therapy to determine whether iron therapy affects total brain blood flow in subjects with iron deficiency anemia
Impact of iron deficiency anemia on visual-motor integration.
Time frame: Day 0 (observation arm)
Visual-motor integration at baseline (day 0) will be assessed using Beery Buktenica Developmental Test of Visual-Motor Integration (6th Edition). Standardized scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Impact of iron therapy on visual-motor integration in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Visual-motor integration will be assessed using Beery Buktenica Developmental Test of Visual-Motor Integration (6th Edition) at day 365 post iron-therapy. Standardized scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Impact of iron deficiency anemia on sustained attention.
Time frame: Day 0 (observation arm)
Sustained attention at baseline (day 0) will be assessed using Conners' Continuous Performance Test (3rd Edition) at day 90 post iron-therapy. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean worse performance.
Impact of iron therapy on sustained attention in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Sustained attention will be assessed using Conners' Continuous Performance Test (3rd Edition) at day 365 post iron-therapy. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean worse performance.
Impact of iron deficiency anemia on working memory function.
Time frame: Day 0 (observation arm)
Working memory function at baseline (day 0) will be assessed using Digit Span, Coding, and Symbol Search Subtests from Wechsler Adult Intelligence Scale-Fourth Edition (WAIS-IV). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Impact of iron therapy on working memory function in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Working memory function will be assessed at day 365 post iron therapy using Digit Span, Coding, and Symbol Search Subtests from Wechsler Adult Intelligence Scale-Fourth Edition (WAIS-IV). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Impact of iron deficiency anemia on the ability to inhibit cognitive interference
Time frame: Day 0 (observation arm)
The ability to inhibit cognitive interference at baseline (day 0) will be assessed using Color-Word Interference Subtest from the Delis-Kaplan Executive Function System (D-KEFS). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Impact of iron therapy on the ability to inhibit cognitive interference in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
The ability to inhibit cognitive interference at day 365 post iron therapy will be assessed using Color-Word Interference Subtest from the Delis-Kaplan Executive Function System (D-KEFS). Scaled scores with a mean of 10 and a standard deviation of 3 are used. Higher scores mean better performance.
Impact of iron deficiency anemia on fine motor control.
Time frame: Day 0 (observation arm)
Fine motor control will be assessed at baseline (day 0) using Reitan Finger Tapping. Z scores with a mean of zero and a standard deviation of one are used. Higher scores mean better performance.
Impact of iron therapy on fine motor control in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Fine motor control will be assessed at day 365 post iron therapy using Reitan Finger Tapping. Z scores with a mean of zero and a standard deviation of one are used. Higher scores mean better performance.
Impact of iron therapy on list learning and recall task in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
List learning and recall task will be assessed at day 365 post iron therapy using California Verbal Learning Test-Third Edition (CVLT-3). Z scores with a mean of zero and a standard deviation of 1 are used. Higher scores mean better performance.
Impact of iron deficiency anemia on visuospatial memory
Time frame: Day 0 (observation arm)
Visuospatial memory will be assessed at baseline (day 0) using Brief Visuospatial Memory Test-Revised (BVMT-R). T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean better performance.
Impact of iron therapy on visuospatial memory in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Visuospatial memory will be assessed at day 365 post iron therapy using Brief Visuospatial Memory Test-Revised (BVMT-R). T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean better performance.
Impact of iron deficiency anemia on general intellectual functioning, verbal and nonverbal abilities.
Time frame: Day 0 (observation arm)
General intellectual functioning, verbal and nonverbal abilities will be assessed at baseline (day 0) using Wechsler Abbreviated Scale of Intelligence-Second Edition (WASI-2). T scores with a mean of 50 and a standard deviation of 10 are used for the subtests, with standard scores (mean of 100 and standard deviation of 15) used for composite scores. Higher scores mean better performance.
Impact of iron therapy on general intellectual functioning, verbal and nonverbal abilities in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
General intellectual functioning, verbal and nonverbal abilities will be assessed at day 365 post iron therapy using Wechsler Abbreviated Scale of Intelligence-Second Edition (WASI-2). T scores with a mean of 50 and a standard deviation of 10 are used for the subtests, with standard scores (mean of 100 and standard deviation of 15) used for composite scores. Higher scores mean better performance.
Impact of iron deficiency anemia on cognitive flexibility and processing speed.
Time frame: Day 0 (observation arm)
Cognitive flexibility and processing speed will be assessed at baseline (day 0) using NIH Toolbox: Dimensional Change Card Sort and Pattern Comparison Processing Speed. Standard scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Impact of iron therapy on cognitive flexibility and processing speed in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Cognitive flexibility and processing speed will be assessed at day 365 post iron therapy using NIH Toolbox: Dimensional Change Card Sort and Pattern Comparison Processing Speed. Standard scores with a mean of 100 and a standard deviation of 15 are used. Higher scores mean better performance.
Impact of iron deficiency anemia on list learning and recall task
Time frame: Day 0 (observation arm)
List learning and recall task will be assessed at baseline (day 0) using California Verbal Learning Test-Third Edition (CVLT-3). Z scores with a mean of zero and a standard deviation of 1 are used. Higher scores mean better performance.
Impact of iron deficiency anemia on emotional health
Time frame: Day 0 (observation arm)
Emotional health will be assessed by using NIH toolbox emotion battery at baseline. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean higher number/frequency of symptoms.
Impact of iron therapy on emotional health in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Emotional health will be assessed at day 365 post iron therapy by using NIH toolbox emotion battery. T-scores with a mean of 50 and a standard deviation of 10 are used. Higher scores mean higher number/frequency of symptoms.
Impact of iron deficiency anemia on executive functions in day-to-day life.
Time frame: Day 0 (observation arm)
Executive functions in day-to-day life will be assessed at baseline using the Behavior Rating Inventory of Executive Function 2 (BRIEF-2). T-scores with a mean of 50 and a standard deviation of 10 are used. Scores above T=65 may indicated problems.
Impact of iron therapy on executive functions in day-to-day life in people with iron deficiency anemia.
Time frame: Day 365 post iron therapy
Executive functions in day-to-day life will be assessed at day 365 using the Behavior Rating Inventory of Executive Function 2 (BRIEF-2). T-scores with a mean of 50 and a standard deviation of 10 are used. Scores above T=65 may indicated problems.
11 secondary outcomes
Patient reported outcomes of health and quality of life will be assessed at baseline.
Time frame: Day 0 (observation arm)
Changes in patient reported outcomes of health and quality of life will be assessed over a period of 1 year post iron therapy.
Time frame: Day 14 (for IV iron group only), Day 90, Day 180, Day 365
Patient reported outcomes of fatigue will be assessed at baseline.
Time frame: Day 0 (observation arm)
Changes in patient reported outcomes of fatigue will be assessed over a period of 1 year post iron therapy.
Time frame: Day 14 (for IV iron group only), Day 90, Day 180, Day 365
Impact of iron deficiency anemia on MRI measured brain iron in deep brain nuclei, hippocampus, whole brain grey and white matter.
Time frame: Day 0 (observation arm)
Changes in the impact of iron therapy on MRI measured brain iron in deep brain nuclei, hippocampus, whole brain grey and white matter.
Time frame: Day 14 (for IV iron group only), Day 90, Day 365
Changes in hemoglobin and ferritin will be assessed to determine durability of hemoglobin and ferritin response to therapy from baseline to 1 year post iron therapy.
Time frame: Day 0 (observation arm), Day 14 (for IV iron treated), Day 90, Day 180, Day 365
Screening for Restless Legs Syndrome (RLS) at baseline
Time frame: Day 0 (observation arm)
Changes in Restless Legs Syndrome (RLS) as an effect of iron therapy in people with iron deficiency anemia will be assessed post iron therapy.
Time frame: Day 14 (for intravenous iron group only), Day, 90, Day 180, Day 365
Acute impact of intravenous (IV) iron therapy on visuospatial memory in people with iron deficiency anemia.
Time frame: Day 14 post IV iron therapy
Acute impact of intravenous (IV) iron therapy on list learning and recall task in people with iron deficiency anemia.
Time frame: Day 14 post IV iron therapy
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2024.
- Individual participant dataSharing planned: No
Related trials for IDA - Iron Deficiency Anemia
RecruitingObservational study
Patient Blood Management in Patients Scheduled for Cardiac Surgery
Anemia, Anemia, Iron-Deficiency
RecruitingPhase 4
Efficacy and Tolerability of Sucrosomial® Iron vs Ferric Maltol in Iron Deficient Women
Anemia, Iron Deficiency Anemia (IDA)
Not yet recruiting
Iron-Rich Snack Intervention for Anemia in Adolescent Girls
Iron Deficiency Anemia (IDA)
Not yet recruitingPhase 3
A Study to Test the Non-Inferiority of Ferric Bepectate IV Against Ferric Carboxymaltose in Patients With Iron Deficiency Anemia
Iron Deficiency Anemia (IDA)
Not yet recruitingPhase 4
Ferric Carboxymaltose Versus Iron Sucrose for Iron Deficiency Anemia After Radical Gastrectomy for Gastric Cancer
Gastric Cancer (GC), Iron Deficiency Anemia (IDA)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
John Wood, MD, PhD principal investigator
Children's Hospital Los Angeles
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Ferric derisomaltose and NovaFerrum in people with IDA - Iron Deficiency Anemia and Anemia, run by Children's Hospital Los Angeles at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Silvie Suriany, MSc (323-361-4783, anemia@chla.usc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Impact of iron deficiency anemia on regional cerebrovascular oxygen delivery (ml O2/100g/min). (time frame: Day 0 (observation arm)). The study lists 37 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2024; AzTrials last refreshed its copy on 26 Oct 2026.