An Integrated Pharmacokinetic and Safety Open-label Basket Trial of Daprodustat for the Treatment of Anemia Associated With Chronic Kidney Disease in Male and Female Children and Adolescents Aged 3 Months to Under 18 Years Requiring or Not Requiring Dialysis

214066

This is an international, multicenter trial, evaluating pharmacokinetics (PK) (4 weeks), safety (52 weeks), and hemoglobin (Hgb) response (52 weeks) to daprodustat in children and adolescent participants with anemia associated with chronic kidney disease (CKD) incorporating 2 independent sub-trials (Non dialysis \[ND\] and Dialysis \[D\]). This study will enroll participants with anemia associated with CKD, in 2 distinct sub-populations differing only by their CKD stage and dialysis requirement (ND: CKD stage 3 to 5 not yet receiving dialysis and D: CKD stage 5d undergoing peritoneal dialysis \[PD\] or hemodialysis \[HD\]). The maximum duration of the study will be approximately 60 weeks, including Screening period (up to 4 weeks), treatment period (52 weeks), and follow-up period (4 weeks). Outcome measures are identical for the ND and D sub-trials, but will be separately assessed in each sub- trials, overall and within each age subgroups (12 to less than \[\<\] 18 years, 6 to \<12 years, 2 to \<6 years, and 3 months to \<2 years). Except for PK and dose change, which is within each age group only.

2023-09-06

2030-07-05

2030-07-05

Recruiting

Early phase I

120

Inclusion Criteria: * Participant must be 3 months to less than (\<)18 years of age. * Anemia associated with CKD stage 3, 4, 5 (not on dialysis) or who have dialysis-dependent CKD, defined as Hgb 7.0 to 11.0 g/dL (if not using erythropoiesis stimulating agents \[ESAs\]) or Hgb 9.5 to 12.0 g/dL if using ESAs. * Written informed consent or assent as appropriate. Exclusion Criteria: * Kidney transplant recipient with a functioning allograft. * Scheduled for elective kidney transplantation within 3 months. * Transferrin saturation (TSAT) \< 20 percent (%), or Ferritin \<25 nanogram (ng)/milliliter (mL). * History of bone marrow aplasia or pure red cell aplasia. * Active hemolysis. * Other causes of anemia. * Active gastrointestinal bleeding within the last 4 weeks. * Active or previous malignancy within the last 2 years. * Acute or chronic infection requiring antimicrobial therapy. * History of significant thrombotic or thromboembolic events within the last 8 weeks. * Heart failure (HF) New York Heart Association (NYHA) Class IV * Uncontrolled hypertension. * Alanine aminotransferase (ALT) \>2× upper limit of normal (ULN), bilirubin \>1.5× ULN (unless bilirubin is fractionated and direct bilirubin \<35%), and cirrhosis or current unstable liver or biliary disease.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'This is an open-label single arm trial, where all participants are on active treatment with daprodustat.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 120, 'type': 'ESTIMATED'}}

2024-03-27