Clinical Trials Register

Summary
EudraCT Number:	2018-000452-18
Sponsor's Protocol Code Number:	ETB115J2411
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2020-12-15
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

2018-000452-18

A.3

Full title of the trial

A phase II, open-label, prospective, single-arm, study to assess ability of eltrombopag to induce sustained remission in subjects with ITP who are refractory or relapsed after first-line steroids (TAPER)

Studio di Fase II, in aperto, prospettico, a braccio singolo, per valutare la capacità di eltrombopag di indurre remissione mantenuta nei soggetti con ITP refrattari o in recidiva dopo corticosteroidi di prima linea (TAPER)

A.3.1

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

The study will determine if subjects are able to stop eltrombopag treatment while maintaining acceptable platelet levels after their disease has become resistant to treatment (refractory) or has a reoccurrence of symptoms after initial treatment with steroids (relapse)

Lo studio determinerà se i soggetti sono
in grado di interrompere il trattamento con eltrombopag mantenendo livelli accettabili di piastrine dopo che la loro malattia è diventata resistente al trattamento (refrattario) o se si è manifestata una ricomparsa dei sintomi dopo il trattamento iniziale con steroidi (recidiva).

A.3.2

Name or abbreviated title of the trial where available

The study will determine if subjects are able to stop eltrombopag treatment while maintaining accept

Studio di valutazione della capacità di eltrombopag di indurre remissione mantenuta in soggetti con

A.4.1

Sponsor's protocol code number

ETB115J2411

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	NOVARTIS PHARMA AG
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	Novartis Pharma AG
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	NOVARTIS FARMA S.p.A.
B.5.2	Functional name of contact point	Drug Regulatory Affairs
B.5.3	Address:
B.5.3.1	Street Address	Largo Umberto Boccioni, 1
B.5.3.2	Town/ city	Origgio (VA)
B.5.3.3	Post code	21040
B.5.3.4	Country	Italy
B.5.4	Telephone number	029641
B.5.5	Fax number	029659066
B.5.6	E-mail	info.studiclinici@novartis.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	Yes
D.2.1.1.1	Trade name	REVOLADE
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm Ltd
D.2.1.2	Country which granted the Marketing Authorisation	Ireland
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	Eltrombopag
D.3.2	Product code	[ETB115]
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.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Eltrombopag
D.3.9.1	CAS number	496775-62-3
D.3.9.2	Current sponsor code	ETB115
D.3.9.3	Other descriptive name	Eltrombopag Olamine
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	equal
D.3.10.3	Concentration number	25
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.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	REVOLADE
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm Ltd
D.2.1.2	Country which granted the Marketing Authorisation	Ireland
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	Eltrombopag
D.3.2	Product code	[ETB115]
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.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Eltrombopag
D.3.9.1	CAS number	496775-62-3
D.3.9.2	Current sponsor code	ETB115
D.3.9.3	Other descriptive name	Eltrombopag Olamine
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	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.IMP: 3
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
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm Ltd
D.2.1.2	Country which granted the Marketing Authorisation	Ireland
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	Eltrombopag
D.3.2	Product code	[ETB115]
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.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Eltrombopag
D.3.9.1	CAS number	496775-62-3
D.3.9.2	Current sponsor code	ETB115
D.3.9.3	Other descriptive name	Eltrombopag Olamine
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	equal
D.3.10.3	Concentration number	75
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

Immune thrombocytopenia

Trombocitopenia immune

E.1.1.1

Medical condition in easily understood language

Immune thrombocytopenia

Trombocitopenia immune

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	23.0
E.1.2	Level	LLT
E.1.2	Classification code	10050245
E.1.2	Term	Autoimmune thrombocytopenia
E.1.2	System Organ Class	100000004851

E.1.2 Medical condition or disease under investigation

E.1.2	Version	22.1
E.1.2	Level	LLT
E.1.2	Classification code	10036735
E.1.2	Term	Primary thrombocytopenia
E.1.2	System Organ Class	100000004851

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 assess ability of eltrombopag to induce sustained remission by month 12 in ITP subjects who relapsed or failed to respond to first-line steroid treatment

Valutare la capacità di eltrombopag di indurre remissione mantenuta entro il mese 12 nei soggetti
con ITP che manifestano recidiva o insuccesso della risposta al trattamento di prima linea con corticosteroidi.

E.2.2

Secondary objectives of the trial

1. To assess the duration of sustained remission after treatment discontinuation
2. To assess the ability of eltrombopag to induce early response by month 1
3. To assess the ability of eltrombopag to induce a recovery response, in case of loss of response during or after tapering of eltrombopag
4. To quantify the platelet count from baseline to 3, 6, 9, 12 months
5. To assess the ability of eltrombopag to maintain platelet count = 30×109/L within 12 months
6. To evaluate patient-Health Related outcome measures for health-related quality
of life (fatigue level of the subjects through FACIT) & FACT-Th6 and SF-36v2 questionnaires
7. To explore the overall impact of side effects on treatment via the GP5 at baseline and EOS
8. To explore treatment satisfaction with TSQM-9
9. To evaluate the safety and tolerability of eltrombopag

1.Valutare la durata della remissione mantenuta dopo la sospensione del trattamento.
2. Valutare la capacità di eltrombopag di indurre una risposta precoce al mese 1.
3.Valutare la capacità di eltrombopag di indurre una risposta di recupero, in caso di perdita della risposta durante o dopo la riduzione graduale di eltrombopag.
4.Quantificare la conta piastrinica dal basale ai mesi 3, 6, 9, 12.
5.Valutare la capacità di eltrombopag di mantenere la conta piastrinica = 30 x 109/L entro 12 mesi.
6.Valutare le misure dell’outcome del paziente correlato alla salute per la qualità della vita correlata alla salute attraverso l'utilizzo di questionari
7. Valutare l’impatto complessivo degli effetti collaterali sulla percezione del trattamento da parte del soggetto, mediante determinazione del GP5 al basale e alla fine dello studio.
8. Valutare la soddisfazione del trattamento mediante questionario TSQM-9.
9.Valutare la sicurezza d’impiego e la tollerabilità di eltrombopag.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Signed informed consent must be obtained prior to participation in the study
2. Subjects = 18 years old
3. Subjects with a confirmed diagnosis of primary ITP, who are not responsive or in relapse after a first line of steroid therapy ± intravenous immunoglobulin (IVIG) (used as a rescue therapy)
4. Platelet count < 30×109/L and assessed as needing treatment (per physician’s discretion

1. La firma del consenso informato deve essere ottenuta prima della partecipazione allo studio.
2. Pazienti di età > 18 anni.
3. Diagnosi confermata di ITP primaria, non responsiva o in recidiva dopo la terapia di prima linea con corticosteroidi ± IVIG (utilizzate come terapia rescue).
4.Conta piastrinica < 30 x 109/L e valutata in base alla necessità di trattamento (a discrezione del medico).

E.4

Principal exclusion criteria

1. ITP subjects previously treated with any ITP second-line therapies, thrombopoietin receptor (TPO-R) agonists for ITP, except steroids / IVIG
2. Subjects who relapsed more than one year after the end of first-line full course of steroid therapy
3. Subjects with a diagnosis of secondary thrombocytopenia
4. Subjects who have life threatening bleeding complications per investigator discretion
5. Subjects who had a deep vein thrombosis or arterial thrombosis in the 6 months preceding enrollment
6. Serum creatinine = 1.5 mg/dL
7. Total bilirubin > 1.5 × upper limit of normal (ULN)
8. Aspartate transaminase (AST) > 3.0 × ULN
9. Alanine transaminase (ALT) > 3.0 × ULN
10. Subjects who are human immune deficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) positive
11. Subjects with hepatic impairment (Child-Pugh score > 5)
12. Subjects who have active malignancy
13. Subjects with any serious and/or unstable pre-existing medical, psychiatric disorder or other conditions that could interfere with subject’s safety, obtaining informed consent or compliance with the study procedures per investigator discretion
14. History or current diagnosis of cardiac disease indicating significant risk of safety for subjects participating in the study
15. Subjects with known active or uncontrolled infections not responding to appropriate therapy
16. Subjects with evidence of current alcohol/drug abuse
17. Women of child-bearing potential and sexually active males unwilling to use adequate contraception during the study
18. Female subjects who are nursing or pregnant (positive serum or urine B-human chorionic gonadotrophin (B-hCG) pregnancy test) at screening or pre-dose on Day 1

Other protocol-defined inclusion/exclusion criteria may apply

1. Soggetti con ITP precedentemente trattata con qualsiasi terapia di seconda linea per ITP, TPO-RA per ITP, a eccezione di corticosteroidi/IVIG, come descritto nei criteri d’inclusione.
2. Soggetti che hanno manifestato recidiva oltre un anno dopo la fine del ciclo completo della terapia di prima linea con corticosteroidi.
3. Soggetti con diagnosi di trombocitopenia secondaria.
4. Soggetti che non possono partecipare a studi di valutazione/biologici.
5. Soggetti con complicanze emorragiche che costituiscono una minaccia per la sopravvivenza, a discrezione dello sperimentatore.
6. Soggetti che hanno manifestato trombosi venosa profonda o trombosi arteriosa nei 6 mesi precedenti l’arruolamento.
7. Creatininemia > 1,5 mg/dL.
8. Bilirubina totale > 1,5 x limite superiore della norma (ULN).
9. Aspartato aminotransferasi (AST) > 3,0 x ULN.
10. Alanina aminotransferasi (ALT) > 3,0 x ULN.
11. Soggetti positivi a virus dell’immunodeficienza umano (HIV), virus dell’epatite C (HCV) o antigene di superficie del virus dell’epatite B (HBsAg).
12. Soggetti con compromissione epatica (punteggio Child-Pugh > 5).
13. Soggetti che non sono disposti a rispettare l’intervallo di 4 ore tra eltrombopag e altri farmaci (per esempio antiacidi), alimenti ricchi di calcio (per esempio latticini e succhi con integratori a base di calcio) o integratori contenenti cationi polivalenti quali ferro, calcio, alluminio, magnesio, selenio e zinco.
14. Soggetti che non possono sospendere i farmaci che sono noti per causare interazione farmacologica con eltrombopag.
15. Soggetti con neoplasia in fase attiva.
16. Soggetti con qualsiasi patologia medica, psichiatrica preesistente seria e/o instabile o altra condizione che possa interferire con la sicurezza del paziente, con l’ottenimento del consenso informato o la compliance alle procedure dello studio, a discrezione dello sperimentatore.
17. Soggetti con una reazione di ipersensibilità nota immediata o ritardata o idiosincrasia a eltrombopag o a farmaci chimicamente correlati a eltrombopag o agli eccipienti che controindichi la partecipazione allo studio.
18. Diagnosi attuale o pregressa di cardiopatia indicativa di un rischio significativo per la sicurezza dei soggetti partecipanti allo studio quale cardiopatia non controllata o rilevante o compromissione della funzionalità cardiaca comprese una qualsiasi delle
seguenti condizioni:
- QTc corretto > 450 msec (maschi), > 460 msec (femmine) utilizzando la correzione di Fredericia (QTcF) all’ECG di screening.
- Frequenza cardiaca a riposo allo screening (esame obiettivo o elettrocardiogramma a 12 derivazioni – ECG) < 50 o > 90 battiti/minuto.
- Infarto miocardico recente (entro gli ultimi 6 mesi).- - Scompenso cardiaco congestizio non controllato.
- Angina instabile (entro gli ultimi 6 mesi).
19. Soggetti con infezioni note in fase attiva o non controllate che non rispondono alla terapia appropriata.
20. Soggetti con evidente abuso di alcool/droghe attuale.
21. Partecipazione concomitante a uno studio sperimentale entro 30 giorni prima dell’arruolamento o entro 5 emivite del farmaco sperimentale, considerando qualunque sia più lungo.
Nota: l’arruolamento parallelo in un registro di malattia è consentito.
22. Fattori di rischio trombofilici noti. Eccezione: Soggetti nei quali i possibili benefici.

Possono essere applicati altri criteri di inclusione/esclusione definiti dal protocollo.

E.5 End points

E.5.1

Primary end point(s)

Percentage of participants with sustained remission (R) by 12 months

Percentuale di soggetti con remissione mantenuta (R) entro il mese 12

E.5.1.1

Timepoint(s) of evaluation of this end point

12 months

12 mesi

E.5.2

Secondary end point(s)

1a. Median duration of sustained remission (weeks) counted from last
dose of eltrombopag to month 12 for patients with sustained remission
(R)
1b. Estimated median duration of sustained remission (weeks) by month
24 for patients who are in sustained remission (R) at month 12 and
enter 12 months follow-up period using Kaplan-Meier method
1c. Estimated median duration of sustained remission (weeks) by month
24 for all patients using Kaplan-Meier method.
2. Proportion of sustained remission (R) patients at months 15, 18, 21,
and 24.
3. Number (%) of patients with platelet count = 50×109/L at least once
by month 1 (first month) without bleeding and no rescue therapy
4. Number (%) of patients with at least one platelet count = 30×109/L
after eltrombopag is re-introduced, in case of loss of response (<
30×109/L and/or bleeding event) without bleeding and no rescue
therapy by month 12 and 24
5. Absolute and relative change in platelet count from baseline to 3, 6,
9,12, 15, 18, 21, and 24 months and end of treatment
6. Number (%) of patients who maintain a platelet count = 30×109/L
from the first time of reaching that level to month 3, 6, 9, 12, 15, 18, 21,
24, and end of treatment without bleeding and no rescue therapy
7. The analysis of HRQoL parameters; fatigue level of the patients
through FACIT & FACT-Th6, SF-36v2 questionnaires. Change in scores
from baseline to month 3, 6, 9, 12, 15, 18, 21, 24, and end of treatment
8. Overall change from baseline to month 12 and end of treatment will
be assessed
9. Overall change from baseline to month 12 and end of treatment will
be assessed
10. Number (%) and severity of patients with AEs, serious AEs (SAEs),
AEs leading to discontinuation, AEs leading to dose adjustments, AEs of
special interest. Change from baseline in vital signs and clinical
laboratory tests

E.5.2.1

Timepoint(s) of evaluation of this end point

1a. 12 months
1b. 24 months
1c. 24 months
2. at months 15, 18, 21 and 24
3. 1 month
4. 12 and 24 months
5. from baseline to 3, 6, 9,12, 15, 18, 21, and 24 months and end of
treatment
6.from the first time of reaching required platelet level to month 3, 6, 9,
12, 15, 18, 21, 24, and end of treatment
7. from baseline to month 3, 6, 9, 12, 15, 18, 21, 24, and end of
treatment
8. from baseline to month 12 and end of treatment
9. from baseline to month 12 and end of treatment
10. from baseline

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

Yes

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

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

Yes

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

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

Australia

Brazil

Chile

France

Germany

Greece

Italy

Japan

Mexico

Norway

Oman

Russian Federation

Saudi Arabia

Spain

Switzerland

Turkey

United Kingdom

United States

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

Study completion is defined as when the last subject finishes their EOS visit, and any repeat assessments associated with this visit have been documented and followed-up appropriately by the Investigator, or in the event of an early study termination decision, the date of that decision

Il completamento dello studio è definito con la visita di fine studio da parte dell'ultimo paziente e qualsiasi valutazione associata a questa visita è stata documentata e seguita in modo appropriato dallo sperimentatore o, nel caso di decisione di chiusura anticipata dello studio, la data di tale decisione.

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:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

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

101

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)

Continuing care to study participants should be provided by the investigator and/or referring physician. The treating physician can request access to eltrombopag for subjects that are continuing to benefit from treatment. Access will be dependent on the local regulatory authority approval of the product and current reimbursement options.

La continuità delle cure ai pazienti partecipanti allo studio dovrebbe essere fornita dallo sprimentatore e/o dal medico di riferimento. Il medico curante può richiedere l'accesso all' eltrombopag per i soggetti che continuano a beneficiare del trattamento. L'accesso al prodotto è subordinato all' approvazione dell' Autorità Regolatorioa locale e dipende dalle opzioni di rimborso attuali.

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

2018-09-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-09-12

P. End of Trial
P.	End of Trial Status	Completed

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