Clinical Trials Register

Summary
EudraCT Number:	2020-003879-18
Sponsor's Protocol Code Number:	SOF-VEL/PED/2020
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2020-11-17
Trial 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

Poland - Office for Medicinal Products

A.2

EudraCT number

2020-003879-18

A.3

Full title of the trial

Treatment of chronic hepatitis C in children aged 6 – 18 years using a pangenotypic direct-acting antiviral (sofosbuvir/velpatasvir)

Leczenie dzieci w wieku 6 – 18 lat z przewlekłym wirusowym zapaleniem wątroby typu C przy użyciu pangenotypowego leku o bezpośrednim działaniu przeciwwirusowym (sofosbuwir/welpataswir)

A.3.1

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

Treatment of chronic hepatitis C in children aged 6 – 18 years using a direct-acting antiviral (sofosbuvir/velpatasvir) acting against all HCV genotypes

Leczenie dzieci w wieku 6 – 18 lat z przewlekłym wirusowym zapaleniem wątroby typu C przy użyciu leku o bezpośrednim działaniu przeciwwirusowym działającego na wszystkie genotypy wirusa HCV

A.4.1

Sponsor's protocol code number

SOF-VEL/PED/2020

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Medical University of Warsaw
B.1.3.4	Country	Poland
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Medical Research Agency
B.4.2	Country	Poland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Medical University of Warsaw
B.5.2	Functional name of contact point	Maria Pokorska-Śpiewak
B.5.3	Address:
B.5.3.1	Street Address	Wolska Str. 37
B.5.3.2	Town/ city	Warsaw
B.5.3.3	Post code	01-201
B.5.3.4	Country	Poland
B.5.4	Telephone number	48223355250
B.5.5	Fax number	48223355379
B.5.6	E-mail	maria.pokorska-spiewak@wum.edu.pl

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	Yes
D.2.1.1.1	Trade name	Epclusa (Sofosbuvir/Velpatasvir)
D.2.1.1.2	Name of the Marketing Authorisation holder	Gilead Sciences Ireland UC, Carrigtohill, County Cork, T45 DP77, Ireland
D.2.1.2	Country which granted the Marketing Authorisation	Poland
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	Epclusa (sofosbuvir/velpatasvir)
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sofosbuvir
D.3.9.1	CAS number	1190307-88-0
D.3.9.3	Other descriptive name	SOFOSBUVIR
D.3.9.4	EV Substance Code	SUB121170
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	400
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	VELPATASVIR
D.3.9.3	Other descriptive name	Velpatasvir
D.3.9.4	EV Substance Code	SUB180213
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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.IMP: 2
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	Yes
D.2.1.1.1	Trade name	Epclusa (Sofosbuvir/Velpatasvir)
D.2.1.1.2	Name of the Marketing Authorisation holder	Gilead Sciences Ireland UC, Carrigtohill, County Cork, T45 DP77, Ireland
D.2.1.2	Country which granted the Marketing Authorisation	Poland
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	Epclusa sofosbuvir/velpatasvir)
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sofosbuvir
D.3.9.1	CAS number	1190307-88-0
D.3.9.3	Other descriptive name	SOFOSBUVIR
D.3.9.4	EV Substance Code	SUB121170
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	200
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	VELPATASVIR
D.3.9.3	Other descriptive name	Velpatasvir
D.3.9.4	EV Substance Code	SUB180213
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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

Chronic hepatitis C

Przewlekłe wirusowe zapalenie wątroby typu C

E.1.1.1

Medical condition in easily understood language

Chronic hepatitis C caused by hepatitis C virus (HCV) infection

Przewlekłe wirusowe zapalenie wątroby typu C wywołane przez wirus HCV

E.1.1.2

Therapeutic area

Diseases [C] - Virus Diseases [C02]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10019744
E.1.2	Term	Hepatitis C
E.1.2	System Organ Class	10021881 - Infections and infestations

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Main objective: to cure patients with chronic hepatitis C: evaluation of the efficacy of sofosbuvir / velpatasvir (SOF / VEL) therapy assessed as SVR (sustained viral response) 12 weeks after the end of treatment

Cel główny: wyleczenie pacjentów z przewlekłym wirusowym zapaleniem wątroby typu C: ocena skuteczności terapii sofosbuwirem/welpataswirem (SOF/VEL) oceniana jako SVR (sustained viral response, trwała odpowiedź wirusologiczna) po 12 tygodniach od zakończenia leczenia;

E.2.2

Secondary objectives of the trial

Secondary goals:
• assessment of the safety of therapy,
• monitoring of patients during treatment,
• long-term post-treatment evaluation of patients - assessment of the durability of treatment effects and possible long-term side effects, including effects on children's development

Cele drugorzędowe:
• ocena bezpieczeństwa terapii,
• monitorowanie pacjentów w trakcie leczenia,
• długoterminowa ocena pacjentów po leczeniu – ocena trwałości efektów terapii i ewentualnych odległych działań niepożądanych, w tym wpływu na rozwój dzieci

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• written, voluntary informed consent to participate in the study
• diagnosis of chronic hepatitis C
• age 6-18 years

• pisemna, dobrowolna i świadoma zgoda na udział w badaniu
• rozpoznanie przewlekłego wirusowego zapalenia wątroby typu C
• wiek 6-18 lat

E.4

Principal exclusion criteria

• refusal to consent to participate in the study
• hypersensitivity to the active substance or excipients of the drug
• no HCV RNA in blood serum in the presence of anti-HCV antibodies
• pregnancy or breastfeeding
• hepatitis B infection (active or past)
• simultaneous intake of drugs that affect the interaction with active substances, including rosuvastatin or strong inhibitors of P-glycoprotein
• active addiction to alcohol or intoxicants

• brak zgody na udział w badaniu
• nadwrażliwość na substancję czynną lub substancje pomocnicze leku
• niestwierdzenie HCV RNA w surowicy krwi przy obecności przeciwciał anty-HCV
• ciąża lub karmienie piersią
• zakażenie wirusem zapalenia wątroby typu B (aktywne lub przebyte)
• równoczesne przyjmowanie leków wpływających na interakcję z substancjami czynnymi, w tym rozuwastatyny lub silnych inhibitorów glikoproteiny P
• czynne uzależnienie od alkoholu lub środków odurzających

E.5 End points

E.5.1

Primary end point(s)

Assessment of the efficacy of sofosbuvir / velpatasvir (SOF / VEL) therapy assessed as SVR (sustained viral response) 12 weeks after the end of treatment;

Ocena skuteczności terapii sofosbuwirem/welpataswirem (SOF/VEL) oceniana jako SVR (sustained viral response, trwała odpowiedź wirusologiczna) po 12 tygodniach od zakończenia leczenia;

E.5.1.1

Timepoint(s) of evaluation of this end point

12 weeks after the end of the treatment

12 tygodni od zakończenia leczenia

E.5.2

Secondary end point(s)

• change in liver enzyme levels
• change in total bilirubin
• reduction in HCV viral load after 4, 8 and 12 weeks of treatment
• change in the stage of liver fibrosis in an elastographic examination,
• change of the patient's physical parameters (body weight, height, BMI z-score),
• assessment of the occurrence of side effects of treatment,
• assessment of the impact of clinical and laboratory factors (including the HCV genotype) on the effectiveness of treatment,
• patients' quality of life assessment

• zmiana stężenia enzymów wątrobowych
• zmiana stężenia bilirubiny całkowitej
• obniżenie wiremii HCV po 4, 8 i 12 tygodniach leczenia
• zmiana zaawansowania włóknienia wątroby w badaniu elastograficznym,
• zmiana parametrów fizycznych pacjenta (masy ciała, wzrostu, BMI z-score),
• ocena występowania działań niepożądanych leczenia,
• ocena wpływu czynników klinicznych oraz laboratoryjnych (w tym genotypu HCV) na skuteczność leczenia,
• ocena jakości życia pacjentów

E.5.2.1

Timepoint(s) of evaluation of this end point

4, 8, 12 weeks after the beginning of the treatment; 12 weeks and a year after the end of the treatment

Po 4, 8, 12 tygodniach leczenia, po 12 tygodniach i po roku od zakończenia leczenia

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

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

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

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)

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

Yes

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

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

Yes

E.8.4

The trial involves multiple sites in the 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

E.8.6.2

Trial being conducted completely outside of the EEA

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

LVLS

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

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

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

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.4.2.2

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)

The patients will be controlled a year after the end of the treatment

Pacjenci będa kontrolowani po roku od zakończenia leczenia

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

2021-04-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-09-14

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2023-10-27

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