Safety and Tolerability of Difelikefalin in Adolescents on Haemodialysis With Moderate-to-Severe Pruritus

NCT06593392 | Recruiting Phase 2

• 1 other identifier: KOR-PED-202
• Study Type: interventional
• Target: 18
• Locations: 7 countries
• Sites: 14

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).

Conditions

Pruritus; Chronic Kidney Diseases

Keywords

Chronic kidney disease associated pruritus

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events (AEs)

  To evaluate the safety of 0.5 μg/kg difelikefalin in HD adolescents (≥12 to \<18 years) with moderate-to-severe pruritus

  Up to 18 weeks

Secondary Outcomes (3)

• Pre-dose difelikefalin plasma concentrations (Cpre)

  12 weeks

• Trough difelikefalin plasma concentrations (Ctrough)

  12 Weeks

• Maximum difelikefalin plasma concentrations (Cmax)

  12 Weeks

Study Arms (1)

Single arm

EXPERIMENTAL

Drug: Difelikefalin

Interventions

Difelikefalin DRUG

The study includes a screening period of up to 4 weeks (including a 7-day run-in period during the week prior to enrolment), a study treatment period of 12 weeks, and a safety follow-up visit at 7 (up to 10) days after EoT. Total study duration for a single participant is up to 17 to 18 weeks.

Single arm

Eligibility Criteria

• Age: 12 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• \. Participant must be ≥12 to \<18 years of age at the time of informed consent.
• \. Participants with CKD on HD 3 times weekly for at least 12 weeks prior to the informed consent procedure who can continue HD without changing its frequency or method.
• \. Participants whose WI-NRS score in the 7-day run-in period (meets both of the below:
• a) WI-NRS scores have been recorded for at least 4 days through a 7-day run-in period.
• b) The mean value of the recorded WI-NRS scores is \>4.0
• \. Over the last 3 months prior to screening, the participant has had at least 1 of the following:
• a) At least 2 single-pool Kt/V measurements ≥1.2 on different dialysis days
• b) At least 2 urea reduction ratio measurements ≥65% on different dialysis days
• c) 1 single-pool Kt/V measurement ≥1.2 and 1 urea reduction ratio measurement ≥65% on different dialysis day
• \. Prescription dry body weight ≥20 kg
• \. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• \. Participant and/or legal guardian (as required) is capable of providing the appropriate signed informed consent and where appropriate, assent.

You may not qualify if:

• \. Known to be non-compliant with HD treatments and deemed unlikely by the Investigator to complete the study
• \. Participants with itching caused by conditions other than chronic renal failure or complications of chronic renal failure, which could in the opinion of the Investigator affect the efficacy evaluation (e.g., atopic dermatitis, chronic urticaria).
• \. Participants with localised itch restricted to the palms of the hands.
• \. Participants with pruritus only during the dialysis session (by participant report).
• \. Participants with known concurrent hepatic cirrhosis or severe hepatic impairment (e.g., Child-Pugh Class C).
• \. Significant systolic or diastolic heart failure (e.g., New York Heart Association Class IV congestive heart failure).
• \. Participants with concurrent malignancy except excised basal cell or squamous cell carcinoma of the skin, or carcinoma in situ that has been excised or resected completely.
• \. Severe mental illness or cognitive impairment (e.g., dementia) or other concurrent mental disorder that, in the opinion of the Investigator, would compromise the validity of study measurements.
• \. Conditions associated with clinically important disruptions to the blood brain barrier (for example, primary brain malignancies, CNS metastases or other inflammatory conditions, active multiple sclerosis, advanced Alzheimer's disease) that in the Investigator's opinion may be associated with unacceptable risk for CNS effects.
• \. Acute or unstable medical condition(s) that in the Investigator's opinion, may be associated with increased risk to the participant, or may interfere with study assessments, outcomes, or the ability to provide written informed consent or comply with study procedures.
• \. Participant is receiving ongoing ultraviolet B treatment and anticipates receiving such treatment during the study.
• \. New or change of treatment received for itch including antihistamines and corticosteroids (oral, IV, or topical) within 14 days prior to screening.
• \. New or change of prescription for opioids, gabapentin, or pregabalin within 14 days prior to screening.
• \. Participant is receiving prohibited medication (e.g., nalfurafine hydrochloride, opioid antagonists) that cannot be stopped at least 14 days before enrolment in the study.
• \. Participant has known hypersensitivity to the study intervention or any components of the difelikefalin formulation.

Sponsors & Collaborators

• Vifor Fresenius Medical Care Renal Pharma: lead

Study Sites (14)

Sichuan University-West China Second University Hospital

Chengdu, 610066, China

RECRUITING

Guangzhou Women and Children's Medical Center

Guangzhou, 510600, China

RECRUITING

The Children's Hospital Zhejiang University School of Medicine

Hangzhou, 310003, China

RECRUITING

The Children's Hospital of Fudan University

Shanghai, 201102, China

RECRUITING

Huazhong University of Science and Technology-Tongji Medical College-The Central Hospital of Wuhan

Wuhan, 430032, China

RECRUITING

Pan and Aglaia Kyriakou Children's Hospital

Athens, 11527, Greece

RECRUITING

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

RECRUITING

Schneider Children's Medical Center of Israel

Petah Tikva, 49202, Israel

RECRUITING

King Abdullah Specialized Children's Hospital (KASCH)

Riyadh, 14611, Saudi Arabia

RECRUITING

Hospital Sant Joan de Déu

Barcelona, " 08950", Spain

RECRUITING

Hospital Universitario Valle de Hebron

Barcelona, 8035, Spain

RECRUITING

Jalila Children's Specialty Hospital

Dubai, 25314, United Arab Emirates

RECRUITING

Royal Hospital for Children Glasgow

Glasgow, G51 4TF, United Kingdom

RECRUITING

Alder Hey Childrens Hospital

Liverpool, L14 5AB, United Kingdom

RECRUITING

MeSH Terms

Conditions

Pruritus; Renal Insufficiency, Chronic

Interventions

difelikefalin

Condition Hierarchy (Ancestors)

Skin Diseases; Skin and Connective Tissue Diseases; Skin Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes

Central Study Contacts

Yael Carmeli

CONTACT

+41 058 851 80 00; clinicaltrials@cslbehring.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open label single arm phase IIb clinical trial

Study Record Dates

• First Submitted: September 9, 2024
• First Posted: September 19, 2024
• Study Start (Estimated): May 13, 2026
• Primary Completion (Estimated): August 15, 2029
• Study Completion (Estimated): August 15, 2029
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
• Access Criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

Locations

ES (2); GB (2); IL (2); SA (1); AE (1); CN (5); GR (1)