Safety and Tolerability of Difelikefalin in Adolescents on Haemodialysis With Moderate-to-Severe Pruritus
brief summary
Rationale: * People with long term kidney disease who are on haemodialysis (a procedure for removing waste products from the blood) commonly develop a condition that makes their skin very itchy. * Difelikefalin is a medicine that can treat the itching related to long term kidney disease. * Clinical studies have shown difelikefalin to reduce itching in adults on haemodialysis, while being safe and tolerable. * The current study is being done in adolescents aged 12 to 17 years on haemodialysis who have moderate to severe itching related to long term kidney disease to assess if difelikefalin is safe in this age group. The aims of the study are: Main aim: To assess the safety of difelikefalin in adolescents who are on haemodialysis and have itching related to long term kidney disease Secondary aim: To measure the amount of difelikefalin that enters the blood in adolescents who are on haemodialysis and have itching related to long term kidney disease Study Design At least 18 adolescents, aged 12 to 17 years, who are on haemodialysis and have itching related to long term kidney disease will take part in this study. All study participants will receive difelikefalin 3 (or up to 4) times weekly for up to 12 weeks. The study duration for a participant is up to 17 to 18 weeks; during this period, participants will visit the clinic 3 times weekly (during their haemodialysis visits).
detailed description
The purpose of this study is to evaluate the safety and tolerability, as assessed by incidence of AEs, of 0.5 μg/kg IV difelikefalin in adolescent participants on HD (3 times weekly) with moderate-to-severe pruritus over 12 weeks. Additionally, difelikefalin plasma concentrations (Cmax and Ctrough) after multiple administrations will be evaluated at a few selected time points and the efficacy of difelikefalin in improving itching and health-related QoL will be investigated as exploratory.
Study details include:
* The study duration for a single participant will be up to 17 to 18 weeks. * The treatment duration for each participant will be up to 12 weeks. * The visit frequency will be 3 times weekly (during HD visits).
Trial Population:
Participants in this study must:
* Be male or female adolescent participants ≥12 to \<18 years of age at the time of informed consent/assent * Have CKD-aP on HD (at least 3 hours in duration 3 times weekly, for at least 12 weeks prior to the informed consent procedure) * Be able to continue HD without changing its frequency or method * Have a mean value WI-NRS score during the run-in period of \>4.0 (moderate-to-severe pruritus)
Participants in this study must not:
* Be planned to receive a kidney transplant during the study * Have known severe hepatic impairment (e.g., Child-Pugh Class C) or concurrent hepatic cirrhosis, serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3 times the reference upper limit of normal (ULN), significant systolic or diastolic heart failure, or concurrent malignancy (except excised basal cell or squamous cell carcinoma of the skin, or carcinoma in situ that has been excised or resected completely)
Number of Participants:
At least 18 participants evaluable for the primary safety endpoint will be enrolled in the study. Note: Enrolled means participants, or their legally acceptable representatives, agreement to participate in a clinical study following completion of the informed consent process and screening. A participant will only be considered enrolled if the informed consent/assent is not withdrawn prior to participating in any study activity after screening including the run-in period.
official title
KOR-PED-202 An Open-label, Single-arm Study to Evaluate the Safety and Tolerability of Intravenous Difelikefalin in Adolescents Aged 12 to 17 Years on Haemodialysis With Moderate-to-Severe Pruritus