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

Phase 3

• Conditions: Anemia Associated With Chronic Kidney Disease
• Interventions: Drug: Roxadustat

• Registration Number: NCT04925011
• Lead Sponsor: Mao Jianhua

Brief Summary

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.

Detailed Description

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

Study Timeline

• Study First Submitted: 2021-06-08
• Study First Submitted QC: 2021-06-08
• Study First Posted: 2021-06-14
• Status Verified: 2021-08
• Last Update Submitted: 2021-08-05
• Last Update Posted: 2021-08-09
• Study Start: 2021-09-01
• Primary Completion: 2022-06-30
• Study Completion: 2022-12-30

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Proportion of patients with mean Hb ≥ 11.0 g/dL	weeks 16-24	After the use of roxadustat, the proportion of patients with mean Hb ≥ 11.0 g/dL

Secondary Outcome Measures

Name	Time	Method
Mean change in Hb	weeks 16-24

Trial Locations

Locations (1)

Children's Hospital, Zhejiang University School of Medicine; 🇨🇳 Hangzhou, Zhejiang, China