Clinical Trials Register

Summary
EudraCT Number:	2018-003210-40
Sponsor's Protocol Code Number:	IMGN632-0801
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:	2021-01-20
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

2018-003210-40

A.3

Full title of the trial

A Phase 1/2, Multi-center, Open-label Study of IMGN632 Monotherapy Administered Intravenously in Patients with CD123- positive Acute Myeloid Leukemia and Other CD123 positive Hematologic Malignancies

Studio in aperto di fase 1/2, multicentrico, su IMGN632 in monoterapia somministrato per via endovenosa in pazienti adulti con leucemia mieloide acuta CD123-positiva o altre malignità ematologiche CD123-positive

A.3.1

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

A study to test IMGN632 in adults with CD123-positive Acute Myeloid Leukemia and other CD123 positive Hematologic cancers

Studio per testare IMGN632 in adulti con leucemia mieloide acuta CD123-positiva o altre malignità ematologiche CD123-positive

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

IMGN632-0801

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03386513

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	IMMUNOGEN, INC.
B.1.3.4	Country	United States
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	ImmunoGen, Inc.
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	ImmunoGen, Inc.
B.5.2	Functional name of contact point	Kenneth Dhimitri
B.5.3	Address:
B.5.3.1	Street Address	830 Winter Street
B.5.3.2	Town/ city	Waltham
B.5.3.3	Post code	02451
B.5.3.4	Country	United States
B.5.4	Telephone number	000000
B.5.5	Fax number	0017812075341
B.5.6	E-mail	IMGN0801@immunogen.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	No
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	IMGN632
D.3.2	Product code	[IMGN632]
D.3.4	Pharmaceutical form	Powder 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	IMGN632
D.3.9.2	Current sponsor code	IMGN632
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	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	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

CD123 positive Acute Myeloid Leukemia and other CD123 positive hematologic malignancies

Leucemia mieloide acuta positiva CD123 e altre neoplasie ematologiche CD123 positive

E.1.1.1

Medical condition in easily understood language

Acute Myeloid Leukemia and other blood cancers

Leucemia mieloide acuta e altri tumori del sangue

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	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10000886
E.1.2	Term	Acute myeloid leukemia
E.1.2	System Organ Class	100000004864

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

Escalation Phase: To determine the maximum tolerability of IMGN632 on
the selected dosing schedule(s)
BPDCN: To assess the response rate in BPDCN patients

Fase di incremento della dose: determinare la dose massima tollerata di IMGN632 sullo/sugli schema/i di somministrazione selezionato/i
Pazienti con BPDCN: valutare il tasso di risposta nei pazienti con BPDCN

E.2.2

Secondary objectives of the trial

1. ALL Patients:
• To characterize the safety profile and tolerability of IMGN632 when given as a single agent across dose levels and schedules
• To characterize the PK of IMGN632, total antibody, and free payload (FGN849); IMGN632 metabolites may be evaluated
• To evaluate the potential immunogenicity of IMGN632
• To assess the preliminary anti-leukemia activity in AML and ALL patients (tumor-specific expansion cohorts) and BPDCN patients
2. BPDCN patients: to assess the durability of benefit in BPDCN patients

1. Pazienti con ALL:
• Caratterizzare il profilo di sicurezza e la tollerabilità di IMGN632 quando somministrato come agente singolo attraverso i livelli di dosaggio e i programmi
• Caratterizzare il PK di IMGN632, anticorpi totali e carico utile libero (FGN849); I metaboliti di IMGN632 possono essere valutati
• Valutare la potenziale immunogenicità di IMGN632
• Valutare l'attività anti-leucemia preliminare in pazienti con AML e ALL (coorti di espansione specifiche del tumore) e pazienti con BPDCN
2. Pazienti con BPDCN: valutare la durata del beneficio nei pazienti con BPDCN

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Disease Characteristics :
a.Confirmation of CD123 positivity by flow cytometry or IHC. Patients who received prior CD123-targeting agents will be allowed as long as the blasts still have detectable CD123 expression
b. Dose Escalation – Relapsed or refractory AML (excluding acute promyelocytic leukemia) or BPDCN, based on World Health Organization Classification .
c. Dose Expansion
- Cohort #1 – Patients with relapsed or refractory BPDCN OR patients with untreated BPDCN who are inappropriate for available therapies. BPDCN patients considered inappropriate for available therapies must be either >= 75 years of age OR 18 to 74 years of age if the patient has at least one comorbidity that the physician judges to be incompatible with intense and available therapies, eg, pulmonary, cardiac, hepatic, vascular comorbidities or is ineligible for available therapies, eg, hypoalbuminemia (serum albumin < 3.2 mg/dL) as an exclusion for tagraxofusp (ELZONRIS).
- Cohort #2 – Patients will have relapsed AML.
- Cohort #3 – Patients will have relapsed or refractory ALL (including any subtypes: B-cell, T-cell, Ph+ and Ph-).
- Cohort #4 – Patients will have relapsed or refractory other hematologic malignancies not included in the cohorts above (eg, high-risk/very highrisk MDS, MPN, CMML, BP-CML). Other CD123+ malignancies may be considered upon discussion with the Sponsor.
Note: BP-CML is defined as >= 30% blasts in blood, marrow, or both and the demonstration of extramedullary infiltrates of leukemic cells.
- Cohort #5 – Patients will have relapsed or refractory (to non-intense therapies) CD123+ AML.
2. Prior therapies:
a. Patients in Dose Escalation and Dose Expansion Cohort #1, #3, and #4 may have received up to four prior lines of therapy.
b. Patients in Dose Expansion Cohort #2 with AML may have received up to two prior lines of therapy
c. Patients in Dose Expansion Cohort #5 with AML may have received up to three prior lines of therapy.
3. Age >= 18 years of age.
4. Eastern Cooperative Oncology Group performance status <= 1. If nonambulatory due to a chronic disability, must be Karnofsky performance status > 70.
5. Previous treatment related toxicities must have resolved to Grade 1 (excluding alopecia).
6. Liver enzymes (aspartate aminotransferase and alanine aminotransferase) <= 2.5 x the upper limit of normal (ULN). Exceptions may be made for patients with elevated liver transaminases secondary to the underlying study disease
7. Total bilirubin <= 1.5 x ULN within 14 days of enrollment.
8. Serum creatinine <= 1.5 × ULN within 14 days of enrollment.
9. Echocardiogram or multigated acquisition scan demonstrating an ejection fraction >= 45%.
10. Patients with a prior autologous and allogeneic bone marrow transplant are eligible. Patients with an allogeneic transplant must meet the following conditions: the transplant must have been performed more than 120 days before the date of informed consent to this study, the patient must not have active >= Grade 2 acute graft versus host disease (GvHD), or either moderate or severe limited chronic GvHD, or extensive chronic GvHD of any severity; the patient must be off all immunosuppression for at least two weeks.
11. Voluntary written informed consent before performance of any study-related procedure not part of normal care.
[...]
For further details, please refer to protocol.

1. Caratteristiche della malattia:
a. Conferma di positività CD123 per mezzo di citometria a flusso o immunoistochimica (IHC). I pazienti precedentemente trattati con agenti mirati a CD123 potranno accedere a condizione che nei blasti l’espressione CD123 risulti ancora rilevabile.
b. Incremento della dose: LMA (esclusa la leucemia promielocitica acuta) o BPDCN recidivante o refrattaria, in base alla Classificazione dell’Organizzazione Mondiale della Sanità.
c. Espansione della dose:
- Coorte n. 1 - pazienti con BPDCN recidivante o refrattaria OPPURE pazienti con
BPDCN non trattata che non sono idonei alle terapie disponibili. I pazienti con BPDCN
considerati non idonei alle terapie disponibili devono avere un’età >=75 anni O compresa
tra 18 e 74 anni qualora il paziente presenti almeno una comorbilità che il medico ritiene
sia incompatibile con le terapie intensive e disponibili, ad es. comorbilità a livello
polmonare, cardiaco, epatico, vascolare, o non risulta idoneo alle terapie disponibili, ad
es. ipoalbuminemia (albumina sierica <3,2 mg/dl) come criterio di esclusione per
tagraxofusp (ELZONRIS).
- Coorte n. 2: i pazienti devono avere LMA recidivante.
- Coorte n. 3: i pazienti devono avere LLA recidivante o refrattaria (compresi eventuali
sottotipi: linfociti B, linfociti T, Ph+ e Ph-).
- Coorte n. 4: i pazienti devono avere “altre” malignità ematologiche recidivanti o
refrattarie non incluse nelle suddette coorti (per es. SMD ad alto/altissimo rischio, NMP,
LMMC, BP-CML). Previa discussione con lo sponsor, potranno essere prese in
considerazione altre malignità CD123+.
Nota: la CML in fase blastica è definita come >=30% di blasti nel sangue, nel midollo o in
entrambi e la dimostrazione di infiltrati extramidollari di cellule leucemiche.
- Coorte n. 5: i pazienti devono avere LMA recidivante o refrattaria (alle terapie non intensive) CD123+.
2. Precedenti terapie:
a. I pazienti nella Coorte di incremento della dose e di espansione della dose n. 1, 3 e 4 possono avere ricevuto un massimo di quattro linee di terapia precedenti.
b. I pazienti nella Corte di espansione della dose n. 2 con LMA possono avere ricevuto un
massimo di due linee di terapia precedenti.
c. I pazienti nella Coorte di espansione della dose n. 5 con LMA possono avere ricevuto un
massimo di tre linee di terapia precedenti
3. Età >=18 anni.
4. Stato di validità secondo l’Eastern Cooperative Oncology Group (gruppo orientale cooperativo di oncologia) <=1. Se il paziente non deambula a causa di disabilità cronica, si deve considerare lo stato di validità secondo la scala di Karnofsky >70
5. Le tossicità correlate al precedente trattamento devono essersi risolte a grado 1 (esclusa l’alopecia).
6. Enzimi epatici (aspartato aminotransferasi e alanina aminotransferasi) <=2,5 x limite superiore della norma (ULN). Possono essere fatte eccezioni per i pazienti con transaminasi epatiche elevate secondarie alla malattia dello studio sottostante.
7. Bilirubina totale <=1,5 x ULN entro 14 giorni dall’arruolamento.
8. Creatinina sierica <=1,5 ULN entro 14 giorni dall’arruolamento.
9. Ecocardiogramma o scansione di acquisizione a gate multipli che dimostri una frazione di eiezione >=45%.
10. I pazienti precedentemente sottoposti a trapianto autologo e allogenico di midollo osseo sono idonei. I pazienti con un trapianto allogenico devono soddisfare le seguenti condizioni: il trapianto deve essere stato eseguito oltre 120 giorni prima della data del consenso informato per questo studio, il paziente non deve presentare malattia del trapianto contro l’ospite (GvHD) acuta di grado >=2 in fase attiva o GvHD cronica limitata moderata o grave, oppure GvHD cronica diffusa di qualsiasi gravità; il paziente deve aver interrotto l’assunzione di tutti i farmaci immunosoppressivi da almeno due settimane.
[...]

Si prega di fare riferimento al protocollo per ulteriori dettagli.

E.4

Principal exclusion criteria

1. Patients who, in the judgment of their treating physician, have appropriate standard of care therapies will be excluded.
2. Patients with active central nervous system (CNS) disease will be excluded. A lumbar puncture does not need to be performed unless there is clinical suspicion of central nervous system involvement. All patients with a known history of CNS disease or brain metastasis must have been treated locally, have at least three consecutive lumbar punctures with no evidence of CNS disease, and must be clinically stable for at least four weeks prior to enrollment and have no ongoing neurological symptoms that in the opinion of the treating physician are related to active CNS disease (sequelae that are a consequence of prior CNS disease or the treatment are acceptable). Concurrent therapy for CNS prophylaxis or continuation of therapy for controlled CNS disease is permitted with the approval of the sponsor.
3. Patients with a history of venous occlusive disease of the liver.
4. Patients with a history of Grade 4 capillary leak syndrome, or non-cardiac Grade 4 edema are ineligible, e.g., related to SL-401 or other etiology.
5. Corrected QT interval (QTc Fridericia formula) > 480 msec.
6. Myocardial infarction within six months prior to enrollment or has New York Heart Association Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities prior to study entry.
7. Patients who have received any anti-cancer therapy including chemotherapy, immunotherapy, radiotherapy, hormonal, biologic, or any investigational agents within 14 days prior to drug administration on this study. Patients must have recovered to baseline from all acute toxicity from this prior therapy.
8. Clinically Relevant active infection including known active hepatitis B or C, human immunodeficiency virus infection, or cytomegalovirus or any other known concurrent infections disease that, in the judgment of the Investigator, would make a patient inappropriate for enrollment into this study (testing not required).
9. Patients who have undergone a major surgery within four weeks (or longer if not fully recovered) prior to study enrollment.
10. Serious or poorly controlled medical conditions that could be exacerbated by treatment or that would seriously compromise safety assessment or compliance with the protocol, in the judgment of the Investigator.

1. I pazienti per i quali, secondo il parere del proprio medico curante, sono a disposizione terapie standard idonee saranno esclusi.
2. I pazienti la cui malattia interessa il sistema nervoso centrale (SNC) saranno esclusi. Non è necessario eseguire una puntura lombare, a meno che non vi sia il sospetto clinico di coinvolgimento del sistema nervoso centrale. Tutti i pazienti con un’anamnesi nota di malattia del SNC o metastasi cerebrale devono essere stati trattati a livello locale, devono essersi sottoposti ad almeno tre punture lombari consecutive senza alcuna evidenza di malattia del SNC e devono essere clinicamente stabili da almeno quattro settimane prima dell’arruolamento senza mostrare sintomi neurologici in corso che, secondo il parere del medico curante, non siano correlati alla malattia attiva a carico del SNC (sono accettabili le sequele insorte come conseguenza di una precedente malattia del SNC o del trattamento). La terapia concomitante per la profilassi del SNC o la continuazione della terapia per la malattia del SNC controllata è consentita se preventivamente approvata dallo sponsor.
3. Pazienti con un’anamnesi di malattia venosa occlusiva del fegato.
4. I pazienti con un’anamnesi di sindrome da perdita capillare di grado 4 o edema non cardiaco di grado 4 non sono idonei, per es. nei casi correlati a SL-401 o ad altra eziologia.
5. Intervallo QT corretto (QTc) con la formula di Fridericia >480 msec.
6. Infarto miocardico nei sei mesi precedenti l’arruolamento o insufficienza cardiaca di classe III o IV secondo la New York Heart Association, angina non controllata, gravi aritmie ventricolari non controllate o evidenza elettrocardiografica di ischemia acuta o anomalie attive del sistema di conduzione prima dell’ingresso nello studio.
7. Pazienti che hanno ricevuto una qualsiasi terapia antitumorale, incluse chemioterapia, immunoterapia, radioterapia, terapia ormonale, terapia biologica o qualsiasi agente sperimentale nei 14 giorni precedenti la somministrazione del farmaco durante questo studio.
I pazienti devono essersi ripresi al basale da tutte le tossicità acute dovuta alla terapia precedente.
a. Nota: fanno eccezione i pazienti che hanno ricevuto un inibitore del checkpoint, i quali non devono aver ricevuto tale terapia nei 28 giorni precedenti la somministrazione del farmaco durante questo studio.
b. Si noti che questo criterio esclude l’idrossiurea, che può essere somministrata durante lo studio (si veda la Sezione 5.3.3).
8. Infezione attiva clinicamente rilevante incluse epatite B o C attiva nota, infezione da virus dell’immunodeficienza umana o citomegalovirus oppure qualsiasi altra malattia infettiva nota concomitante che, secondo il giudizio dello sperimentatore, renderebbe il paziente inadatto all’arruolamento in questo studio (non sono necessari test).
9. Pazienti che si sono sottoposti a un intervento di chirurgia maggiore nelle quattro settimane (o più in caso di mancata guarigione completa) precedenti l’arruolamento nello studio.
10. Condizioni mediche gravi o scarsamente controllate che potrebbero essere acutizzate dal trattamento o che potrebbero compromettere seriamente la valutazione della sicurezza o la conformità al protocollo secondo il giudizio dello sperimentatore.

E.5 End points

E.5.1

Primary end point(s)

Escalation Phase:
MTD and RP2D

BPDCN:
Complete remission rate (CR/CRi/CRc/complete remission with partial hematologic recovery [CRh])

Fase di incremento della dose:
MTD e RP2D

BPDCN:
Tasso di remissione completa (CR / CRI / CRc / remissione completa con recupero ematologico parziale [CRh])

E.5.1.1

Timepoint(s) of evaluation of this end point

End of Study (MTD, RP2D)

Fine dello studio (MTD, RP2D)

E.5.2

Secondary end point(s)

BPDCN:
Duration of response of patients with CR, CRc, or CRh

All patients:
-Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and DLTs
-PK parameters, including but not limited to observed maximum concentration
(Cmax) and area under the concentration versus time curve (AUC)
-ADA
- ANti-tumor activity in AML and ALL patients - Overall response rate (CR without minimal residual disease [CRMRD- ] + CR + CRh + complete remission with incomplete recovery [CRi] + morphologic leukemia-free state [MLFS] + partial remission [PR]), complete remission rate (CRMRD-, CR, CRh), composite complete remission (CCR) rate (CRMRD-, CR, CRh, CRi), and time to event outcomes (duration of response, event free survival [EFS], relapse-free survival [RFS], OS)
Anti-tumor activity in BPDCN:
-ORR (CR + CRc + CRh + CRi + PR)
- CCR rate (CR/CRc/CRh/CRi) and CR/CRc rate
- Time-to-event outcomes will be calculated using Kaplan Meier estimates (OS, RFS, and EFS)

BPDCN:
Durata della risposta nei pazienti con CR, CRc o CRh.

Tutti i pazienti:
- Eventi Avversi Emergenti dal Trattamento (TEAE), Eventi Avversi Gravi (SAE) e Tossicità Dose Limitanti (DLT)
- Parametri PK, incluso ma non limitato alla concentrazione massima osservata (Cmax) e l'area sotto la curva della concentrazione versus il tempo (AUC)
- ADA
- Attività antitumorale in pazienti LMA e LLA - Tasso di risposta globale (CR senza malattia minima residua [CRMRD-] + CR + CRh + CRi + stato morfologico libero da leucemia [MLFS] + remissione parziale [PR]), Tasso di risposta completa (CRMRD-, CR, CRh), tasso di remissione completa composta (CCR) (CRMRD-, CR, CRh, CRi) e tempo dell'outcome (durata della risposta, sopravvivenza libera da eventi [EFS], sopravvivenza libera da recidive [RFS], OS)
Attività antitumorale in BPDCN:
- ORR (CR + CRc + CRh + PR)
- tasso CCR (CR / CRc / CRh / CRi) e il tasso CR / CRc
- Gli outcome dei tempi all'evento saranno calcolati utilizzanzo stime Kaplan Meier (OS, RFS, and EFS)

E.5.2.1

Timepoint(s) of evaluation of this end point

End of Study

Fine 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

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

Yes

E.7.1.1

First administration to humans

Yes

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

Phase 1/2

fase 1/2

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

United States

France

Italy

Spain

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

The end of study will be defined as the latter of the completion of the safety follow-up visit for the last patient remaining on treatment or one year from the last accrued patient’s first visit.

La fine dello studio sarà definita come l'ultima visita di follow-up per il completamento della sicurezza dell'ultimo paziente rimasto in trattamento o per un anno dalla prima visita del paziente in scadenza.

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:

155

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

232

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)

There are no plans for treatment or care after subjects have ended their participation in the study.

Non ci sono piani per trattamenti o cure dopo che i soggetti terminano la loro 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

2019-02-27

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-02-13

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

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IMP with orphan designation in the indication
Orphan Designation Number:
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