Clinical Trials Register

Summary
EudraCT Number:	2015-002401-10
Sponsor's Protocol Code Number:	TAL2015
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-09-10
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2015-002401-10

A.3

Full title of the trial

Pilot multicenter open label study on treatment with Sofosbuvir/Ledipasvir FDC for patients with thalassemia major and HCV infection.

Studio pilota multicentrico con LDV/SOF in pazienti con talassemia major e infezione cronica da HCV

A.3.1

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

Pilot multicenter open label study on treatment with Sofosbuvir/Ledipasvir FDC for patients with thalassemia major and HCV infection.

Studio pilota multicentrico con LDV/SOF in pazienti con talassemia major e infezione cronica da HCV

A.3.2

Name or abbreviated title of the trial where available

Pilot multicenter open label study on treatment with Sofosbuvir/Ledipasvir FDC for patients with tha

Studio pilota multicentrico con LDV/SOF in pazienti con talassemia major e infezione cronica da HCV

A.4.1

Sponsor's protocol code number

TAL2015

A.5.4

Other Identifiers

Name:

TAL2015

Number:

TAL2015

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	FONDAZIONE CASA SOLLIEVO DELLA SOFFERENZA OPERA DI SAN PIO DA PIETRELCINA OSPEDALE IRCCS
B.1.3.4	Country	Italy
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	IRCCS CASA SOLLIEVO DELLA SOFFERENZA
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	IRCCS OSPEDALE CASA SOLLIEVO DELLA SOFFERENZA DI SAN GIOVANNI ROTONDO
B.5.2	Functional name of contact point	ANNAMARIA CIACCIO
B.5.3	Address:
B.5.3.1	Street Address	VIALE CAPPUCCINI
B.5.3.2	Town/ city	SAN GIOVANNI ROTONDO
B.5.3.3	Post code	71013
B.5.3.4	Country	Italy
B.5.4	Telephone number	+390882410831
B.5.5	Fax number	+390882410813
B.5.6	E-mail	comitatoetico@operapadrepio.it

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	HARVONI - 90 MG/400 MG - COMPRESSE RIVESTITE CON FILM - USO ORALE - FLACONE (HDPE) - 28 COMPRESSE RIVESTITE CON FILM
D.2.1.1.2	Name of the Marketing Authorisation holder	GILEAD SCIENCES INTERNATIONAL LIMITED
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	HARVONI
D.3.2	Product code	700016355
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
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

Patients with thalassemia major and HCV infection either naïve or on nucleo(s)tide analogs treatment, data on Sofosbuvir-based treatment are currently lacking, while, due to the long infection duration, this category of patients has a urgent treatment need.

Pazienti con talassemia major e infezione cronica da HCV di genotipo ¼ (naïve o precedentemente trattati senza successo)

E.1.1.1

Medical condition in easily understood language

Pazienti con talassemia major e infezione cronica da HCV di genotipo ¼ (naïve o precedentemente trattati senza successo)

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	20.1
E.1.2	Level	PT
E.1.2	Classification code	10008912
E.1.2	Term	Chronic 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

Efficacy and safety of SOF/LDV without RBV in patients with HCV GT1 or 4 and thalassemia major

Valutare l’efficacia e la sicurezza di SOF/LDV in pazienti con HCV GT 1 e 4 e talassemia major.

E.2.2

Secondary objectives of the trial

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

o Patients >18 years; HCV RNA-positive and HCV Ab positive, HBsAg negative, genotype 1 or 4 infected; Naïve or prior treatment failure.

• Eliggibiità:
o Pazienti di età >18 anni;
o HCV RNA positivo, anti HCV positivo, con infezione da genotipo 1 e 4.
o Naïve al trattamento o che abbiano fallito un precedente ciclo di terapia per relapse o partial response.
o Proporzione di pazienti con cirrosi non superiore al 50%

E.4

Principal exclusion criteria

o HIV co-infected patients;
o HBsAg /HDV co-infected
o Creatinine clearance 50 ml/min
o PLT < 90.000/mm3;
o HCC and other neoplasia;
o Pregnancy;
o Breastfeeding
o Patients with Child Pugh > B7;
o Patients with AFP levels higher than 40.
o Chronic liver diseases other than HCV such as but not limited to: hemocromatosis, autoimmune hepatitis, metabolic liver diseases, alcoholic liver diseases, toxin exposure),
o Patients with severe or uncontrolled cardiac disease during the previous 24 weeks,
o Patients with debilitating diseases.

• HIV positività, HBsAg/HDV positività, valori di piastrine < 90.000/mm3, clearance della creatinina < 50 ml/min, gravidanza, allattamento, anamnesi positiva per grave malattia cardiaca pre-esistente, inclusa malattia cardiaca instabile, o non controllata, nei sei mesi precedenti lo studio, pazienti con situazioni cliniche gravemente debilitanti, pazienti in emodialisi, pazienti con cirrosi scompensata CPT > B7, pazienti con emocromatosi, epatiti autoimmuni, malattie metaboliche del fegato, malattia epatica da abuso di alcool, esposizione a sostanze tossiche, presenza di HCC o di altra neoplasia, valori alfafeto proteina > 40 ng/ml.

E.5 End points

E.5.1

Primary end point(s)

• Post-treatment sustained virological response (pTVR): SVR12.

• Proporzione dei pazienti con risposta virologica sostenuta post-trattamento: SVR12.

E.5.1.1

Timepoint(s) of evaluation of this end point

24 weeks

24 settimane

E.5.2

Secondary end point(s)

• Number of patients developing side effects while on treatment.leading to treatment discontinuation

• Numero di pazienti che sviluppino durante il trattamento effetti collaterali che portino all’interruzione della terapia

E.5.2.1

Timepoint(s) of evaluation of this end point

24 weeks

24 settimane

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)

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

Yes

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

Yes

E.8.1.7.1

Other trial design description

CONTROLLO STORICO

HISTORICAL CONTROL

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

Yes

E.8.2.3.1

Comparator description

CONTROLLO STORICO

HISTORICAL CONTROL

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.8.5.1

Number of sites anticipated in the EEA

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

Information not present in EudraCT

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

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

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)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

100

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

100

F.4.2

For a multinational trial

F.4.2.1

In the EEA

100

F.4.2.2

In the whole clinical trial

100

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)

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

2015-10-12

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-09-14

P. End of Trial
P.	End of Trial Status	Ongoing

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