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

An Open-label, One-center Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Pediatric Patients With Chronic Kidney Disease

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.

Study Overview

• Status: Recruiting
• Conditions: Anemia Associated With Chronic Kidney Disease
• Intervention / Treatment: Drug: Roxadustat

Detailed Description

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

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: jianhua Mao, MD; Phone Number: 086-571-86670015; Email: maojh88@zju.edu.cn

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Recruiting
      • Children's Hospital, Zhejiang University School of Medicine
      • Contact: Mao Jianhua; Phone Number: 13616819071; Email: maojh88@zju.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
OTHER: Roxadustat; Starting doses of 20, 50, 70 or 100 mg/30,70,90,or 120mg based on weight and dialysis or not.	Drug: Roxadustat; Starting doses of 20, 50, 70 or 100 mg/30,70,90,or 120mg based on weight and dialysis or not.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients with mean Hb ≥ 11.0 g/dL	After the use of roxadustat, the proportion of patients with mean Hb ≥ 11.0 g/dL	weeks 16-24

Secondary Outcome Measures

Outcome Measure	Time Frame
Mean change in Hb	weeks 16-24

Collaborators and Investigators

• Sponsor: Mao Jianhua
• Investigators: Principal Investigator: jianhua Map, MD, Children's Hospital, Zhejiang University School of Medicine

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): September 1, 2021
• Primary Completion (ANTICIPATED): June 30, 2022
• Study Completion (ANTICIPATED): December 30, 2022

Study Registration Dates

• First Submitted: June 8, 2021
• First Submitted That Met QC Criteria: June 8, 2021
• First Posted (ACTUAL): June 14, 2021

Study Record Updates

• Last Update Posted (ACTUAL): August 9, 2021
• Last Update Submitted That Met QC Criteria: August 5, 2021
• Last Verified: August 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Hematologic Diseases; Renal Insufficiency; Kidney Diseases; Renal Insufficiency, Chronic; Anemia
• Other Study ID Numbers: Roxadustat for pediatric CKD

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No