Clinical Trials Register

Summary
EudraCT Number:	2018-003969-33
Sponsor's Protocol Code Number:	IPH4102-201
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:	2021-01-29
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

2018-003969-33

A.3

Full title of the trial

TELLOMAK: T-cell Lymphoma anti-KIR3DL2 therapy. An open label, multicohort, multi-center phase II study evaluating the efficacy and safety of IPH4102 alone or in combination with chemotherapy in patients with Advanced T-cell lymphoma.

TELLOMAK: T-cell Lymphoma anti-KIR3DL2 therapy (Terapia anti-KIR3DL2 per il linfoma a cellule T) Studio di fase II in aperto, multicoorte, multicentrico per la valutazione dell’efficacia e della sicurezza di IPH4102 in monoterapia o in combinazione con chemioterapia in pazienti con linfoma a cellule T in stadio avanzato

A.3.1

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

Study of IPH4102 alone or in combination with chemotherapy in patients with Advanced T-cell Lymphoma.

Studio di IPH4102 in monoterapia o in combinazione con chemioterapia in pazienti con linfoma a cellule T in stadio avanzato

A.3.2

Name or abbreviated title of the trial where available

TELLOMAK

A.4.1

Sponsor's protocol code number

IPH4102-201

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	INNATE PHARMA
B.1.3.4	Country	France
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	INNATE PHARMA
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	INNATE PHARMA
B.5.2	Functional name of contact point	Medical Director
B.5.3	Address:
B.5.3.1	Street Address	117, avenue de Luminy - BP30191
B.5.3.2	Town/ city	Marseille Cedex 09
B.5.3.3	Post code	13276
B.5.3.4	Country	France
B.5.4	Telephone number	00330430303030
B.5.5	Fax number	00330430303010
B.5.6	E-mail	info@innate-pharma.fr

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	No
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/3/14/1322
D.3 Description of the IMP
D.3.1	Product name	IPH4102
D.3.2	Product code	[na]
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.3.9.1	CAS number	2187368-16-5
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB176312
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	15
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	Yes
D.3.11.13.1	Other medicinal product type	Humanised IgG1 monoclonal antibody
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	Oxaliplatin 5 mg/ml concentrate for solution for infusion
D.2.1.1.2	Name of the Marketing Authorisation holder	Fresenius Kabi Oncology Plc
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Oxaliplatin 5 mg/ml concentrate for solution for infusion
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Concentrate for 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	OXALIPLATINO
D.3.9.1	CAS number	61825-94-3
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB09490MIG
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	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.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Gemcitabine 38 mg/ml concentrate for solution for infusion
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Concentrate for 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	GEMCITABINA
D.3.9.1	CAS number	95058-81-4
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB07892MIG
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	38
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

Advanced T-Cell Lymphomas (TCL), i.e. Cutaneous T Cell Lymphomas (CTCL) and Peripheral T Cell Lympomas (PTCL). CTCL subtypes under investigation: relapsed/refractory Sézary Syndrome (SS), stage IB to IV Mycosis Fungoides (MF). Relapsed/refractory PTCL subtypes under investigation: PTCL-Not Otherwise Specified (PTCL-NOS), AngioImmunoblastic T-cell Lymphoma (AITL), Anaplastic Large Cell Lymphoma (ALCL).

Linfomi a cellule T avanzate (TCL), cioè linfomi a cellule T cutanee (CTCL) e linfomi a cellule T periferici (PTCL). Sottotipi CTCL sotto indagine: Sindrome di Sézary recidivante / refrattaria (SS), stadio IB-IV Mycosis Fungoides (MF). Sottotipi PTCL recidivanti / refrattari sotto esame: PTCL-Non altrimenti specificato (PTCL-NOS), linfoma angiolitico a cellule T (AITL), linfoma anaplastico a grandi cellule (ALCL).

E.1.1.1

Medical condition in easily understood language

Rare types of cancers of the immune system

Tipologie rare di cancro del sistema immunitario

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	HLT
E.1.2	Classification code	10034622
E.1.2	Term	Peripheral T-cell lymphomas NEC
E.1.2	System Organ Class	100000004851

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	HLT
E.1.2	Classification code	10002450
E.1.2	Term	Angioimmunoblastic T-cell lymphomas
E.1.2	System Organ Class	100000004851

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	HLT
E.1.2	Classification code	10002235
E.1.2	Term	Anaplastic large cell lymphomas T- and null-cell types
E.1.2	System Organ Class	100000004851

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	HLT
E.1.2	Classification code	10028484
E.1.2	Term	Mycoses fungoides
E.1.2	System Organ Class	100000004851

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the clinical activity of IPH4102 alone (Cohorts 1-3) or in combination with GEMOX chemotherapy (Cohorts 4 and 5) in patients with advanced T-cell lymphoma.

Valutare il tasso di risposta obiettiva (objective response rate, ORR) di IPH4102 in monoterapia (nelle Coorti 1-3) o in combinazione con la chemioterapia a base di GEMOX (Coorti 4 e 5) in pazienti con linfoma a cellule T in stadio avanzato.

E.2.2

Secondary objectives of the trial

-To characterize the safety and tolerability of IPH4102 alone (Cohorts 1- 3) or in combination with GEMOX chemotherapy (Cohorts 4 and 5);
-To further characterize the clinical activity of IPH4102 alone or in combination with GEMOX chemotherapy;
-To evaluate the pharmacokinetics (PK) and immunogenicity of IPH4102 alone or in combination with GEMOX chemotherapy.

- Caratterizzare la sicurezza e la tollerabilità di IPH4102 in monoterapia (Coorti 1-3) o in combinazione con la chemioterapia a base di GEMOX (Coorti 4 e 5).
- Caratterizzare ulteriormente l'attività clinica di IPH4102 in monoterapia o in combinazione con la chemioterapia GEMOX;
- Valutare la farmacocinetica (PK) e l'immunogenicità dell'IPH4102 in monoterapia o in combinazione con la chemioterapia GEMOX.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Cohort 1:
1.Patients with relapsed/refractory SS who have received at least 2 prior systemic therapies;
2.Prior treatment with mogamulizumab;
3.Patients should have blood stage B2 at screening based on central evaluation by flow cytometry;
4.Feasibility of obtaining at least 1 skin biopsy at screening.
Cohorts 2 and 3:
5.Patients with stage IB to IV MF;
6.KIR3DL2 expression (Cohort 2) or non-expression (Cohort 3) in at least 1 skin lesion;
7.Patients should have received at least 2 prior systemic therapies that may include biological agents;
8.Feasibility of obtaining at least 1 skin biopsy at screening;
9.Adequate baseline laboratory data: Hematology: CD4+ T-cells = 200/µL.
Cohorts 4 and 5:
10. Patients with relapsed/refractory PTCL of the following subtypes: PTCL-NOS, angioimmunoblastic T-cell lymphoma (AITL), or anaplastic large cell lymphoma (ALCL);
11.KIR3DL2 expression (Cohort 4) or non-expression (Cohort 5) based on 1 involved lymph node;
12.Patients should have received at least 1 prior systemic therapy including an anthracycline-based chemotherapy. Patients who are not eligible for treatment with anthracycline based therapy are eligible for inclusion provided that they were treated with at least one prior systemic therapy;
13.Presence of at least 1 target lesion on PET/CT scan at screening;
14.Feasibility of obtaining 1 lymph node biopsy at screening.
All cohorts:
15.Male or Female, at least 18 years of age;
16.ECOG performance status =2;
17.The patient must have a minimum wash-out period of 4 weeks between the last dose of prior systemic therapy (8 weeks for biological agents) and the first dose of IPH4102
18.Patients should have recovered from all adverse events related to prior therapy to = grade 1;
19.Adequate baseline laboratory data
20.Women of childbearing potential (WOCBP) must have a negative serum beta-HCG pregnancy test result within seven days from start of treatment;
21.Women of childbearing potential and all men (and their female partners of childbearing potential) who are sexually active must agree to use adequate method of contraception at study entry, during treatment and for at least 9 months (270 days) following the last dose of study drug.

Coorte 1:
1. Pazienti con SS recidivante/refrattaria che hanno ricevuto almeno due terapie sistemiche precedenti.
2. Precedente trattamento con mogamulizumab.
3. Stadio ematico B2 allo screening in base alla valutazione centrale con citometria a flusso.
4. Fattibilità della raccolta di almeno una biopsia cutanea allo screening.
Coorti 2 e 3:
5. MF in stadio IB, IIA, IIB, III, IV.
6. Espressione (Coorte 2) o assenza di espressione (Coorte 3) di KIR3DL2 in almeno una lesione cutanea in base alla valutazione centrale con IHC.
7. Precedente trattamento con almeno due terapie sistemiche, che possono includere agenti biologici.
8. Fattibilità della raccolta di almeno una biopsia cutanea allo screening.
9. Dati di laboratorio adeguati al basale: Ematologia: cellule T CD4+ =200/µl.
Coorti 4 e 5:
10. PTCL recidivante/refrattario dei seguenti sottotipi: PTCL-NAS, linfoma angioimmunoblastico a cellule T (angioimmunoblastic T-cell lymphoma, AITL) o linfoma anaplastico a grandi cellule (anaplastic large cell lymphoma, ALCL).
11. Espressione (Coorte 4) o assenza di espressione (Coorte 5) di KIR3DL2 su un linfonodo coinvolto in base alla valutazione centrale con IHC.
12. Precedente trattamento con almeno una terapia sistemica, inclusa una chemioterapia a base di antracicline. I pazienti non eleggibili al trattamento con terapia a base di antracicline possono essere inclusi a condizione che abbiano ricevuto almeno una terapia sistemica precedente.
13. Presenza di almeno una lesione target alla scansione PET/TC eseguita allo screening.
14. Fattibilità della raccolta di una biopsia linfonodale allo screening.
Tutte le coorti:
15. Sesso maschile o femminile, età pari almeno a 18 anni.
16. Stato di validità secondo ECOG (Eastern Cooperative Oncology Group [Gruppo cooperativo orientale di oncologia]) =2.
17. Periodo di washout minimo di 4 settimane tra l’ultima dose della precedente terapia sistemica (8 settimane per gli agenti biologici) e la prima dose di IPH4102.
18. Recupero fino a un grado =1 da tutti gli eventi avversi correlati alla precedente terapia.
19. Dati di laboratorio adeguati al basale:
20. Le donne in età fertile (women of childbearing potential, WOCBP) devono risultare negative a un test di gravidanza mediante dosaggio nel siero della beta-HCG (human chorionic gonadotropin [gonadotropina corionica umana]) entro sette giorni dall’inizio del trattamento.
21. Le donne in età fertile e tutti gli uomini (nonché le rispettive partner in età fertile) che siano sessualmente attivi devono accettare di utilizzare un metodo contraccettivo adeguato al momento dell’ingresso nello studio, durante il trattamento e per almeno 9 mesi (270 giorni) dopo l’ultima dose di farmaco dello studio. I metodi adeguati comprendono contraccettivi orali o metodi a doppia barriera (preservativo più spermicida o diaframma più spermicida).

E.4

Principal exclusion criteria

Cohorts 1 to 3:
1.Patients with evidence of large cell transformation (LCT) based on central histologic evaluation.
Cohorts 4 and 5:
2.Prior administration of gemcitabine and/or oxaliplatin;
3.Presence of grade 2 neurotoxicity or higher.
All Cohorts:
4.Known central nervous system (CNS) lymphoma;
5.Prior administration of IPH4102;
6.Concomitant administration of radiotherapy or systemic anti-cancer therapy including but not restricted to: chemotherapy, biological agents or immunotherapy;
7.Autologous stem cell transplantation less than 3 months prior to enrollment;
8.Prior allogenic transplantation;
9.Concomitant corticosteroid use, systemic or topical. However, stable dosage of topical steroids (maximum strength Class III according to World Health Organization (WHO) Classification of Topical Corticosteroids) and/or systemic steroids (=10 mg prednisone
equivalent/day) are allowed, if patient has been on a stable dose for at least 4 weeks prior to treatment start;
10.Patients who have undergone major surgery = 4 weeks prior to study entry;
11.Patients with known NCI CTCAE grade 3 or higher active systemic or cutaneous viral, bacterial, or fungal infection;
12.Patients who have active Hepatitis B or C virus infection;
13.Patients who are known to be HIV-positive;
14.Patients with a history of other malignancies during the past 5 years apart from the disease subject of this study. The following are exempt from the five-year limit: non-melanoma skin cancer, lymphomatoid papulosis, resected thyroid cancer, biopsy-proven cervical intraepithelial neoplasia or cervical carcinoma in situ;
15.Pregnant or breastfeeding women;
16.Patients with congestive heart failure, Class III or IV, by New York Heart Association (NYHA) criteria;
17.Patients with known active autoimmune disease;
18.Patients with any serious underlying medical condition that would impair their ability to receive or tolerate the planned treatment and/or comply with study protocol;
19.Patients with dementia or altered mental status that would preclude understanding and rendering of informed consent document.

Coorti 1-3:
1. Pazienti con evidenza di trasformazione a grandi cellule (large cell transformation, LCT) in base alla valutazione istologica centrale.
Coorti 4 e 5:
2. Precedente somministrazione di gemcitabina e/o oxaliplatino.
3. Presenza di neurotossicità di grado 2 o superiore.
Tutte le coorti:
4. Linfoma noto del sistema nervoso centrale (SNC).
5. Precedente somministrazione di IPH4102.
6. Somministrazione concomitante di radioterapia o terapia antitumorale sistemica, tra cui, in modo non limitativo: chemioterapia, agenti biologici o immunoterapia.
7. Trapianto di cellule staminali autologhe meno di 3 mesi prima dell’arruolamento.
8. Pregresso trapianto allogenico.
9. Uso concomitante di corticosteroidi, per via sistemica o topica. Tuttavia, è ammesso l’uso d un dosaggio stabile di steroidi topici (potenza massima di classe III secondo la Classificazione dei corticosteroidi topici dell’Organizzazione mondiale della sanità [OMS]) e/o di steroidi sistemici (=10 mg/die di un equivalente del prednisone), laddove la dose sia rimasta stabile per almeno 4 settimane prima dell’inizio del trattamento.
10. Intervento chirurgico maggiore =4 settimane prima dell’ingresso nello studio.
11. Nota infezione virale, batterica o fungina in fase attiva, con coinvolgimento sistemico o cutaneo, di grado NCI CTCAE pari o superiore a 3.
12. Infezione da virus dell’epatite B o C in fase attiva.
13. Positività per HIV (human immunodeficiency virus [virus dell’immunodeficienza umana]) nota.
14. Anamnesi di altri tumori maligni durante gli ultimi cinque anni oltre alla malattia oggetto di questo studio. Sono esclusi dal limite dei cinque anni i seguenti casi: tumore cutaneo non melanoma, papulosi linfomatoide, tumore tiroideo sottoposto a resezione, neoplasia cervicale intraepiteliale o carcinoma cervicale in situ confermati da biopsia.
15. Donne in stato di gravidanza o allattamento.
16. Insufficienza cardiaca congestizia di classe III o IV secondo i criteri della New York Heart Association (NYHA) [Associazione dei cardiologi di New York].
17. Malattia autoimmune nota, in fase attiva.
18. Qualsiasi grave condizione medica di base che potrebbe compromettere la capacità del paziente di ricevere o tollerare il trattamento previsto e/o di attenersi al protocollo dello studio.
19. Demenza o alterazione dello stato mentale che potrebbe impedire al paziente di comprendere e restituire il modulo di consenso informato.

E.5 End points

E.5.1

Primary end point(s)

Objective Response Rate (ORR)

Tasso di risposta obiettiva (ORR)

E.5.1.1

Timepoint(s) of evaluation of this end point

ORR: until End of treatment (EOT) and during Follow-Up (FU) if applicable as per Protocol.

ORR: fino alla fine del trattamento (EOT) e durante il follow-up (FU) se applicabile come da protocollo.

E.5.2

Secondary end point(s)

Quality of life (QoL) (Cohorts 1-3), ORR using blinded central review (Cohort 1), duration of response (DOR), ORR lasting at least 4 months (ORR4) (Cohorts 1-3), progression free survival (PFS), overall survival (OS), minimal residual disease (MRD).

Qualità della vita (QoL) (Coorti 1-3), ORR mediante revisione centrale in cieco (Coorte 1), durata della risposta (DOR), ORR della durata di almeno 4 mesi (ORR4) (Coorti 1-3), sopravvivenza libera da progressione ( PFS), sopravvivenza globale (OS), malattia residua minima (MRD).

E.5.2.1

Timepoint(s) of evaluation of this end point

Until EOT and during FU if applicable.

Fino all'EOT e durante FU, se applicabile.

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

Yes

E.6.7

Pharmacodynamic

Yes

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

Yes

E.8.1.7.1

Other trial design description

In aperto

Open

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

United States

France

Germany

Italy

United Kingdom

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

Last visit of the last subject

LVLP

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:

125

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

125

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

120

F.4.2.2

In the whole clinical trial

250

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)

SOC

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

2019-08-01

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-07-17

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

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Orphan Designation Number:
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