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

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

This open-labeled, multicenter study is designed to evaluate the efficacy, safety and PK/PD of roxadustat in ESA-naïve and ESA-treated pediatric patients with CKD Stages 3, 4, and 5, as well as end-stage renal disease (ESRD) who are receiving either hemodialysis (HD) or peritoneal dialysis (PD). The study will enroll patients between the ages of 2 to <18 years in two sequential cohorts, with the older cohort of ages 12 to <18 years enrolled first. Approximately 30 patients will be enrolled in each age-based cohort.

Study Overview

• Status: Withdrawn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

• Study Type: Interventional
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann and Robert H. Lurie Children's Hospital of Chicago
  • Nebraska
    • Omaha, Nebraska, United States, 68198
      • University of Nebraska Medical Center
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
  • Texas
    • San Antonio, Texas, United States, 78215
      • Investigative Site

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

Important Inclusion Criteria:

• Clinically stable CKD in the opinion of the investigator.
• Estimated glomerular filtration rate (Bedside Schwartz formula) of < 60 ml/min/1.73 m2 (stage 3, 4 and 5 CKD) for non-dialysis patients, or patients who are receiving chronic dialysis (hemodialysis or peritoneal dialysis) for ESRD.
• For ESA-naïve patients (either NDD or DD; ESA-naïve is defined as those patients whose total duration of prior ESA exposure is ≤ 3 weeks within the preceding 12 weeks at the time informed consent is obtained), 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 ≥ 10.0 g/dL and ≤ 12.5 g/dL.
• Ferritin >50 ng/mL and transferrin saturation >10% (obtained from screening visit).
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2x upper limit of normal (ULN) and total bilirubin (Tbili) ≤1.5x ULN at randomization (obtained from screening visit).
• Serum folate and vitamin B12 > LLN (obtained from screening visit).

Important Exclusion Criteria:

• Uncontrolled hypertension as judged by the principal investigator in the 2 weeks prior to screening.
• Known hematologic disease other than anemia associated with CKD.
• Known malignancy within the past 5 years before screening.
• Any prior organ transplant or any planned organ transplant during the study period.
• Any RBC transfusion during the past 8 weeks before screening.
• Any condition leading to significant blood loss (e.g., gastrointestinal bleeding, surgical procedures) within 8 weeks before screening or during the screening period.
• History of chronic liver disease.
• Pure red cell aplasia (PRCA) or history of PRCA.
• History of epileptic seizures.
• History of hyperlipidemia or significant thrombotic/thromboembolic event (e.g., deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction).
• History of thrombosis of an arteriovenous fistula/graft within 12 weeks prior to enrollment.
• Any active systemic or significant infection or episode of peritonitis within 30 days of screening.
• Any statin use within 30 days of screening.
• 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
EXPERIMENTAL: Roxadustat; Starting doses of 20, 50, 70 or 100 mg based on weight.	Drug: Roxadustat; HIF-PH inhibitor for treatment of anemia in CKD; Other Names: FG-4592

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Proportion of patients with mean Hb ≥ 11.0 g/dL	Averaged over weeks 16-24

Secondary Outcome Measures

Outcome Measure	Time Frame
Mean change in Hb	Baseline to averaged over weeks 16-24
Time to first Hb response (this only applies to NDD patients)	From baseline without rescue therapy

Collaborators and Investigators

• Sponsor: FibroGen
• Collaborators: AstraZeneca

Study record dates

Study Major Dates

• Study Start (ACTUAL): May 23, 2022
• Primary Completion (ACTUAL): May 23, 2022
• Study Completion (ACTUAL): May 23, 2022

Study Registration Dates

• First Submitted: November 3, 2020
• First Submitted That Met QC Criteria: November 3, 2020
• First Posted (ACTUAL): November 9, 2020

Study Record Updates

• Last Update Posted (ACTUAL): July 5, 2022
• Last Update Submitted That Met QC Criteria: June 29, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Keywords: Anemia, Chronic Kidney Disease (CKD)
• Additional Relevant MeSH Terms: Urologic Diseases; Hematologic Diseases; Renal Insufficiency; Kidney Diseases; Renal Insufficiency, Chronic; Anemia
• Other Study ID Numbers: FGN-PED-CLIN-02

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: Yes
• Studies a U.S. FDA-regulated device product: No