Clinical Trials Register

Summary
EudraCT Number:	2013-005157-75
Sponsor's Protocol Code Number:	EMN12/HO129PCL
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:	2021-05-27
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

2013-005157-75

A.3

Full title of the trial

Carfilzomib and lenalidomide-based treatment for younger and elderly newly diagnosed primary plasma cell leukemia patients

TRATTAMENTO DI CARFILZOMIB E LENALIDOMIDE PER I PAZIENTI GIOVANI E PER I PAZIENTI ANZIANI DI NUOVA DIAGNOSI AFFETTI DA LEUCEMIA PLASMACELLULARE PRIMARIA (pPCL)

A.3.1

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

Carfilzomib and lenalidomide-based treatment for younger and elderly newly diagnosed primary plasma cell leukemia patients

TRATTAMENTO CON CARFILZOMIB E LENALIDOMIDE PER I PAZIENTI GIOVANI E ANZIANI AFFETTI DA LEUCEMIA PLASMACELLULARE PRIMARIA ALLA DIAGNOSI

A.3.2

Name or abbreviated title of the trial where available

EMN12/HO129 PCL

A.4.1

Sponsor's protocol code number

EMN12/HO129PCL

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	HOVON
B.1.3.4	Country	Netherlands
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	Celgene International
B.4.2	Country	Switzerland
B.4.1	Name of organisation providing support	Onyx Pharmaceuticals
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	HOVON
B.5.2	Functional name of contact point	HOVON Data Center
B.5.3	Address:
B.5.3.1	Street Address	P.O. Box 2040
B.5.3.2	Town/ city	Rotterdam
B.5.3.3	Post code	3000 CA
B.5.3.4	Country	Netherlands
B.5.4	Telephone number	+310107041560
B.5.5	Fax number	+310107041028
B.5.6	E-mail	hdc@erasmusmc.nl

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	KYPROLIS
D.2.1.1.2	Name of the Marketing Authorisation holder	AMGEN EUROPE B.V.
D.2.1.2	Country which granted the Marketing Authorisation	Netherlands
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.3.4	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	CARFILZOMIB
D.3.9.1	CAS number	868540-17-4
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB32911
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
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	REVLIMID - 5 MG CAPSULA RIGIDA - USO ORALE BLISTER (PCTFE/PVC/ALU) 21 CASPULE
D.2.1.1.2	Name of the Marketing Authorisation holder	CELGENE EUROPE 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	Yes
D.2.5.1	Orphan drug designation number	EU/3/04/192 (MDS)
D.3 Description of the IMP
D.3.1	Product name	REVLIMID
D.3.4	Pharmaceutical form	Capsule, hard
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	00309600
D.3.9.1	CAS number	191732-72-6
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB25389
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	5
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	REVLIMID - 10 MG CAPSULA RIGIDA - USO ORALE BLISTER (PCTFE/PVC/ALU) 21 CAPSULE
D.2.1.1.2	Name of the Marketing Authorisation holder	CELGENE EUROPE 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	Yes
D.2.5.1	Orphan drug designation number	EU/3/04/192 (MDS)
D.3 Description of the IMP
D.3.1	Product name	Revlimid
D.3.4	Pharmaceutical form	Capsule, hard
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	00309600
D.3.9.1	CAS number	191732-72-6
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB25389
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	10
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: 4
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	REVLIMID - 15 MG CAPSULA RIGIDA - USO ORALE BLISTER (PCTFE/PVC/ALU) 21 CAPSULE
D.2.1.1.2	Name of the Marketing Authorisation holder	CELGENE EUROPE 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	Yes
D.2.5.1	Orphan drug designation number	EU/3/04/192 (MDS)
D.3 Description of the IMP
D.3.1	Product name	Revlimid
D.3.4	Pharmaceutical form	Capsule, hard
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	00309600
D.3.9.1	CAS number	191732-72-6
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB25389
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	15
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: 5
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	REVLIMID - 25 MG CAPSULA RIGIDA - USO ORALE BLISTER (PCTFE/PVC/ALU) 21 CAPSULE
D.2.1.1.2	Name of the Marketing Authorisation holder	CELGENE EUROPE 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	Yes
D.2.5.1	Orphan drug designation number	EU/3/04/192 (MDS)
D.3 Description of the IMP
D.3.1	Product name	Revlimid
D.3.4	Pharmaceutical form	Capsule, hard
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	00309600
D.3.9.1	CAS number	191732-72-6
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB25389
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.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

Primary plasma cell leukemia

Leucemia Plasma Cellulare

E.1.1.1

Medical condition in easily understood language

Primary plasma cell leukemia

Leucemia Plasma Cellulare

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	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10035223
E.1.2	Term	Plasma cell leukemia
E.1.2	System Organ Class	100000004864

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 progression-free survival in adult pPCL patients by incorporation of carfilzomib and lenalidomide in induction, consolidation, and maintenance therapy

Valutare la sopravvivenza libera da progressione nei pazienti adulti con PPCL trattati con carfilzomib e lenalidomide durante il trattamento di induzione, di consolidamento e di mantenimento.

E.2.2

Secondary objectives of the trial

- To assess overall response rate and (s)CR + VGPR ((stringent) complete and very good partial response) rate after induction therapy, after HDM, after CRd consolidation, after RIC allo-SCT or a second HDM, and during maintenance.
- To evaluate overall survival.
- To assess safety and toxicity
- To assess the prognostic value of risk factors at diagnosis, including
¿2-microglobulin, LDH, FISH abnormalities del1p, ampli 1q, t(4;14), t(14;16), t(11;14), ampli 9, del13q/13-, del17p as analyzed in purified bone marrow plasma cells with respect to progression-free survival
- To analyze the prognostic value of myeloma gene expression profiles
- To analyze the prognostic value of minimal-residual disease negativity
- To assess the prognostic value of mutations as determined by sequencing
- To analyze the prognostic value of minimal-residual disease negativity
- To establish the frequency of second primary malignancies (SPM)

- Valutare la percentuale di risposta globale e (s)CR + VGPR (risposta completa (stringente) e risposta parziale molto buona) dopo la terapia di induzione, dopo HDM, dopo il consolidamento con CRd, dopo RIC allo-SCT o un secondo HDM, e durante il mantenimento.
- Valutare la sopravvivenza globale
- Valutare la sicurezza e le tossicit¿
- Valutare il valore prognostico dei fattori di rischio alla diagnosi, inclusi ¿2-microglobulina, LDH, le anormalit¿ del1p, ampli 1q, t(4;14), t(14;16), t(11;14), ampli 9, del13q/13-, del17p tramite FISH, analizzando le plasmacellule del midollo osseo purificate, in relazione alla sopravvivenza libera da progressione
- Analizzare il valore prognostico dei profili d¿espressione genetica del mieloma
- Analizzare il valore prognostico della negativit¿ della malattia minima residua
- Analizzare il valore prognostico delle mutazioni come determinato dal sequenziamento
- Stabilire la frequenza dei secondi tumori primari (SPM)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Patients with diagnosis of symptomatic pPCL (see appendix A)
- Measurable disease as defined by the presence of M-protein in serum or urine (serum M-protein > 10 g/l or urine M-protein > 200 mg/24 hours or abnormal FLC ratio with involved free light chain (FLC) > 100 mg/l) or proven plasmacytoma by biopsy)
- Age =18 years
- WHO-performance status 0-3 (but WHO=3 is allowed only when caused by pPCL and not by co-morbid conditions)
- Written informed consent
- Patient capable of giving informed consent (patient is legally, physically and mentally capable of giving consent)
- All men and women of childbearing potential should use adequate contraception during the study. Sperm could be frozen from men with child wish before start of treatment
- Negative pregnancy test at entry (if applicable)
- Patient is willing and able to adhere to the requirements of the lenalidomide Pregnancy Prevention Program (PPP)

- Pazienti con diagnosi di PPCL sintomatica (vedi appendice A)
- Malattia misurabile definita dalla presenza della proteina M nel siero o nelle urine (siero proteina M> 10 g/l o nelle urine proteina M> 200 mg/24 o rapporto anormale delle catene libere leggere con catene libere leggere (FLC)> 100 mg/l) o accertato plasmacitoma da biopsia
- Età =18 anni
- WHO performance status 0-3 (WHO = 3 è consentito solo quando è causato da PPCL e non da comorbidità concomitanti)
- Consenso informato scritto
- Paziente in grado di dare un consenso informato (paziente è legalmente, fisicamente e mentalmente in grado di dare il consenso)
- Tutti gli uomini e le donne in età fertile devono utilizzare un adeguato metodo contraccettivo durante lo studio. Lo sperma potrebbe essere congelato per gli uomini che hanno desiderio di avere un bambino, prima dell'inizio del trattamento
- Test di gravidanza negativo all'inizio dello studio (se applicabile)
- Il paziente deve essere disposto e in grado di rispettare i requisiti del Programma di Prevenzione della Gravidanza per la lenalidomide (PPP)

E.4

Principal exclusion criteria

- Any current CNS involvement with disease refractory to intrathecal chemotherapy.
- Female patients who are pregnant or breast feeding.
- HIV positive patients
- Active malignancy other than pPCL requiring treatment, or a malignancy that has been treated with chemotherapy currently affecting bone marrow capacity
- Patients with active, uncontrolled infections
- Severe neurological or psychiatric disease
- Severe cardiac dysfunction (NYHA classification II-IV, see appendix E)
- Severe pulmonary dysfunction
- Significant hepatic dysfunction (serum bilirubin or transaminases = 3.0
times normal level), unless related to pPCL
- Patients with GFR < 15 ml/min
- Known history of allergy to Capsidol (a cyclodextrin derivative used to solubilize carfilzomib)
- Previous chemotherapy or radiotherapy except local radiotherapy in case of local myeloma progression or corticosteroids maximum 7 days
for symptom control or stabilization(this includes dexamethasone 40 mg daily) or inthrathecal chemotherapy in case of CNS involvement
- Systemic AL amyloidosis
- Any psychological, familial, sociological and geographical condition potentially hampering compliance with the study protocol and follow-up schedule

- Qualsiasi attuale coinvolgimento del sistema nervoso centrale con malattia refrattaria alla chemioterapia intratecale.
- Donne in gravidanza o in allattamento.
- Pazienti HIV positivi
- Tumore maligno attivo diverso da PPCL che richiede trattamento, o un tumore maligno che è stato trattato con chemioterapia che colpisce attualmente la capacità del midollo osseo
- Pazienti con infezioni non controllate, attive
- Malattia neurologica o psichiatrica grave
- Grave disfunzione cardiaca (NYHA classificazione II-IV, vedi appendice E)
- Disfunzione polmonare grave
- Disfunzione epatica significativa (bilirubina sierica o transaminasi = 3.0 volte del livello normale), a meno che non sia correlata a PPCL
- Pazienti con GFR <15 ml / min
- Storia nota di allergia a Capsidol (un derivato ciclodestrina usato per solubilizzare carfilzomib)
- Precedente chemioterapia o radioterapia, ad eccezione della radioterapia locale in caso di progressione di mieloma locae o corticosteroidi massimo 7 giorni per il controllo dei sintomi o la stabilizzazione (questo include desametasone 40 mg al giorno) o chemioterapia intratecale in caso di coinvolgimento del sistema nervoso centrale
- Amiloidosi AL Sistemica
- Qualsiasi condizione psicologica, familiare, sociologica e geografica che potrebbe potenzialmente ostacolare il rispetto del protocollo di studio e la pianificazione di follow-up

E.5 End points

E.5.1

Primary end point(s)

Progression-free survival (PFS, i.e. time from registration until progression or death, whichever comes first).

La sopravvivenza libera da progressione (PFS, cioè il tempo dalla registrazione fino alla progressione o morte, qualunque sia l'evento che si verifica per primo).

E.5.1.1

Timepoint(s) of evaluation of this end point

At the end of the trial. After last patient has completed maintenance treatment.

Alla fine dello studio. Dopo che l'ultimo paziente ha completato il trattamento di mantenimento.

E.5.2

Secondary end point(s)

- Overall response rate (at least PR) after the different phases of treatment
- (s)CR + VGPR ((stringent) complete and very good partial response) after the different phases of treatment
- Overall survival, defined as time from registration until death from any cause. Patients still alive at the date of last contact, will be censored
- Toxicity and tolerability of the different phases of treatment
- Explore the value of prognostic factors including including FISH abnormalities, ¿2-microgloublin, LDH, MRD-negativity, pPCL gene expression profiles and sequencing results on the overall response, overall survival and progression¿free survival
- Frequency of second primary malignancies

- La percentuale di risposta (almeno PR) dopo le varie fasi di trattamento
- (s)CR + VGPR ((severe) risposta completa e risposta parziale molto buona)
- Sopravvivenza globale, definita come il tempo dalla registrazione fino alla morte per qualsiasi causa. I pazienti ancora in vita alla data dell'ultimo contatto, saranno censurati
- Tossicit¿ e tollerabilit¿ delle varie fasi di trattamento
- Valutare il ruolo dei fattori prognostici, tra cui le anomalie FISH, ¿2-microglobulina, LDH, negativit¿ MRD, profili di espressione genica di PPCL e risultati sequenziamento nella risposta complessiva, la sopravvivenza globale e la sopravvivenza libera da progressione.
- Frequenza di secondi tumori primari

E.5.2.1

Timepoint(s) of evaluation of this end point

At the end of the trial. After last patient has completed maintenance treatment.

Alla fine dello studio. Dopo che l'ultimo paziente ha completato il trattamento di mantenimento.

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

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

stratificazione in base all'et¿ del paziente

stratification based on age of patient

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.8.3

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

Belgium

Denmark

Germany

Italy

Netherlands

Norway

Sweden

E.8.7

Trial has a data monitoring committee

Yes

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:

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

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.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

116

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)

patients will be followed by their physician to the end of the study.

I pazienti verranno seguiti dal proprio medico al termine della partecipazione allo studio.

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

2016-05-10

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-04-12

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

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