Clinical Trials Register

Summary
EudraCT Number:	2010-022403-22
Sponsor's Protocol Code Number:	CLCI699C2201
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:	2011-09-20
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

2010-022403-22

A.3

Full title of the trial

A proof-of concept, open-label, forced titration, multicenter study to assess the safety/tolerability and efficacy of 10-weeks treatment of LCI699 in patients with Cushing’s disease

Studio multicentrico, in aperto, a titolazione forzata, proof of concept, per valutare la sicurezza/tollerabilita' ed efficacia di un trattamento della durata di 10 settimane con LCI699 in pazienti con malattia di Cushing

A.3.1

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

Open-label, multicenter study to assess the safety/tolerability and efficacy of 10-weeks treatment of LCI699 in patients with Cushing’s disease

Studio multicentrico, in aperto per valutare la sicurezza/tollerabilita' ed efficacia di un trattamento della durata di 10 settimane con LCI699 in pazienti con malattia di Cushing

A.4.1

Sponsor's protocol code number

CLCI699C2201

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	NOVARTIS FARMA
B.1.3.4	Country	Italy
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	Novartis Pharma
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novartis Farma
B.5.2	Functional name of contact point	Drug Regulatory Affairs
B.5.3	Address:
B.5.3.1	Street Address	Largo Umberto Boccioni, 1
B.5.3.2	Town/ city	ORIGGIO (VA)
B.5.3.3	Post code	21040
B.5.3.4	Country	Italy
B.5.4	Telephone number	+39 02 96541
B.5.5	Fax number	+39 02 9659066
B.5.6	E-mail	info.italia@novartis.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.3.2	Product code	LCI699
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.3.9.2	Current sponsor code	LCI699
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.3	Concentration number	1
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	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.3.2	Product code	LCI699
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.3.9.2	Current sponsor code	LCI699
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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

Cushing's disease

malattia di Cushing

E.1.1.1

Medical condition in easily understood language

insufficienza surrenalica

E.1.1.2

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.0
E.1.2	Level	PT
E.1.2	Classification code	10011652
E.1.2	Term	Cushing's syndrome
E.1.2	System Organ Class	10014698 - Endocrine disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the effects of 10 weeks treatment of LCI699 on 24 hour urine free cortisol (UFC) in patients with Cushing’s disease.

Valutare gli effetti di 10 settimane di trattamento con LCI699 sull’UFC nelle 24 ore in pazienti con la malattia di Cushing.

E.2.2

Secondary objectives of the trial

To assess the safety and tolerability of multiple doses of LCI699 in patients with Cushing’s disease. To assess the effect of LCI699 on steroid hormones of the HPA-axis in plasma, urine and saliva. To assess the effects of LCI699 on improving the metabolic abnormalities (hypertension, dyslipidaemia, obesity, insulin sensitivity) of Cushing’s disease. To determine steady state trough plasma concentrations of LCI699.

 Valutare la sicurezza e la tollerabilita' di dosi multiple di LCI699 in pazienti con la malattia di Cushing  Valutare l’effetto di LCI699 sugli ormoni steroidei dell’asse ipotalamo-ipofisi-surrene (Hyphothalamic-pituitary-adrenal - HPA) nel plasma, nelle urine e nella saliva  Valutare gli effetti di LCI699 nel migliorare le alterazioni metaboliche (ipertensione, dislipidemia, obesita', sensibilita' all’insulina) della malattia di Cushing  Determinare il livello minimo delle concentrazioni plasmatiche di LCI699 allo stato stazionario.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent must be obtained before any assessment is performed.2. Male or female patients aged 18 – 75 years.3. Patients must have confirmed Cushing’s Disease (including de novo patients) as evidenced by:a) UFC >1.5XULN (Mean value of three 24-hour urine samples collected within 14days);b) Morning plasma ACTH above 10 pg/mllower limit of normal;c) Confirmation of pituitary origin of excess ACTH by at least one of the following three:- History of MRI confirmation of pituitary adenoma (greater than or equal to 6mm)with positive dynamic test (e.g. CRH or high dose dexamethasone suppression test);- History of inferior petrosal sinus gradient >3 after CRH stimulation;- Prior pituitary surgery with histopathology confirming an ACTH staining adenoma; - Subjects are permitted to washout current drug therapy to meet these entry criteria if they have a known diagnosis of Cushing’s disease;4. For patients on medical treatment for Cushing’s disease the following washout periods must be completed before baseline efficacy assessments are performed:- Inhibitors of steroidogenesis (ketoconazole, metyrapone, rosiglitazone): 1 week; - Dopamine agonists (bromocriptine, cabergoline), PPAR-gamma agonists (rosiglitazone, pioglitazone): 4 weeks; - Octreotide LAR, Pasireotide LAR and Lanreotide autogel: 8 weeks; - Lanreotide SR: 4 weeks; - Octreotide and Pasireotide (immediate release formulation): 1 week; - Other experimental therapy: at least 5 half-lives.

1.Consenso informato scritto ottenuto prima dell’effettuazione di qualsiasi valutazione. 2.Pazienti di sesso maschile o femminile di eta' compresa tra 18 e 75 anni. 3.Pazienti con malattia di Cushing confermata, dimostrata da: a) UFC >1.5XULN (valore medio di tre campioni di urine delle 24 ore raccolti entro 14 giorni); b) ACTH plasmatico al mattino al di sopra del 10pg/mL limite inferiore di normalita'; c) conferma dell’origine pituitaria dell’eccesso di ACTH data da almeno uno dei tre elementi seguenti: - Anamnesi di adenoma pituitario (maggiore o uguale a 6 mm) confermato con risonanza magnetica nucleare con test dinamico positivo (ad es. test al CRH o test della soppressione di dose elevata di desametasone); - Anamnesi di gradiente del seno petroso > 3 dopo stimolazione del CRH; - Pregressa chirurgia pituitaria con conferma istopatologica di adenoma ACTH dipendente; - Ai soggetti sara' consentito di effettuare un periodo di washout dalla terapia in corso per soddisfare questi criteri di ingresso se hanno una diagnosi nota di malattia di Cushing. 4.Per i pazienti in trattamento medico per la malattia di Cushing, devono essere completati i seguenti periodi di washout prima dell’effettuazione delle valutazioni basali di efficacia:-Inibitori della steroidogenesi (chetoconazolo, metirapone, rosiglitazone): 1 settimana; -Agonisti della dopamina (bromocriptina, cabergolina), agonisti PPAR-gamma (rosiglitazone o pioglitazone): 4 settimane;-Octreotide LAR, pasireotide LAR e Lanreotide Autogel:8 settimane;-Lanreotide SR: 4 settimane;-Octreotide e pasireotide (formulazione a rilascio immediato): 1 settimana;-Altre terapie sperimentali: almeno 5 emivite.

E.4

Principal exclusion criteria

1.Use of other investigational drugs at the time of enrollment, or within 30 days or 5 halflives of enrollment, whichever is longer; or longer if required by local regulations, and for any other limitation of participation in an investigational trial based on local regulations;2. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes;3. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases;4. Pregnant or nursing (lactating) women.;5. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are also using two acceptable methods of contraception,- Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural amenorrhea with an appropriate clinical profile or six months of spontaneous amenorrhea with serum FSH levels > 40 mIU/mL; or have had surgical bilateral oophorectomy or tubal ligation at least six months ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential;6. Fertile males, defined as all males physiologically capable of conceiving offspring UNLESS the subject agrees to comply with two effective contraceptive methods comprising a barrier method for the entire duration of the study, up to the Study Completion visit, and refrain from fathering a child for at least three (3) months following the last study drug administration;7. Patients who have been treated with mitotane during the last 6 months prior to Visit 1;8. Patients with compression of the optic chiasm, in order to exclude patients with a tumor causing chiasmal compression requiring surgery;9. Patients who have a known inherited syndrome as the cause for hormone over secretion (i.e. Carney Complex, McCune-Albright syndrome, MEN-1, API);10. Patients with Cushing’s syndrome due to ectopic ACTH secretion or adrenal Cushing’s syndrome;11. Patients with pseudo-Cushing’s syndrome (for patients with a mean UFC < 3xULN further testing to rule out this condition will be required unless Cushing’s disease is confirmed by histopathology);12. Patients with renal impairment (estimated creatinine clearance < 60 ml/min by the MDRD formula), serum creatinine >2.0 X ULN;13. Patients who are not biochemically euthyroid;14. Patients who have undergone major surgery within 1 month prior to screening;15. Diabetic patients with poorly controlled diabetes as evidenced by HbA1c >9%;16. Patients who have congestive heart failure (NYHA Class III or IV), unstable angina,sustained ventricular tachycardia, clinically significant bradycardia, advanced heart block,history of acute MI less than one year prior to study entry or clinically significant impairment in cardiovascular function;17. Patients with liver disease such as cirrhosis, chronic active hepatitis, or chronic persistent hepatitis, or patients with ALT/AST more than 3 X ULN, serum bilirubin >2.0 X ULN; 18. Patients who have any current or prior medical condition that can interfere with the conduct of the study or the evaluation of its results in the opinion of the investigator or the sponsor’s medical monitor; 19. Patients who have a history of alcohol or drug abuse in the 6 month period prior to Treatment;20. Patients with a history of non-compliance to medical regimens or who are considered potentially unreliable or will be unable to complete the entire study.

1.Utilizzo di altri farmaci sperimentali al momento dell’arruolamento, o entro 30 giorni o 5 emivite dall’arruolamento, a seconda di quale sia il periodo piu' lungo; o piu' a lungo se richiesto dalle normative locali e in conformita' a qualsiasi altro limite alla partecipazione ad uno studio clinico sulla base delle normative locali; 2.Anamnesi di ipersensibilita' al farmaco sperimentale o a farmaci di classi chimiche simili; 3.Anamnesi di tumore (ad eccezione di quello pituitario – malattia di Cushing – e del carcinoma cutaneo basocellullare in situ), trattato o non trattato, nei 5 anni precedenti, indipendentemente dalla presenza di evidenze di recidiva locale o metastatica;4.Donne in gravidanza o allattamento. Donne in grado di avere figli, definite come tutte le donne fisiologicamente in grado di iniziare una gravidanza, incluse le donne la cui carriera, stile di vita od orientamento sessuale precludono il rapporto sessuale con un partner di sesso maschile e le donne i cui partner sono stati sottoposti a sterilizzazione tramite vasectomia o altri metodi, A MENO CHE utilizzino due metodi contraccettivi adeguati (ad esempio, dispositivo intrauterino associato a preservativo, gel spermicida associato a preservativo, diaframma associato a preservativo, ecc.);5.Uomini fertili, definiti come tutti gli uomini fisiologicamente in grado di procreare A MENO CHE acconsentano ad utilizzare due metodi contraccettivi adeguati di cui uno sia un metodo di barriera (preservativo o diaframma con spermicida) per l’intera durata dello studio, fino alla visita di completamento dello studio, e ad astenersi dal procreare per almeno tre (3) mesi dopo l’ultima somministrazione di farmaco;6.Pazienti che sono stati trattati con mitotane nei 6 mesi precedenti la Visita 1;7.Pazienti con compressione del chiasma ottico che causa un qualsiasi difetto al campo visivo, al fine di escludere pazienti con un tumore che causi compressione chiasmatica e che richieda un intervento chirurgico; 8.Pazienti con sindrome ereditaria nota come causa della sovra-secrezione ormonale (cioe' complesso di Carney, sindrome di McCune-Albright, MEN-1, APIAIP (aryl hydrocarbon receptor interacting protein); 9.Pazienti con sindrome di Cushing dovuta a secrezione ectopica di ACTH o a sindrome di Cushing surrenale; 10.Pazienti con pseudo-sindrome di Cushing (per pazienti con UFC<3xULN medio sara' richiesto un ulteriore test per escludere questa condizione, a meno che la malattia di Cushing non sia confermata con istopatologia);11.Pazienti con insufficienza renale (clearance della creatinina stimata < 60 mL/min secondo la formula MDRD), creatinina sierica >2.0 X ULN, bilirubina sierica >2.0 X ULN, albumina sierica >1.5 X ULN;12.Pazienti non eutiroidei da un punto di vista biochimico;13.Pazienti che sono stati sottoposti a interventi chirurgici maggiori nel mese precedente lo screening;14.Pazienti diabetici in trattamento con farmaci antidiabetici il cui valore di glicemia a digiuno noncon diabete e' scarsamenteefficacemente controllato, come evidenziato da un valore HbA1C >9%.Per maggiori dettagli consultare i paragrafi 4.1 e 4.2 del protocollo originale.

E.5 End points

E.5.1

Primary end point(s)

The primary objective of the study is to assess the effects of 10 weeks treatment of LCI699 on UFC in patients with Cushing’s disease

L'obiettivo primario dello studio e' valutare gli effetti di 10 settimane di trattamento con LCI699 sull’UFC nelle 24 ore in pazienti con la malattia di Cushing.

E.5.1.1

Timepoint(s) of evaluation of this end point

10 weeks

10 settimane

E.5.2

Secondary end point(s)

Main secondary objective • To assess the safety and tolerability of multiple doses of LCI699 in patients with Cushing’s disease

Obiettivi secondario principale  Valutare la sicurezza e la tollerabilità di dosi multiple di LCI699 in pazienti con la malattia di Cushing

E.5.2.1

Timepoint(s) of evaluation of this end point

10 weeks

10 settimane

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

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

Yes

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

Information not present in EudraCT

E.8.1.3

Single blind

Information not present in EudraCT

E.8.1.4

Double blind

Information not present in EudraCT

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Information not present in EudraCT

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

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

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

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 study will complete when the last subject completes their Study
Completion visit, and any repeat assessments associated with this visit
have been documented and followed-up appropriately by the
Investigator.

Lo studio si completa quando l'ultimo soggetto completa la visita di conclusione dello studio, e le eventuali valutazioni di ripetere associata a questa visita sono stati documentati e follow-up adeguato dallo sperimentatore.

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	No
F.1.1	Number of subjects for this age range:	0
F.1.1.1	In Utero	Information not present in EudraCT
F.1.1.2	Preterm newborn infants (up to gestational age < 37 weeks)	Information not present in EudraCT
F.1.1.3	Newborns (0-27 days)	Information not present in EudraCT
F.1.1.4	Infants and toddlers (28 days-23 months)	Information not present in EudraCT
F.1.1.5	Children (2-11years)	Information not present in EudraCT
F.1.1.6	Adolescents (12-17 years)	Information not present in EudraCT
F.1.2	Adults (18-64 years)	Yes
F.1.3	Elderly (>=65 years)	Yes
F.2 Gender
F.2.1	Female	Yes
F.2.2	Male	Yes
F.3 Group of trial subjects
F.3.1	Healthy volunteers	No
F.3.2	Patients	Yes
F.3.3	Specific vulnerable populations	Yes
F.3.3.1	Women of childbearing potential not using contraception	No
F.3.3.2	Women of child-bearing potential using contraception	Yes
F.3.3.3	Pregnant women	No
F.3.3.4	Nursing women	No
F.3.3.5	Emergency situation	No
F.3.3.6	Subjects incapable of giving consent personally	No
F.3.3.7	Others	No
F.4 Planned number of subjects to be included
F.4.1	In the member state	6
F.4.2	For a multinational trial
F.4.2.1	In the EEA	10
F.4.2.2	In the whole clinical trial	15

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

2011-07-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-04-14

P. End of Trial
P.	End of Trial Status	Completed

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IMP with orphan designation in the indication
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