Clinical Trials Register

Summary
EudraCT Number:	2018-003526-88
Sponsor's Protocol Code Number:	CZAR-1
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:	2021-08-30
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

2018-003526-88

A.3

Full title of the trial

Efficacy and safety of Carfilzomib in combination with Ibrutinib vs. Ibrutinib alone in Waldenström’s Macroglobulinemia

Efficacia e sicurezza di Carfilzomib in combinazione con Ibrutininb versus Ibrutininb in monoterapia nella macroglobulinemia di Waldenström.

A.3.1

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

Efficacy and safety of Carfilzomib in combination with Ibrutinib vs. Ibrutinib alone in Waldenström’s Macroglobulinemia

Efficacia e sicurezza di Carfilzomib in combinazione con Ibrutininb versus Ibrutininb in monoterapia nella macroglobulinemia di Waldenström.

A.3.2

Name or abbreviated title of the trial where available

CZAR-1

A.4.1

Sponsor's protocol code number

CZAR-1

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04263480

A.5.4

Other Identifiers

Name:

CZAR-1

Number:

CZAR-1

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	UNIVERSITäTSKLINIK ULM
B.1.3.4	Country	Germany
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	Amgen Inc.
B.4.2	Country	European Union
B.4.1	Name of organisation providing support	Janssen Pharmaceutica NV
B.4.2	Country	European Union
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	University Hospital Ulm
B.5.2	Functional name of contact point	Christian Buske
B.5.3	Address:
B.5.3.1	Street Address	Albert-Einstein-Allee 23
B.5.3.2	Town/ city	Ulm
B.5.3.3	Post code	89091
B.5.3.4	Country	Germany
B.5.4	Telephone number	004973150065800
B.5.5	Fax number	004973150065802
B.5.6	E-mail	christian.buske@uni-ulm.de

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	Imbruvica
D.2.1.1.2	Name of the Marketing Authorisation holder	Janssen Pharmaceutica NV
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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	Imbruvica
D.3.2	Product code	[Imbruvica]
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	Ibrutinib
D.3.9.1	CAS number	936563-96-1
D.3.9.2	Current sponsor code	Imbruvica
D.3.9.3	Other descriptive name	Ibrutinib
D.3.9.4	EV Substance Code	SUB120863
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	140
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	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	European Union
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	Kyprolis
D.3.2	Product code	[Kyprolis]
D.3.4	Pharmaceutical form	Powder for solution for injection/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	Kyprolis
D.3.9.3	Other descriptive name	CARFILZOMIB
D.3.9.4	EV Substance Code	SUB32911
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	60
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

Waldenström's Macroglobulinemia

Macroglobulinemia di Waldenström

E.1.1.1

Medical condition in easily understood language

Waldenström's Macroglobulinemia

Macroglobulinemia di Waldenström

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	PT
E.1.2	Classification code	10047801
E.1.2	Term	Waldenstrom's macroglobulinaemia
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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

The primary objective of the study is to explore the efficacy of Carfilzomib in combination with Ibrutinib compared to Ibrutinib alone in patients with treatment naïve or relapsed WM.

Studiare l’efficacia di Carfilzomib in combinazione con Ibrutinib rispetto a Ibrutininb in monoterapia in pazienti affetti da Macroglobulinemia di Waldenström naive al trattamento o con ricaduta.

E.2.2

Secondary objectives of the trial

Response rate (CR, VGPR, PR, MR) 12 and 24 months after the start of treatment
Best response
Time to best response
Time to first response
Time to treatment failure,
Remission Duration
Progression free survival
Cause specific survival
Overall survival
Safety
Quality of life

Percentuale di risposta (Completa, Parziale Molto Buona, Parziale, Minore) e tasso di risposte obiettive (Completa, Parziale Molto Buona, Parziale) 12 e 24 mesi dopo l’inizio del trattamento.
Miglior risposta
Tempo alla miglior risposta
Tempo alla prima risposta
Tempo al fallimento del trattamento
Durata della remissione
Sopravvivenza libera da progressione
Sopravvivenza causa-specifica
Sopravvivenza globale
Sicurezza
Qualità della vita

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Proven clinicopathological diagnosis of WM as defined by consensus panel one of the Second IWWM4.
• De novo or relapsed / refractory WM independent of the genotype.
• Patients must have at least one of the following criteria to start study treatment as as defined by the protocol criteria.
• World Health Organization (WHO) / ECOG performance status = 2.
• Left ventricular ejection fraction = 40% as assessed by transthoracic echocardiogram (TTE).
• Age = 18 years (male and female),
• Life expectancy > 3 months in the opinion of the investigator, adequate laboratory values.
• Baseline platelet count = 50 x109/L, absolute neutrophil count = 0.75 x 109/L (if not due to BM infiltration by the lymphoma).
• Meet the following pre-treatment laboratory criteria at the screening visit conducted within 30 days prior to randomization:
¿ ASAT (SGOT): < 3.0 times the ULN.
¿ ALAT (SGPT): < 3.0 times the ULN.
¿ Total Bilirubin: < 1.5 times the ULN, unless clearly related to the disease (except if due to Gilbert’s syndrome).
¿ Serum creatinine: = 1.5 times the ULN
• Females of childbearing potential (FCBP), i.e. fertile, following menarche and until becoming postmenopausal, must agree to use a highly effective method of birth control for the duration of the therapy up to 6 months after end of therapy with Carfilzomib or Ibrutinib.
• Men must agree not to father a child for the duration of therapy and 6 months after (use of a condom) and must agree to advice a female
partner to use a highly effective method of birth control. Males must refrain from sperm donation for at least 6 months after the last dose of Carfilzomib or Ibrutinib.
• Each patient must sign an informed consent form in a fluent language of the patient indicating that he or she understands the purpose of and procedures required for the study and are willing to participate in the study. Patients must be willing and able to adhere to the prohibitions and restrictions specified in this protocol.

• Diagnosi clinico-patologica confermata di WM, definita secondo le raccomandazioni promosse dal Secondo Workshop Internazionale sulla MW.
• WM di nuova diagnosi, ricaduta o refrattaria indipendentemente dal genotipo.
• I pazienti devono avere almeno uno dei seguenti criteri per iniziare il trattamento in studio, come definito dai criteri indicati in protocollo.
• WHO/ECOG Performance status = 2.
• Frazione di eiezione ventricolare sinistra = 40%, misurata con ecocardiografia transtoracica.
• Età = 18 anni (maschi e femmine).
• Aspettativa di vita secondo giudizio dello sperimentatore > 3 mesi.
• Conta basale piastrinica = 50 x 109/L, assolta di neutrofili = 0,75 x 109/L (se non dovuta ad infiltrazione midollare da parte del linfoma).
• Rispetto dei seguenti criteri di laboratorio alla valutazione di screening effettuata nei 30 giorni che precedono la randomizzazione:
o AST (SGOT) < 3 volte il limite superiore di normalità
o ALT (SGPT) < 3 volte il limite superiore di normalità
o Bilirubina totale < 1,5 volte il limite superiore di normalità, a meno che non sia chiaramente correlata alla patologia in studio (eccezion fatta per la sindrome di Gilbert)
o Creatinina sierica: = 1,5 volte l'ULN
• Donne potenzialmente fertili (ovvero donne che si trovano nel periodo tra il menarca e l’ingresso in post-menopausa) devono accettare di impiegare metodi contraccettivi altamente efficaci per tutta la durata della terapia fino a 6 mesi successivi alla fine del trattamento con Carfilzomib o Ibrutininb (vedi protocollo per ulteriori dettagli)
• Uomini devono accettare di non procreare per tutta la durata della terapia e nei 6 mesi successivi, pertanto devono usare il preservativo e avvertire il partner femminile rispetto alla necessità di adottare un metodo contraccettivo altamente efficace. Devono inoltre astenersi dalla donazione di sperma per l’intera durata del trattamento e per almeno 6 mesi dopo l’ultima dose di Carfilzomib o Ibrutinib.
• Consenso informato scritto. Ciascun paziente deve firmare un modulo di consenso che attesti la comprensione dello scopo e delle procedure dello studio nonché la volontà a partecipare. Volontà e capacità di aderire ai divieti e alle restrizioni specificate dal protocollo di studio

E.4

Principal exclusion criteria

• Previous treatments with following substances Prior exposure to Ibrutinib or other BTK inhibitors and Prior exposure to Carfilzomib
• Serious medical or psychiatric illness
• Active HIV, HBV or HCV infection
• Central Nervous System involvement by lymphoma.
• History of a non-lymphoid malignancy except
• Uncontrolled illness including, but not limited to. (See the protocol).
• Recent major surgery within 30 days prior to randomization.
• Known cirrhosis
• Approved or investigational anticancer treatment within 21 days prior to randomization.
• Glucocorticoid therapy within 14 days prior to randomization
• Focal radiation therapy within 7 days prior to randomization.
• Contraindication to any of the required concomitant drugs or supportive treatments, including hypersensitivity to antiviral drugs.
• Hypersensitivity to the active substances or to any of the excipients of the investigational medicinal products
• Active infection
• Ascites
• Uncontrolled hypertension
• History of stroke or intracranial hemorrhage
• Known interstitial lung disease.
• Pleural effusions.
• Infiltrative pulmonary disease, known pulmonary hypertension.
• Known chronic obstructive pulmonary•
• Known severe persistent asthma
• Autologous or allogeneic stem cell transplant less
• Vaccination with
• Patients who require strong or moderate inducers or inhibitors for cytochrome
• Patients who have an uncontrolled bleeding disorder or require an anticoagulant
• History or evidence of any other clinically significant disorder, condition or disease (with the exception of those outlined above) that, in the opinion of the investigator or sponsor, if consulted, would pose a risk to patient safely or interfere with the study evaluation, procedures or completion.
• Patient is a woman who is pregnant or breastfeeding (and do not consent to discontinue breast-feeding) or planning to become pregnant while enrolled in this study or within 6
months after the last study treatment.
• Vulnerable patients, e.g. patients who are incapable of giving informed consent (severe dementia or psychosis, patients kept in detention).
• Participation in another interventional clinical study within 30 days before randomization in this study.

• Trattamenti pregressi con Ibrutininb o altri inibitori della tirosin-chinasi di Bruton (TBK) e Carfilzomib.
• Serie condizioni patologiche mediche o psichiatriche
• Infezioni attive di HIV, HBV o HCV
• Coinvolgimento del sistema nervoso centrale da parte del linfoma
• Storia di malignità non linfoide con alcune eccezioni
• Patologie non controllate incluse ma non limitate. (Vedi protocollo)
• Chirurgia maggiore, nei 30 giorni precedenti la randomizzazione.
• Cirrosi
• Trattamento antitumorale approvato o sperimentale nei 21 giorni che precedono la randomizzazione.
• Terapia con glucocorticoidi nei 14 giorni che precedono la randomizzazione.
• Radioterapia focale nei 7 giorni precedenti la randomizzazione.
• Controindicazione ad ognuno dei farmaci concomitanti richiesti o ai trattamenti di supporto, inclusa ipersensibilità ai farmaci antivirali.
• Ipersensibilità ai principi attivi o a uno degli eccipienti dei farmaci sperimentali.
• Infezione attiva.
• Ascite
• Ipertensione non controllata
• Storia di ictus o emorragia intracranica
• Malattia polmonare interstiziale nota.
• Versamenti pleurici.
• Malattia polmonare infiltrante, ipertensione polmonare nota.
• Malattia polmonare cronica.
• Nota asma grave persistente
• Trapianto autologo o allotrapianto di staminali
• Vaccinazione con vaccini vivi
• Pazienti che richiedono forti o moderati induttori o inibitori del citocromo
• Pazienti che, allo screening, presentano un disordine di sanguinamento non controllato o richiedono un anticoagulante
• Storia o evidenza di qualunque altro disordine, condizione o malattia clinicamente significativi
• Donne in gravidanza o allattamento, che non accettano di interrompere l’allattamento al seno, o che programmano di iniziare una gravidanza durante il periodo dello studio o nei 6 mesi successivi alla fine dell’ultimo trattamento.
• Pazienti vulnerabili, ovvero incapaci di dare il consenso informato (es. pazienti con grave demenza o psicosi, detenuti).
• Partecipazione ad un altro studio clinico interventistico nei 30 giorni precedenti la randomizzazione.

E.5 End points

E.5.1

Primary end point(s)

The aim of this study is to investigate the rate of CR or VGPR 12 months after the start of treatment using the response criteria updated at the Sixth IWWM (CR/VGPR).

Lo scopo di questo studio è quello di indagare il tasso di CR o VGPR 12 mesi dopo l'inizio del trattamento utilizzando i criteri di risposta aggiornati al Sesto IWWM (CR / VGPR).

E.5.1.1

Timepoint(s) of evaluation of this end point

After 12 months

Dopo 12 mesi

E.5.2

Secondary end point(s)

Response rate (CR, VGPR, PR, MR) and ORR (CR, VGPR, PR) 12 and 24 months after the start of treatment
Best response
Time to best
response
Time to first response
Time to treatment failure
Remission Duration
Progression free survival
Cause specific survival
Overall survival
Safety
Quality of life

E.5.2.1

Timepoint(s) of evaluation of this end point

after 12 and 24 months

dopo 12 e 24 mesi

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

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

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

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

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

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

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

184

F.4.2.2

In the whole clinical trial

184

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 who benefit from Ibrutinib treatment will receive further treatment with Ibrutinib after7 years after first patient in. Further Treatment and follow up is in the direction of the investigator.

I pazienti che traggono beneficio dal trattamento con Ibrutinib riceveranno un ulteriore trattamento con Ibrutinib 7 anni dopo l'ingresso del primo paziente. Ulteriori trattamenti e follow-up sono nella direzione dello sperimentatore.

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	German Lymphoma Alliance - GLA
G.4.3.4	Network Country	Germany

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2021-07-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-07-01

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

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IMP with orphan designation in the indication
Orphan Designation Number:
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