Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Pediatric Patients With CKD

Mao Jianhua

Status and phase: Unknown; Phase 3

Conditions: Anemia Associated With Chronic Kidney Disease

Treatments: Drug: Roxadustat

Study type: Interventional

Funder types: Other

Identifiers

NCT04925011

Roxadustat for pediatric CKD

Details and patient eligibility

About

This open-labeled, one-center study is designed to evaluate the efficacy and safety of roxadustat in ESA-naïve and ESA-treated pediatric patients with CKD Stages 3, 4, and 5 who are receiving dialysis or not. The study will enroll patients between the ages of 2 to <18 years. Approximately 30 patients will be enrolled.

Full description

This study is to evaluate the use of roxadustat for renal anemia in pediatric patients with CKD

Enrollment: 30 estimated patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Estimated glomerular filtration rate (Bedside Schwartz formula) of < 60 ml/min/1.73 m2 (stage 3, 4 and 5 CKD) for dialysis or non-dialysis patients.
2. For ESA-naïve patients, mean of two most recent central laboratory Hb values during the screening period obtained at least 2 days apart must be < 11.0 g/dL. For patients currently receiving stable ESA dosing who will discontinue ESA and convert to roxadustat during study, mean of two most recent central laboratory Hb values during the screening period obtained at least 2 days apart must be ≥ 9.0 g/dL and ≤ 12.5 g/dL
3. Ferritin >50 ng/mL and transferrin saturation >10%

Exclusion criteria

1. Uncontrolled hypertension as judged by the principal investigator prior to screening.
2. Known hematologic disease related anemia (including PRCA)
3. Known malignancy within the past 5 years before screening.
4. History of severe anaphylaxis or known allergic to ingredient of roxadusta.
5. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3x upper limit of normal (ULN) and total bilirubin (Tbili) ≤2x ULN(obtained from screening visit).
6. Serum folate and vitamin B12 > LLN and cannot be corrected (obtained from screening visit).
7. Any RBC transfusion during the past 4 weeks before screening.
8. Weight<10kg
9. Any prior exposure to roxadustat or any other HIF-PH inhibitor.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

30 participants in 1 patient group

Roxadustat

Other group

Description:

Starting doses of 20, 50, 70 or 100 mg/30,70,90,or 120mg based on weight and dialysis or not.

Treatment:

Drug: Roxadustat