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

NCT ID: NCT04621331

Last Updated: 2022-07-05

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE3
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-05-23
• Study Completion Date: 2022-05-23

Brief Summary

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.

Conditions

Anemia Associated With Chronic Kidney Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Roxadustat

Starting doses of 20, 50, 70 or 100 mg based on weight.

Group Type: EXPERIMENTAL

Roxadustat

Intervention Type: DRUG

HIF-PH inhibitor for treatment of anemia in CKD

Interventions

Roxadustat

HIF-PH inhibitor for treatment of anemia in CKD

Intervention Type: DRUG

Other Intervention Names

FG-4592

Eligibility Criteria

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).

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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

AstraZeneca

INDUSTRY

Sponsor Role: collaborator

FibroGen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

University of Nebraska Medical Center

Omaha, Nebraska, United States

Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)

New York, New York, United States

Investigative site

San Antonio, Texas, United States

Countries

United States

Other Identifiers

FGN-PED-CLIN-02

Identifier Type: -

Identifier Source: org_study_id