- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06593392
Safety and Tolerability of Difelikefalin in Adolescents on Haemodialysis With Moderate-to-Severe Pruritus
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
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).
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Arms and Duration:
- This is a single-arm, open-label study. All participants will receive IV difelikefalin at a dose of 0.5 μg/kg of dry body weight after each HD treatment 3 times weekly for 12 weeks.
- 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 follow-up period of 7 (up to 10) days.
- If for any reason difelikefalin is interrupted for more than 1 week, the participant should be withdrawn from the study intervention.
Study Type
Enrollment (Estimated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Yael Carmeli
- Phone Number: +41 058 851 80 00
- Email: clinicaltrials@cslbehring.com
Study Locations
-
-
-
Glasgow, United Kingdom, G51 4TF
- Royal Hospital for Children Glasgow - 82600073
-
Contact:
- Use Central Contact
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 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:
- WI-NRS scores have been recorded for at least 4 days through a 7-day run-in period.
- 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:
- At least 2 single-pool Kt/V measurements ≥1.2 on different dialysis days
- At least 2 urea reduction ratio measurements ≥65% on different dialysis days
- 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.
Exclusion Criteria:
- Known to be non-compliant with HD treatments and deemed unlikely by the Investigator to complete the study
- Planned to receive a kidney transplant during the study. Note: being listed on a kidney transplant list is not an exclusion criterion.
- 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.
- Known or suspected history of alcohol, narcotic, or other drug abuse or substance dependence within 12 months prior to screening or participant with any alcoholic beverage intake of more than two units per day more than once per week.
- Participation in any other investigational device or drug study <30 days prior to screening, or current treatment with other investigational agent(s).
- Serum ALT, AST greater than 3× the reference ULN.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Single arm
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of adverse events (AEs)
Time Frame: Up to 18 weeks
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Pre-dose difelikefalin plasma concentrations (Cpre)
Time Frame: 12 weeks
|
To evaluate difelikefalin plasma concentrations after multiple administrations of 0.5 μg/kg difelikefalin in HD adolescents (≥12 to <18 years) with moderate-to-severe pruritus
|
12 weeks
|
Trough difelikefalin plasma concentrations (Ctrough)
Time Frame: 12 Weeks
|
To evaluate difelikefalin plasma concentrations after multiple administrations of 0.5 μg/kg difelikefalin in HD adolescents (≥12 to <18 years) with moderate-to-severe pruritus
|
12 Weeks
|
Maximum difelikefalin plasma concentrations (Cmax)
Time Frame: 12 Weeks
|
To evaluate difelikefalin plasma concentrations after multiple administrations of 0.5 μg/kg difelikefalin in HD adolescents (≥12 to <18 years) with moderate-to-severe pruritus
|
12 Weeks
|
Collaborators and Investigators
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Urogenital Diseases
- Pathologic Processes
- Male Urogenital Diseases
- Urologic Diseases
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Chronic Disease
- Disease Attributes
- Skin Manifestations
- Skin Diseases
- Renal Insufficiency
- Kidney Diseases
- Renal Insufficiency, Chronic
- Pruritus
Other Study ID Numbers
- KOR-PED-202
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Chronic Kidney Diseases
-
3-C Institute for Social DevelopmentUniversity of North Carolina, Chapel HillCompletedChronic Kidney Diseases | Chronic Kidney Disease Stage 5 | Chronic Kidney Disease stage4 | Pediatric Kidney Disease | Chronic Kidney Disease stage3 | Chronic Kidney Disease Stage V | Chronic Kidney Disease, Stage IV (Severe) | Chronic Kidney Disease Stage 2 | Chronic Kidney Disease, Stage IUnited States
-
Universiti Putra MalaysiaRecruitingChronic Kidney Diseases | Chronic Kidney Disease Stage 5 | Chronic Kidney Disease stage4 | Chronic Kidney Disease stage3 | Chronic Kidney Disease Requiring Chronic DialysisMalaysia
-
American Academy of Family PhysiciansUniversity of Colorado, Denver; National Institute of Diabetes and Digestive... and other collaboratorsCompletedChronic Kidney Disease | Chronic Renal Insufficiency | Chronic Kidney Insufficiency | Chronic Renal Diseases | Kidney Insufficiency, ChronicUnited States
-
Centre Hospitalier le MansLe Mans UniversiteWithdrawnFatigue | Chronic Kidney Disease Stage 5 | Chronic Kidney Disease stage3 | Chronic Kidney Failure | Chronic Kidney Disease, Stage 4 (Severe)
-
National Taiwan University HospitalCompletedChronic Kidney Disease stage4 | Chronic Kidney Disease stage3 | Chronic Kidney Disease Stage 2 | Chronic Kidney Disease Stage 1Taiwan
-
Centre Hospitalier le MansLe Mans UniversiteRecruitingFatigue | Chronic Kidney Disease Stage 5 | Chronic Kidney Disease stage4 | Chronic Kidney Disease Stage 3BFrance
-
University of WashingtonJohns Hopkins University; National Institute of Diabetes and Digestive and... and other collaboratorsRecruitingChronic Kidney Diseases | Acute Renal Failure | Acute Renal Injury | Acute Kidney Failure | Chronic Renal Insufficiency | Kidney Failure, Acute | Renal Insufficiency, Acute | Acute Renal Insufficiency | Acute Kidney Insufficiency | Renal Failure, Acute | Chronic Kidney Insufficiency | Chronic Renal Diseases | Kidney... and other conditionsUnited States
-
University of the State of Santa CatarinaUnknownKidney Diseases | Chronic Kidney Diseases | Hemodialysis | Chronic Renal Insufficiency | Renal Dialysis | Chronic Kidney Insufficiency | Chronic Renal DiseasesBrazil
-
Texas A&M UniversityWithdrawnChronic Kidney FailureUnited States
-
Lund UniversityBaxter Healthcare Corporation; Universidad de CórdobaCompletedEnd Stage Kidney Disease | Chronic Kidney Disease Requiring Chronic DialysisArgentina
Clinical Trials on Difelikefalin
-
Cara Therapeutics, Inc.Approved for marketing
-
Cara Therapeutics, Inc.CompletedPruritus | Atopic DermatitisUnited States, Canada
-
Cara Therapeutics, Inc.TerminatedPruritus | Notalgia ParestheticaUnited States, Canada, Poland, Spain, Germany
-
Vifor Fresenius Medical Care Renal PharmaTigermed Consulting Co., LtdCompletedEnd Stage Renal Disease on HemodialysisChina
-
Cara Therapeutics, Inc.Terminated
-
Cara Therapeutics, Inc.CompletedPruritus | Notalgia ParestheticaUnited States, Canada
-
Vifor Fresenius Medical Care Renal PharmaTigermed Consulting Co., LtdActive, not recruiting
-
Cara Therapeutics, Inc.CompletedUremic PruritusUnited States, Canada, Czechia, Australia, Germany, Hungary, Korea, Republic of, New Zealand, Poland, Taiwan, United Kingdom
-
Cara Therapeutics, Inc.Completed
-
Cara Therapeutics, Inc.CompletedUremic PruritusUnited States, Czechia, Hungary, Poland