Clinical Trials Register

Summary
EudraCT Number:	2014-000148-14
Sponsor's Protocol Code Number:	PLX108-10
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-01-13
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2014-000148-14

A.3

Full title of the trial

A double-blind, Randomized, Placebo-controlled Phase 3 Study of Orally Administered PLX3397 in Subjects with Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath ENLIVEN

Studio di fase III randomizzato, in doppio cieco, controllato con placebo, su PLX3397 per via orale in soggetti con sinovite villonodulare pigmentosa o tumore a cellule giganti delle guaine tendinee ENLIVEN

A.3.1

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

A clinical Trial comparing the effectiveness of PLX3397 against placebo in the treatment of Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath ENLIVEN

Sudio clinico per comparare l'efficacia di PLX3397 verso il placebo nel trattamento della sinovite villonodulare pigmentosa o tumore a cellule giganti delle guainee tendinee ENLIVEN

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

PLX108-10

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02371369

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	DAIICHI SANKYO 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	Daiichi Sankyo Pharma Development
B.4.2	Country	United States
B.4.1	Name of organisation providing support	Daiichi Sankyo Company, Limited
B.4.2	Country	Japan
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Daiichi Sankyo Inc
B.5.2	Functional name of contact point	Clinical Trial Information contact
B.5.3	Address:
B.5.3.1	Street Address	399 Thornall Street
B.5.3.2	Town/ city	EDISON
B.5.3.3	Post code	NJ 08837
B.5.3.4	Country	United States
B.5.4	Telephone number	0017325905000
B.5.5	Fax number	0017329065690
B.5.6	E-mail	eu_cta@dsi.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	Yes
D.2.5.1	Orphan drug designation number	EMA/OD/279/14
D.3 Description of the IMP
D.3.1	Product name	PLX3397
D.3.2	Product code	PLX3397
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	pexidartinib cloridrato
D.3.9.1	CAS number	1029044-16-3
D.3.9.2	Current sponsor code	PLX3397
D.3.9.3	Other descriptive name	Pexidartinib
D.3.9.4	EV Substance Code	SUB33175
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	200
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Pigmented villonodular synovitis (PVNS) / giant cell tumor of tendon sheat (GCT-TS)

Sinivite villonodulare pigmentosa (PVNS) / tumore a cellule giganti delle guainee tendinee

E.1.1.1

Medical condition in easily understood language

Tumor of the tendon sheath

Tumore delle guainee tendinee

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	LLT
E.1.2	Classification code	10062856
E.1.2	Term	Giant cell tumour of tendon sheath benign
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

The primary objective of this study is to compare the response rate of PLX3397 with that of placebo per Response Evaluation Criteria in Solid Tumor, version 1.1 (RECIST 1.1) at week 25 in subjects with symptomatic, locally advanced PVNS or GCT-TS

L'obiettivo primario dello studio ¿ confrontare, alla Settimana 25, il tasso di risposta di PLX3397 rispetto al placebo in soggetti con PVNS/GCT-TS sintomatico localmente avanzato, valutato secondo i Criteri di valutazione della risposta nei tumori solidi, versione 1.1 (Response Evaluation Criteria in Solid Tumors, RECIST 1.1).

E.2.2

Secondary objectives of the trial

The secondary efficacy objectives are to evaluate: (i) patient-reported outcomes (PROs), including the Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) item, Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale, and Worst Stiffness NRS item, at week 25; (ii) response based on TVS at week 25; (iii) range of motion at week 25; and (iv) duration of response

Gli obiettivi secondari di efficacia consistono nel valutare: (i) gli esiti riferiti dai pazienti (Patient-Reported Outcomes, PRO), comprese la voce "Peggior dolore" della Scala numerica per la misurazione del dolore (Numeric Rating Scale, NRS) del Breve inventario del dolore (Brief Pain Inventory, BPI), la Scala della funzionalit¿ fisica (Physical Function Scale, PFS) del Sistema di misurazione degli esiti riferiti dai pazienti (Patient-Reported Outcomes Measurement Information System, PROMIS) e la voce "Peggior rigidit¿" della scala NRS, alla Settimana 25; (ii) la risposta in base al Punteggio del volume tumorale (Tumor Volume Score, TVS) alla Settimana 25; (iii) l'escursione di movimento alla Settimana 25; e (iv) la durata della risposta.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Age =18 years.
2.A diagnosis of PVNS or GCT-TS (i) that has been histologically confirmed either by a pathologist at the treating institution or a central pahologist, and (ii) where surgical resection would be associated with potentially worsening functional limitation or severe morbidity (locally advanced disease), with morbidity determined consensually by qualified personnel (eg, two surgeons or a mutli-disciplinary tumor board).
3.Measurable disease as defined by RECIST 1.1 (except that a minimal size of 2 cm is required), assessed from MRI scans by a central radiologist.
4.Symptomatic disease because of active PVNS or GCT-TS. a. a worst pain of at least 4 at any time during the week preceding the Screening Visit (based on scale of 0 to 10, with 10 representing "pain as bad as you can imagine"). b. a worst stiffness of at least 4 at any time during the week preceding the Screening Visit (based on a scale of 0 to 10, with 10 representing "stiffness as bad as you can imagine").
5.Stable prescription of analgesic regimen during the 2 weeks prior to randomization.
6.During the 2 week prior to randomization, at least 4 of 7 consecutive days of BPI Worst Pain NRS items and Wirst Stiffness NRS items completed correctly.
7.Women of childbearing potential must have a negative serum pregnancy test within the 14-day period prior to randomization. (Where demanded by local regulations, this test may be required within 72 hours of randomization).
8.Males and females of childbearing potential are permitted in the study so long as they consent to avoid getting their partner pregnant or becoming pregnant, respectively, by using a highly effective
contraception method, as described below, throughout the study and for up to 90 days after completion. Highly effective methods of contraception include: intra-uterine device (nonhormonal or hormonal), bilateral tubal occlusion, vasectomy, sexual abstinence, or barrier methods (e.g., condom, diaphragm) used in combination with hormonal methods associated with inhibition of ovulation. Women of nonchildbearing potential may be included if they are either surgically sterile or have been postmenopausal for = 1 year. Women who have documentation of at least 12 months of spontaneous amenorrhea and have an FSH level > 40 mIU/mL will be considered postmenopausal.
9.Adequate hematologic, hepatic, and renal function.
10.Willingness and ability to complete the BPI Worst Pain NRS item, Worst Stiffness NRS item, PROMIS Physical Function Scale, and other self-assessment instruments throughout the study.
11.Willingness and ability to use a diary.
12.Signature of Informed Consent Form, prior to any study-related procedures and to comply with all study requirements

1. Età =18 anni.
2. Pazienti con diagnosi di PVNS o GCT-TS (i) confermata istologicamente da un patologo che opera nel centro di trattamento o da un patologo centrale, (ii) per i quali la resezione chirurgica comporterebbe un potenziale peggioramento della limitazione funzionale o una grave morbilità (malattia localmente avanzata); la morbilità dovrà essere determinata consensualmente da personale qualificato (ad esempio, due chirurghi o un consiglio multidisciplinare per la valutazione dei tumori).
3. Malattia misurabile come definita dal RECIST 1.1 (a meno che sia richiesta una dimensione minima di 2 cm), valutata da un radiologo centrale tramite scansioni MRI.
4. Malattia sintomatica dovuta a PVNS/GCT-TS attivo, definita secondo una o più delle seguenti descrizioni:
a. punteggio minimo di 4 nella voce "peggior dolore" in qualsiasi momento durante la settimana precedente alla Visita di screening (basato sulla scala di valutazione da 0 a 10, laddove 10 rappresenta il "peggior dolore immaginabile").
b. punteggio minimo di 4 nella voce "peggiore rigidità" in qualsiasi momento durante la settimana precedente alla Visita di screening (basato sulla scala di valutazione da 0 a 10, laddove 10 rappresenta la "peggiore rigidità possibile").
5. Prescrizione di regime analgesico stabile nelle 2 settimane precedenti alla randomizzazione.
6. Corretta compilazione della voce "Peggior dolore" della NRS del BPI e della voce "Peggior rigidità" della NRS per almeno 4 su 7 giorni consecutivi delle due settimane precedenti alla randomizzazione.
7. Le donne in età fertile devono presentare un test di gravidanza sul siero negativo entro 14 giorni dalla randomizzazione (ove previsto dalle normative locali, tale test può essere richiesto entro 72 ore dalla randomizzazione).
8. Gli uomini e le donne in età fertile possono prendere parte allo studio a condizione che acconsentano ad evitare di procreare/iniziare una gravidanza adottando, durante la sperimentazione e fino a 90 giorni dopo la sua conclusione, uno dei seguenti metodi anticoncezionali altamente efficaci: contraccettivi ormonali associati all’inibizione dell’ovulazione, dispositivo intrauterino, sterilizzazione chirurgica (compresa vasectomia del partner) o astinenza sessuale. Le donne non fertili possono essere incluse se chirurgicamente sterili o in post-menopausa da =1 anno. Saranno considerate in post-menopausa le donne con amenorrea spontanea documentata di almeno 12 mesi che presentano un livello di ormone follicolo-stimolante >40 mIU/ml.
9. Funzionalità renale, epatica ed ematologica adeguata, definita in base ai seguenti valori:
• Conta assoluta dei neutrofili =1,5×109/l
• Emoglobina >10 g/dl
• Conta piastrinica =100×109/l
• AST/ALT =1,5×ULN
• Bilirubina totale =1,5×ULN
• Creatinina sierica =1,5×ULN
10. Volontà e capacità di compilare, durante lo studio, la voce "Peggior dolore" della NRS del BPI, la voce "Peggiore rigidità" della NRS, la Scala della funzionalità fisica del PROMIS e altri eventuali strumenti di autovalutazione.
11. Volontà e capacità di usare un diario elettronico.

E.4

Principal exclusion criteria

1. Investigational drug use within 28 days of randomization. 2. Previous use of pexidartinib or any biologic treatment targeting CSF-1 or the CSF1R; previous use of oral tyrosine kinase inhibitors, eg, imatinib or nilotinib, are allowed. 3. Active cancer (either concurrent or within the last year of starting study treatment) that requires non-surgical therapy (eg, surgical, chemotherapy or radiation therapy), with the exception of PVNS/GCT TS, adequately treated basal or squamous cell carcinoma of the skin, melanoma in-situ, or carcinoma in-situ of the cervix or breast, or prostate carcinoma with a prostate specific antigen value <0,2 ng/mL. 4. Known metastatic PVNS/GCT-TS. 5. Active or chronic infection with hepatitis C virus (HCV) or hepatitis B virus or know active or chronic infection with human immunodeficiency virus. 6. Known active tuberculosis. 7. Significant concomitant arthropathy in the affected joint, serious illnes, uncontrolled infection, or a medical or psychiatric history that, in the investigator's opinion, would likely interfere with the person's study participation or the interpretation of his or her results. 8. Women who are breastfeeding. 9. A screening Fridericia corrected QT interval (QTcF) = 450 ms(men) or = 470 ms (women). 10. MRI contraindications. 11. History of hypersensitivity to any excipients in the investigational product.
12. Inability to swallow capsules.

1. Uso di un farmaco sperimentale nei 28 giorni precedenti alla randomizzazione;
2. Uso precedente di pexidartinib o di qualsiasi trattamento biologico mirato al fattore di stimolazione delle colonie 1 (Colony Stimulating Factor 1, CSF-1) o al recettore del CSF-1; è ammesso l'uso precedente di inibitori orali della tirosin-chinasi, quali, ad esempio, imatinib o nilotinib.
3. Tumore attivo (concomitante o insorto nell'anno precedente all'inizio del trattamento in studio) che richiede una terapia (ad esempio, chirurgica, chemioterapia o radioterapia), ad eccezione del carcinoma cutaneo a cellule basali o squamose adeguatamente trattato, del melanoma in situ, del carcinoma in situ della cervice o mammario, o del carcinoma prostatico con un valore di antigene prostatico specifico <0,2 ng/ml.
4. PVNS/GCT-TS metastatico noto.
5. Infezione nota attiva o cronica da virus dell'epatite B o C, o da virus dell'immunodeficienza umana.
6. Tubercolosi attiva nota.
7. Artropatia concomitante significativa a carico delle articolazioni interessate, malattia grave, infezione non controllata oppure anamnesi medica o psichiatrica che, a giudizio dello Sperimentatore, potrebbe compromettere la partecipazione allo studio di un candidato o l'interpretazione dei suoi risultati.
8. Donne in allattamento.
9. Intervallo QT corretto mediante la formula di Fridericia (QTcF) =450 ms (uomini) o =470 ms (donne) allo screening.
10. Controindicazioni per la MRI.
11. Anamnesi di ipersensibilità a uno degli eccipienti del prodotto sperimentale.
12. Incapacità di ingoiare capsule.

E.5 End points

E.5.1

Primary end point(s)

The proportion of subjects who achive a complete or partial response at the week 25 visit based on centrally read MRI scans and RECIST 1.1.

Percentuale di soggetti che raggiungono una risposta completa (Complete Response, CR) o parziale (Partial Response, PR) alla Visita della Settimana 25, sulla base di una valutazione eseguita tramite la lettura centrale delle scansioni MRI e i criteri RECIST 1.1.

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 25

Settimana 25

E.5.2

Secondary end point(s)

1. Proportion of responders based on the BPI Worst Pain NRS item and analgesic use by BPI-30 definition 2. Proportion of responders based on centrally evaluated MRI scans and TVS at the week 25 visit 3. Mean change from baseline in range of motion of the affectd joint, relative to a reference standard for the same joint, at the week 25 visit 4. Mean change from baseline score in the PROMIS Physical Function Scale at the Week 25 visit 5. Mean change from the baseline score in the Worst Stiffness NRS item at th week 25 visit 6. Duration of response (CR or PR) based on MRI and RECIST 1.1 7.Duration of response (CR or PR) based on MRI and TVS

1. Percentuale di responder in base alla voce "Peggior dolore" della NRS del BPI e all'uso di analgesici secondo la definizione BPI-30 2.Percentuale di responder in base alla valutazione centrale delle scansioni MRI e al TVS alla Visita della Settimana 25 3. ¿ Variazione media alla Visita della Settimana 25 rispetto al basale nell'escursione di movimento dell'articolazione interessata rispetto ad uno standard di riferimento per la stessa articolazione 4.Variazione media alla Visita della Settimana 25 rispetto al basale nel punteggio della Scala della funzionalit¿ fisica del PROMIS 5.Variazione media alla Visita della Settimana 25 rispetto al basale nel punteggio della voce "Peggiore rigidit¿" della NRS 6.Durata della risposta (CR o PR) in base alla MRI e ai criteri RECIST 1.1. 7.Durata della risposta (CR o PR) in base alla MRI e al TVS

E.5.2.1

Timepoint(s) of evaluation of this end point

week 25

settimana 25

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)

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

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

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

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Canada

United States

Denmark

France

Germany

Hungary

Italy

Netherlands

Poland

Spain

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

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:

100

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

126

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)

Subjects who complete Part 2 will be eligible to continue for longer efficacy and safety follow-up or to enter a separate protocol to continue receiving PLX3397 until disease progression, unacceptable
toxicity, or the occurrence of other termination criteria.

I soggetti che completano la Parte 2 saranno elegibili per continuare per efficacia prolungata o per follow-up di sicurezza o per entrare in un protocollo separato per ricevere PLX3397 fino a che non vi sia progressione di malattia, tossicit¿ inaccettabile o la manifestazione di un altro criterio di terminazione.

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

2015-07-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-04-29

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-04-30

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