Ferric Citrate and Chronic Kidney Disease in Children
Purpose
We will conduct a 12-month, double-blind, randomized, placebo-controlled trial to assess the effects of therapy with ferric citrate (FC) on changes in intact FGF23 levels (iFGF23, primary endpoint) in 160 pediatric patients (80 in each of the two arms) aged 6-18 years of either sex with chronic kidney disease (CKD) stages 3-4 and age-appropriate normal serum phosphate levels. Participants will be randomized to one of the two groups: 1) FC or 2) FC placebo. Participants will be recruited from 20 core clinical sites.
Condition
- Chronic Kidney Diseases
Eligibility
- Eligible Ages
- Between 6 Years and 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Ages 6 to 18 years (inclusive); 2. Estimated Glomerular Filtration Rate (GFR) of 15-59 ml/min per 1.73 m2 by modified Chronic Kidney disease in Children (CKiD) formula;56 3. Serum phosphate within age appropriate normal levels; 4. Serum ferritin <500 ng/ml and TSAT <50%; 5. For those patients treated with growth hormone, calcitriol, nutritional vitamin D, iron, and/or erythropoiesis-stimulating agents (ESAs) such treatments must have stable dosing for at least 2 weeks prior to screening; 6. Able to swallow tablets; 7. Able to eat at least two meals a day; 8. In the opinion of the investigator, willing and able to follow the study treatment regimen and comply with the site investigator's recommendations.
Exclusion Criteria
- Patients currently treated with phosphate binders. 2. History of allergy to all ingredients (including non-medical ingredients) in both products (i.e. investigational product and placebo) 3. Current intestinal malabsorption, documented in the medical record; significant GI disorders including GI bleeding or active inflammatory bowel disease, inflammatory bowel syndrome, and/or Crohn's Disease 4. Anticipated initiation of dialysis or kidney transplantation within 6 months 5. Current or planned future systemic immunosuppressive therapy 6. Prior solid organ transplantation 7. Receipt of bone marrow transplant within two years of screening 8. Current pregnancy, lactation or female subjects who have reached menarche, unless using highly-effective contraception as outlined in section 7.1.1 of Protocol 9. Patients participating in other interventional study (observational study participation permitted) 10. Poor adherence to medical treatments in the opinion of the investigator 11. Hemochromatosis or laboratory tests indicating possible hemochromatosis or other iron overload (primary or secondary) syndrome 12. Cystinosis 13. Fanconi syndrome
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Treatment Arm |
During the 12-month trial, participants will be given a fixed weight-based dose of Ferric Citrate (FC). The full medication dose will be 3g/day for participants weighing <31 kg, 5g/day for those weighing >31 - <51 kg, and 6g/day for participants >51 kg. These doses will be divided into three doses to be taken with meals. |
|
|
Placebo Comparator Control Arm |
During the 12-month trial, participants will be given a fixed weight-based dose of Placebo. The full medication dose will be 3g/day for participants weighing <31 kg, 5g/day for those weighing >31 - <51 kg, and 6g/day for participants >51 kg. These doses will be divided into three doses to be taken with meals. |
|
Recruiting Locations
More Details
- Status
- Recruiting
- Sponsor
- University of California, Los Angeles
Detailed Description
We will conduct a double-blind, randomized, placebo-controlled trial to assess the effects of therapy with ferric citrate (FC) on changes in intact FGF23 levels (iFGF23, primary endpoint) aged 6-18 years of either sex with chronic kidney disease (CKD) stages 3-4 and age-appropriate normal serum phosphate levels. Participants will be randomized to one of the two groups: 1) FC or 2) FC placebo. Participants will be recruited from 20 core clinical sites. Schedule of Intervention: During the 12-month trial, participants will be given a daily fixed weight-based dose of FC. Schedule for data collection/analyses to be performed: Blood for primary outcome assessments will be collected at screening, baseline and at months 3, 6, 9, 12. Blood for safety assessments will be collected at the same intervals. The primary analyses for this 2-arm trial will evaluate changes from baseline in iFGF23 levels over 12 months between the treatment and the placebo arms. The analysis will use a linear mixed-effects model, with random participant effects accounting for repeated measurements, random site effects accounting for clustering of participants into study sites, and a fixed treatment effect, which interacts with a time indicator (Months 3-12 vs. Baseline/Screening). Primary objectives: - To assess the effects of therapy with FC on changes in iFGF23 levels - To determine safety and tolerability of FC. Secondary objectives: • To assess the effects of FC on anemia and indices of mineral and bone metabolism. Primary Endpoint: • Change in iFGF23 level Safety and Tolerability Endpoints: • Ability to safely tolerate FC Secondary Endpoints: - Change in anemia - Change in the indices of mineral and bone metabolism This is a Phase 2 study with participation from 20 sites that will take 36 months to complete enrollment and a total of 48 months to complete data collection with each participant being part of the study for 12 months. Study website: fit4kid.dgsom.ucla.edu