Clinical Trials Register

Summary
EudraCT Number:	2011-004657-11
Sponsor's Protocol Code Number:	VI-Plt-01
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:	2012-08-31
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

2011-004657-11

A.3

Full title of the trial

Open label multicenter study of Eltrombopag for the treatment of Immune ThrombocytoPenia (ITP) secondary to Chronic Lymphoproliferative Disorders (LPDs)

A.3.1

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

Multicenter study of Eltrombopag for the treatment of Immune ThrombocytoPenia (ITP) secondary to Chronic Lymphoproliferative Disorders (LPDs)

Studio clinico di trattamento con Eltrombopag della piastrinopenia autoimmune secondaria a malattie linfoproliferative

A.3.2

Name or abbreviated title of the trial where available

Eltrombopag for secondary Immune ThrombocytoPenia

A.4.1

Sponsor's protocol code number

VI-Plt-01

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 PROGETTO EMATOLOGIA
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	Glaxo Smithkline
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	ULSS 6 Vicenza
B.5.2	Functional name of contact point	Ematologia
B.5.3	Address:
B.5.3.1	Street Address	Via Rodolfi 37
B.5.3.2	Town/ city	Vicenza
B.5.3.3	Post code	36100
B.5.3.4	Country	Italy
B.5.4	Telephone number	0444 753626
B.5.5	Fax number	0444 920708
B.5.6	E-mail	rodeghiero@hemato.ven.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	REVOLADE*28CPR RIV 25MG
D.2.1.1.2	Name of the Marketing Authorisation holder	GLAXOSMITHKLINE SpA
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	Yes
D.2.5.1	Orphan drug designation number	EMEA/H/C/001110
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Coated tablet
D.3.4.1	Specific paediatric formulation	No
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	ELTROMBOPAG OLAMINE
D.3.9.1	CAS number	496775-62-3
D.3.9.4	EV Substance Code	SUB30141
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	up to
D.3.10.3	Concentration number	150
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

Immune ThrombocytoPenia (ITP) secondary to Chronic Lymphoproliferative Disorders (LPDs)

Piastrinopenia autoimmune secondaria a malattie linfoproliferative

E.1.1.1

Medical condition in easily understood language

autoimmune thrombocytopenia decrease in platelets) as a complication of lymphoproliferative disease (lymphoma or Chronic leukemia)

piastrinopenia autoimmune (calo delle piastrine) come complicanza della malattia linfoproliferativa (linfoma o leucemia linfatica cronica)

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	15.0
E.1.2	Level	PT
E.1.2	Classification code	10050245
E.1.2	Term	Autoimmune thrombocytopenia
E.1.2	System Organ Class	10005329 - Blood and lymphatic system 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

Proportion of responders to eltrombopag as defined by changes in the platelet count, in platelet transfusion requirements and/or in the bleeding symptoms during the 6 months of treatment.

Deteminazione della percentuale dei soggetti che rispondono a eltrombopag come definito dai cambiamenti della conta piastrinica, della necessità di trasfusioni di piastrine e/o dai sintomi emorragici durante i 6 mesi di trattamento.

E.2.2

Secondary objectives of the trial

Assessment of the safety profile of eltrombopag in patients with LPD using the CTCAE criteria.

Valutazione il profilo di sicurezza di eltrombopag nei pazienti con LPDs utilizzando i criteri CTCAE.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1) Diagnosis of any of the following B-cell chronic LPD, as defined by WHO 2008 classification: small lymphocytic lymphoma/chronic lymphocytic leukemia, follicular lymphoma, marginal zone lymphoma, mantle cell lymphoma, lymphoplasmacytic lymphoma, hairy cell leukemia, Hodgkin’s lymphoma. 2) Occurrence of ITP diagnosed on the basis of predefined criteria. 3) Not likely to necessitate any cytotoxic treatment for the following 6 months, according to clinical stage and performance status. 4) Platelet count less than 30,000/µL; patients with platelet count between 30 and 50,000/µL only in case of bleeding signs or symptoms. 5) Age greater than or equal to 18 years. 6) Absence of a personal or family (up to first degree relatives) history of venous or arterial thromboembolism. 7) ECOG performance status ≤2. 8) Adequate liver and renal function. 9) Absence of active Hepatitis B (HBsAg+ or HBV-DNA+), Hepatitis C (HCV-Ab+), or HIV infection. 9) Provided informed consent. 10) Negative pregnancy test or lactation 11) No antiplatelet or anticoagulant ongoing treatments

1)Diagnosi di una delle seguenti LPDs definite secondo la classificazione WHO del 2008: piccolo linfoma linfocitico/leucemia linfatica cronica, linfoma follicolare, linfoma della zona marginale, linfoma a cellule del mantello, linfoma linfoplasmacellulare, leucemia a cellule capellute, linfoma di Hodgkin. 2)Presenza di ITP diagnosticata sulla base di criteri predefiniti. 3)Pazienti per i quali non è previsto un trattamento chemioterapico per i successivi 6 mesi, a seconda dello stadio clinico e performance status. 4)Conta piastrinica inferiore di 30000/µL; pazienti con conta piastrinica tra 30000µL e 50000µL solo in caso di sintomi o episodi di sanguinamento. 5)Età ≥18 anni. 6)Assenza di una storia personale o familiare (fino a parenti di primo grado) di tromboembolia venosa o arteriosa. 7)ECOG performance status ≤2. 8)Adeguata funzionalità epatica e renale. 9)Sierologia negativa per Epatite B (HBsAg o HBV-DNA), Epatite C (HCV-Ab) o HIV. 10)Consenso informato firmato dal paziente. 11)Test di gravidanza negativo o in allattamento. 12) Nessun trattamento antipiastrinico o anticoagulante in corso.

E.4

Principal exclusion criteria

1) Subjects with any clinically relevant abnormality, other than LPD or ITP, or any other medicalcondition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study. 2) Subjects with any concurrent malignant disease other that the LPD and/or a recent history of cancer treatment with systemic chemotherapy and/or radiotherapy. Exception: Subjects with a history of completely resected non-melanoma skin cancer or successfully treated in situ carcinoma are eligible. 3) Subjects with screening bone marrow fibers of either MF Grade 3 using European Consensus scale or Grade 4 using Bauermeister scale (see Appendix 1). 4) Subjects with a QTc >450 msec or > 480 msec for subjects with Bundle Branch Block. 5) Subjects with recent history of alcohol/drug abuse as determined by the investigator.

1)Soggetti con qualsiasi anormalità clinicamente rilevante, diversa da ITP o LPD, o qualsiasi altra condizione medica che a parere dello sperimentatore rende il soggetto inadatto alla partecipazione allo studio 2)Soggetti con neoplasie concomitanti e/o un trattamento recente con chemioterapia sistemica o radioterapia. Eccezione:soggetti con una storia di resezione completa di un cancro della pelle (non-melanoma) o un carcinoma trattato in situ. 3) soggetti con fibre midollari di grado 3 o 4 4) soggetti con QTc >450 msec o <480 msec per soggetti con blocco di branca 5)soggetti con una storia recente di abuso di alcol/droghe

E.5 End points

E.5.1

Primary end point(s)

Proportion of responders to eltrombopag as defined by changes in the platelet count, in platelet transfusion requirements and/or in the bleeding symptoms during the 6 months of treatment.

E.5.1.1

Timepoint(s) of evaluation of this end point

during the 6 months of treatment.

6 mesi di terapia

E.5.2

Secondary end point(s)

Assessment of the safety profile of eltrombopag in patients with LPD using the CTCAE criteria.

Valutazione il profilo di sicurezza di eltrombopag nei pazienti con LPDs utilizzando i criteri CTCAE.

E.5.2.1

Timepoint(s) of evaluation of this end point

for the duration of the study

durata dello studio

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

E.8.1.1

Randomised