Clinical trial NCT04621331: Investigating the Efficacy, Safety and PK of Roxadustat (FG-4592) for Treatment of Anemia in Pediatric Patients With CKD
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE3
Conditions: Anemia Associated With Chronic Kidney Disease
Keywords: Anemia, Chronic Kidney Disease (CKD)
Lead sponsor: FibroGen
Sponsor class: INDUSTRY
Collaborators: name: AstraZeneca
First posted: 2020-11-09
Enrollment: 0
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Proportion of patients with mean Hb ≥ 11.0 g/dL
timeFrame: Averaged over weeks 16-24
Secondary outcomes:
measure: Mean change in Hb
timeFrame: Baseline to averaged over weeks 16-24
measure: Time to first Hb response (this only applies to NDD patients)
timeFrame: From baseline without rescue therapy
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
state: New York
city: San Antonio
country: United States
facility: Investigative site
state: Texas
Study officials:
References: