Clinical Trials Register

Summary
EudraCT Number:	2021-000894-94
Sponsor's Protocol Code Number:	KOR-PED-201
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-08-30
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-000894-94

A.3

Full title of the trial

An Open-label, Single Arm Study to Evaluate the Pharmacokinetics of a Single Dose of Intravenous Difelikefalin in Adolescents Aged 12 to 17 Years on Haemodialysis

Studio in aperto, a braccio singolo volto a valutare la farmacocinetica di una dose singola di Difelikefalin per via endovenosa in adolescenti di età compresa tra 12 e 17 anni in emodialisi

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Single dose study to evaluate how intravenous administered difelikefalin is absorbed by and cleared from Adolescents on Haemodialysis

Studio a dose singola per valutare come difelikefalin somministrato per via endovenosa viene assorbito ed eliminato dagli adolescenti in emodialisi

A.3.2

Name or abbreviated title of the trial where available

Pharmacokinetics and Safety of Difelikefalin in Adolescents on Haemodialysis

Farmacocinetica e sicurezza di Difelikefalin in adolescenti in emodialisi

A.4.1

Sponsor's protocol code number

KOR-PED-201

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/172/2020

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Vifor Fresenius Medical Care Renal Pharma Ltd.
B.1.3.4	Country	Switzerland
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Vifor Fresenius Medical Care Renal Pharma Ltd.
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Vifor Fresenius Medical Care Renal Pharma Ltd.
B.5.2	Functional name of contact point	Clinical Trial Information Desk
B.5.3	Address:
B.5.3.1	Street Address	Flughofstrasse 61
B.5.3.2	Town/ city	Glattbrugg
B.5.3.3	Post code	CH-8152
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+41588518000
B.5.5	Fax number	+41588518001
B.5.6	E-mail	KOR-PED-201.study@viforpharma.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Difelikefalin
D.3.2	Product code	[CR845]
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	DIFELIKEFALIN
D.3.9.1	CAS number	1024828-77-0
D.3.9.2	Current sponsor code	CR845
D.3.9.3	Other descriptive name	DIFELIKEFALIN
D.3.9.4	EV Substance Code	SUB195302
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Renal disease in the final stage

Nefropatia allo stadio terminale

E.1.1.1

Medical condition in easily understood language

Renal disease in the final stage

Malattia renale allo stadio terminale

E.1.1.2

Therapeutic area

Diseases [C] - Female diseases of the urinary and reproductive systems and pregancy complications [C13]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10014646
E.1.2	Term	End stage renal disease (ESRD)
E.1.2	System Organ Class	10038359 - Renal and urinary disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	PT
E.1.2	Classification code	10077512
E.1.2	Term	End stage renal disease
E.1.2	System Organ Class	10038359 - Renal and urinary disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the PK profile of a single dose of difelikefalin in adolescent subjects aged 12 to 17 years on HD

Valutare il profilo farmacocinetico (PK) di una dose singola di difelikefalin in adolescenti di età compresa tra 12 e 17 anni in HD.

E.2.2

Secondary objectives of the trial

To evaluate the safety and tolerability of a single dose of difelikefalin in adolescent subjects aged 12 to 17 years on HD

Valutare la sicurezza e la tollerabilità di una dose singola di difelikefalin in adolescenti di età compresa tra 12 e 17 anni in HD.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.End-stage renal disease (ESRD) subjects who have been on HD for at least 3 months and are currently on HD at least 3 times per week. Subjects with or without associated pruritus may enroll.
2.Males or females 12 to 17 years of age, at the time of consent.
3.Has a prescription dry body weight =20 kg and =100 kg

1. Soggetti con malattia renale in stadio terminale (ESRD) che sono stati in HD per almeno 3 mesi e sono attualmente in trattamento con HD almeno 3 volte alla settimana. Possono essere arruolati soggetti con o senza prurito associato.
2. Soggetti di sesso maschile o femminile di età compresa fra 12 e 17 anni al momento del consenso.
3. Soggetti con un peso corporeo secco prescritto =20 kg e =100 kg.

E.4

Principal exclusion criteria

1.Known to be non compliant with HD treatments and deemed unlikely to complete the study by the Investigator (i.e., has a history of missed HD sessions due to non adherence in the past 2 months).
2.Planned or anticipated to receive a kidney transplant during the study. Note: Being on a kidney transplant list is not an exclusion criterion.
3.Serum alanine aminotransferase (ALT), aspartate aminotransferase (AST) greater than 2.5 × the reference upper limit of normal (ULN), or bilirubin greater than 4 × the ULN at screening.
4.Subject has known hypersensitivity to the study drug or any components of the difelikefalin formulation.
5.Known history of allergic reaction to opiates such as hives. Note: Side effects related to the use of opioids such as constipation or nausea would not exclude the subjects from the study.
6.Previous participation in this study.
7.Known or suspected history of alcohol, narcotic, or other drug abuse or substance dependence within 12 months prior to screening.
8.Acute or unstable medical condition(s) which in the opinion of the Investigator would pose undue risk to the subject or would impede complete collection of evaluable data.

1. Nota mancata compliance ai trattamenti di HD e improbabilità che lo studio sia completato secondo il giudizio dello Sperimentatore (ovvero, anamnesi di sessioni di HD saltate per mancata aderenza negli ultimi 2 mesi).
2. Trapianto di rene pianificato o previsto durante lo studio. Nota: Essere in lista per un trapianto di rene non costituisce criterio di esclusione.
3. Alanina aminotransferasi (ALT), aspartato aminotransferasi (AST) nel siero superiori a 2,5 volte il limite superiore della norma (ULN) o bilirubina superiore a 4 x ULN allo screening.
4. Soggetto con ipersensibilità nota al farmaco in studio o a qualsiasi componente della formulazione di difelikefalin.
5. Anamnesi nota di reazione allergica agli oppiacei, ad esempio orticaria. Nota: Gli effetti collaterali associati all'uso di oppioidi come la costipazione o la nausea non escluderebbero i soggetti dallo studio.
6. Precedente partecipazione a questo studio
7. Anamnesi nota o sospetta di abuso di alcol, narcotici o altre droghe o dipendenza da sostanze nei 12 mesi precedenti lo screening.
8. Condizioni mediche acute o instabili che, secondo il giudizio dello Sperimentatore, comporterebbero un rischio eccessivo per il soggetto o impedirebbero la raccolta completa di dati valutabili

E.5 End points

E.5.1

Primary end point(s)

Pharmacokinetics profile of difelikefalin after a single dose post haemodialysis

Profilo PK di difelikefalin dopo una dose singola post HD

E.5.1.1

Timepoint(s) of evaluation of this end point

Over a 3 day period

un periodo di 3 giorni

E.5.2

Secondary end point(s)

Incidence of adverse events (AEs) and serious adverse events (SAEs) after a single dose of difelikefalin. Change in vital signs, 12 lead electrocardiogram (ECG), and clinical safety laboratory evaluations after a single dose of difelikefalin

Incidenza di eventi avversi (AE) ed eventi avversi seri (SAE) dopo una dose singola di difelikefalin. Alterazione dei parametri vitali, dell'elettrocardiogramma a 12 derivazioni (ECG) e delle valutazioni cliniche di laboratorio di sicurezza dopo una dose singola di difelikefalin

E.5.2.1

Timepoint(s) of evaluation of this end point

Over 7 days

7 giorni successivi

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

tolerability

Tollerabilita

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Italy

United Kingdom

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

Last Subject Last Visit (LSLV)

Ultima visita ultimo paziente

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

Non applicabile

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2021-08-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-06-08

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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