Study to Evaluate the Safety and Tolerability of KRX-0502 (Ferric Citrate) in Children With Iron Deficiency Anemia Associated With Non-Dialysis Dependent Chronic Kidney Disease

Study Overview
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Brief Summary
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This study will be conducted to evaluate the safety and tolerability of ferric citrate in pediatric participants with iron deficiency anemia (IDA) associated with non-dialysis dependent chronic kidney disease (NDD-CKD).

Official Title
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A 24-Week, Open-Label, Randomized, 2-Arm Study to Evaluate the Safety and Tolerability of KRX-0502 (Ferric Citrate) in Children With Iron Deficiency Anemia Associated With Non-Dialysis Dependent Chronic Kidney Disease

Conditions
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Iron Deficiency Anemia Associated With Non-Dialysis Dependent Chronic Kidney Disease

Intervention / Treatment
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* Drug: ferric citrate
* Drug: standard of care


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age 6 years to <18 years at Screening Body Weight ≥12 kilograms (kg) at Screening Chronic kidney disease (CKD) stage 3 to 5, not on dialysis, with estimated glomerular filtration rate (eGFR) <60 milliliters per minute (mL/min)/1.73 meters squared (m^2) utilizing the Bedside Schwartz equation Hemoglobin (Hgb) ≥8.5 and ≤11.5 grams per deciliter (g/dL) at Screening Transferrin saturation (TSAT) ≤25% at Screening Ferritin ≤200 nanograms per milliliter (ng/mL) at Screening Exclusion Criteria: Serum phosphorus level at Screening: 6 to <13 years: ≤4.0 milligrams per deciliter (mg/dL); 13 to <18 years: ≤2.7 mg/dL Liver transaminases (aspartate aminotransferase [AST] and/or alanine aminotransferase [ALT]) ˃3× the upper limit of normal at Screening Active significant gastrointestinal (GI) disorder, including overt gastrointestinal (GI) bleeding or active inflammatory bowel disease Unable to swallow pills Anemia due to causes other than iron deficiency anemia (IDA) of CKD Intravenous iron therapy or blood transfusion within 4 weeks before the Screening visit Participants with a functioning organ transplant Receipt of any investigational drug within 4 weeks before Screening Phosphate binder use during the Screening period

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ferric citrate<br>Participants aged 12 to <18 years and 6 to <12 years will receive ferric citrate for 24 weeks at a starting dose based on body weight categories. | Drug: ferric citrate<br>* oral tablets<br>* Other names: KRX-0502;|
| Active Comparator: Standard of care<br>Participants aged 12 to <18 years and 6 to <12 years will receive standard of care treatment for 24 weeks. | Drug: standard of care<br>* administered per the approved label and at the Investigator's discretion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Serious and Non-serious Treatment-emergent Adverse Events |  | up to Week 28 | 
| Number of Participants with Clinically Significant Laboratory Abnormalities or Changes in Laboratory Results |  | up to Week 24 | 
| Number of Participants with Treatment-emergent Adverse Events Leading to the Discontinuation of Ferric Citrate |  | up to Week 28 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline in Hemoglobin to Week 24/Early Termination Visit |  | Baseline; Week 24 | 
| Change from Baseline in Transferrin Saturation (TSAT) to Week 24/Early Termination Visit |  | Baseline; Week 24 | 
| Change from Baseline in Ferritin to Week 24/Early Termination Visit |  | Baseline; Week 24 | 
| Change from Baseline in Serum Phosphorus to Week 24/Early Termination Visit |  | Baseline; Week 24 | 
| Change from Baseline in Calcium to Week 24/Early Termination Visit |  | Baseline; Week 24 | 
| Change from Baseline in Bicarbonate to Week 24/Early Termination Visit |  | Baseline; Week 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Iron Deficiency Anemia, Non-Dialysis Dependent Chronic Kidney Disease, CKD, ferric citrate, pediatric, dialysis