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

Sponsor
FibroGen (Industry)
Overall Status
Withdrawn
CT.gov ID
NCT04621331
Collaborator
AstraZeneca (Industry)
0
4
1
0

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
0 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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
Actual Study Start Date :
May 23, 2022
Actual Primary Completion Date :
May 23, 2022
Actual Study Completion Date :
May 23, 2022

Arms and Interventions

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
  • Outcome Measures

    Primary Outcome Measures

    1. Proportion of patients with mean Hb ≥ 11.0 g/dL [Averaged over weeks 16-24]

    Secondary Outcome Measures

    1. Mean change in Hb [Baseline to averaged over weeks 16-24]

    2. Time to first Hb response (this only applies to NDD patients) [From baseline without rescue therapy]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    2 University of Nebraska Medical Center Omaha Nebraska United States 68198
    3 Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) New York New York United States 10029
    4 Investigative site San Antonio Texas United States 78215

    Sponsors and Collaborators

    • FibroGen
    • AstraZeneca

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    FibroGen
    ClinicalTrials.gov Identifier:
    NCT04621331
    Other Study ID Numbers:
    • FGN-PED-CLIN-02
    First Posted:
    Nov 9, 2020
    Last Update Posted:
    Jul 5, 2022
    Last Verified:
    Jun 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by FibroGen
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Jul 5, 2022