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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 (ASCEND-P)

Anemia Studies in CKD: Erythropoiesis via a Novel PHI Daprodustat -Pediatric (ASCEND-P) (ASCEND-P)

Takeuchi Rika

Syneos Health Clinical K.K.

10F Umeda Daibiru, 3-3-10 Umeda, Kita-ku, Osaka

rika.takeuchi@syneoshealth.com

Takeuchi Rika

Syneos Health Clinical K.K.

10F Umeda Daibiru, 3-3-10 Umeda, Kita-ku, Osaka

+81-70-1415-7743

rika.takeuchi@syneoshealth.com

Pending

Feb. 03, 2023

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

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%, or Ferritin <25 nanogram (ng)/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.
- 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) >2x upper limit of normal (ULN), bilirubin >1.5x ULN (unless bilirubin is fractionated and direct bilirubin <35%), and cirrhosis or current unstable liver or biliary disease.

Both

Renal anemia

All participants will receive oral daprodustat for a total of 52 weeks and will be followed for an additional 4 weeks after completing treatment. The lowest dose level defined (0.125 mg once daily [QD]) will be achieved by dosing 0.25 mg three times a week (TIW). All other dose steps involve QD dosing frequency.
The initial starting dose is dependent on the participants Hgb at baseline, ESA and dialysis status. Dose adjustment, according to an age specific algorithm within the range 0.125 mg to 24 mg QD equivalent, will be performed to achieve and maintain the Hgb within a target range.

Describe the safety of daprodustat, overall (all ages) and in each age group.
- Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to study intervention discontinuation.

Nov. 17, 2022

Nov. 17, 2022

No

US/Argentina/ Australia/Belgium/France/Germany/Italy/Netherlands/Russia/Spain/South Korea/Poland/Turkey/Canada/UK