Clinical Trials Register

Summary
EudraCT Number:	2015-003614-24
Sponsor's Protocol Code Number:	CLTT462X2101
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:	2020-11-04
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

2015-003614-24

A.3

Full title of the trial

A phase I dose finding study of oral LTT462 in adult patients with advanced solid tumors harboring MAPK pathway alterations

A phase I dose finding study of oral LTT462 in adult
patients with advanced solid tumors harboring MAPK
pathway alterations

A.3.1

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

A phase I clinical study with investigational compound LTT462 in adult patients with specific advanced cancers

Studio di Fase I, di valutazione della dose, con LTT462, somministrato per via orale, in pazienti adulti con tumori solidi in stadio avanzato che presentano alterazioni della via MAPK

A.3.2

Name or abbreviated title of the trial where available

A phase I clinical study with investigational compound LTT462 in adult patients with specific advanc

Studio di Fase I, di valutazione della dose, con LTT462, somministrato per via orale, in pazienti ad

A.4.1

Sponsor's protocol code number

CLTT462X2101

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 PHARMA SERVICES AG
B.1.3.4	Country	Switzerland
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 SERVICES AG
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 SpA
B.5.2	Functional name of contact point	DRA office
B.5.3	Address:
B.5.3.1	Street Address	l.go 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	0296542862
B.5.5	Fax number	029659066
B.5.6	E-mail	info.studiclinici@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	LTT462
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	LTT462
D.3.9.4	EV Substance Code	SUB178983
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	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 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.3.1	Product name	LTT462 20 mg
D.3.2	Product code	LTT462
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	LTT462
D.3.9.4	EV Substance Code	SUB178983
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	20
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	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	LTT462 50 mg
D.3.2	Product code	LTT462
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	LTT462
D.3.9.4	EV Substance Code	SUB178983
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	50
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: 4
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	LTT 462 100 mg
D.3.2	Product code	LTT462
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	LTT462
D.3.9.4	EV Substance Code	SUB178983
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	100
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

Adult and adolescent patients with advanced solid tumors harboring MAPK pathway alterations

Pazienti adulti ed adolescenti con tumori solidi in stadio avanzato che presentano alterazioni della via MAPK

E.1.1.1

Medical condition in easily understood language

Adult and adolescent with advanced cancer whose harbor genetic alterations in the MAPK pathway (MAPK pathway helps the cells to work normally. If this pathway is altered cells may grow abnormally)

Pazienti adulti ed adolescenti con tumore avanzato che presentano specifiche alterazioni genetiche nella via MAPK (l'attivazione di MAPK aiuta le cellule a funzionare normalmente. Se questa via
risult

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	10048683
E.1.2	Term	Advanced cancer
E.1.2	System Organ Class	100000004864

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 characterize safety and tolerability of LTT462 and identify a recommended dose and regimen for future studies in adult and adolescent patients with advanced solid tumors harboring MAPK pathway alterations.

Valutare la sicurezza d¿impiego e la tollerabilit¿ di LTT462 e identificare la dose e il regime
raccomandati per gli studi futuri in pazienti adulti ed adolescenti con tumori solidi in stadio avanzato che presentano alterazioni della via MAPK.

E.2.2

Secondary objectives of the trial

- To evaluate the preliminary anti-tumor activity of LTT462
- To evaluate the pharmacokinetic (PK) profile of LTT462
- To assess the pharmacodynamic (PD) effect of LTT462

Obiettivi secondari: ¿Valutare l¿attivit¿ antitumorale preliminare di LTT462
¿Valutare il profilo farmacocinetico di LTT462
¿Valutare l¿effetto farmacodinamico di LTT462

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

-Patient (male or female) =12 years of age
- ECOG (Eastern Cooperative Oncology Group) performance status =1
- Must have progressed following standard therapy, or for whom, in the opinion of the Investigator, no effective standard therapy
exists, is tolerated or appropriate.
-Patients must have a site of disease amenable to biopsy and be a
candidate for tumor biopsy. Patients must be willing to undergo a new
tumor biopsy at screening/baseline and during therapy.
- Patients must be willing to undergo study required biopsies.
- Presence of at least one measurable lesion according to RECIST v1.1.
- Documented MAPK Pathway alteration
Other protocol-defined inclusion criteria may apply

- Pazienti di entrambi i sessi ed età = 12 anni.
- ECOG (Eastern Cooperative Oncology Group) performance status < 1.
- Tutti i pazienti partecipanti a questo studio clinico devono aver manifestato progressione dopo terapia standard, o devono essere
pazienti per i quali, secondo l’opinione dello sperimentatore, non esista alcuna terapia standard efficace, la terapia standard non
sia tollerata o non sia appropriata.
-I pazienti devono presentare una sede di malattia che può essere sottoposta a biopsia ed essere candidati alla biopsia tumorale, in base alle linee-guida istituzionali del trattamento.
I pazienti devono essere disposti a sottoporsi a una nuova biopsia tumorale allo screening-basale e durante il trattamento
- I pazienti devono essere disposti a sottoporsi alle biopsie tumorali richieste dallo studio (se clinicamente fattibile), in base alle
linee-guida istituzionali e ai requisiti per questa procedura.
- Presenza di almeno una lesione misurabile in base a RECIST v1.1
- Documentata alterazione della via MAPK
Per gli altri criteri di inclusione fare riferimento al protocollo

E.4

Principal exclusion criteria

-- Prior treatment with ERK inhibitors.
- History or current evidence of retinal vein occlusion (RVO) or current risk factors for RVO.
- Any medical condition that would, in the investigator’s judgment, prevent the patient’s participation in the clinical study due to
safety concerns or compliance with clinical study procedures.
- Patients receiving proton pump inhibitors (PPI) which cannot be discontinued 3 days prior to the start study treatment and for the
duration of the study.
- Clinically significant cardiac disease
Other protocol-defined exclusion criteria may apply

Trattamento precedente con inibitori di ERK.
- Evidenza pregressa o attuale di occlusione della vena retinica (RVO) o presenza di fattori di rischio per RVO.
¿ Pazienti che ricevono un farmaco con un rischio noto di indurre torsione di punta che non può essere sospeso o sostituito da un farmaco alternativo sicuro
- Qualsiasi condizione medica che, secondo l’opinione dello sperimentatore, possa compromettere la partecipazione del paziente
allo studio clinico a causa di problemi di sicurezza o di compliance con le procedure dello studio.
- Pazienti in trattamento con inibitori della pompa protonica che non possono essere sospesi 3 giorni prima dell’inizio del trattamento in studio e durante il corso dello studio.
-Patologie cardiache clinicamente significative
- Pazienti con neoplasia maligna, diversa da quella trattata in questo studio. Eccezioni a questo criterio includono le seguenti condizioni: neoplasie trattate curativamente e che non hanno manifestato recidiva entro 2 anni prima della somministrazione del trattamento in studio, carcinoma cutaneo a cellule basali e a cellule squamose completamente escisso e carcinoma in situ di qualsiasi tipo completamente escisso.
Per gli altri criteri di esclusione fare riferimento al protocollo

E.5 End points

E.5.1

Primary end point(s)

Safety and tolerability as assessed by
incidence and severity of adverse events (AEs), dose interruptions, reductions and dose intensity
- Incidence and nature of dose limiting toxicities (DLTs) (dose escalation only)

Siicurezza e la tollerabilità valutata mediante l'incidenza e la gravità degli eventi avversi (AE),
interruzioni della dose, riduzione e intensità della dose
- Incidenza e natura di tossicità limitante la dose (DLT) (solo aumento della dose)

E.5.1.1

Timepoint(s) of evaluation of this end point

Cycle 1 day 1 until 30 days post study treatment (expected duration approximately 12 months)
1 cicle (28 days)

Ciclo 1 giorno 1 fino a 30 giorni dopo il trattamento dello studio (durata prevista di circa 12 mesi)
1 ciclo (28 giorni)

E.5.2

Secondary end point(s)

For both parts: Overall response rate (ORR), Disease Control Rate (DCR), Duration of Response (DOR), and Progression Free Survival (PFS)
Plasma concentration and derived PK parameters of LTT462 (AuC, Cmax, Tmax and T1/2)
- For dose expansion part only:
Overall Survival (OS)
Changes from baseline of the PD marker DUSP6 in tumor tissue and in blood

Tasso di risposta globale (ORR), Tasso di controllo della malattia (DCR), durata della risposta (DOR) e sopravvivenza libera da
progressione (PFS)
- Concentrazione plasmatica e parametri farmacocinetici derivati da LTT462 (AUC, Cmax, Tmax e T1 / 2)
- Solo per la parte di espansione della dose:
Sopravvivenza globale (OS)
Modifiche rispetto al basale della PD del marcatore DUSP6 nel tessuto tumorale e nel sangue

E.5.2.1

Timepoint(s) of evaluation of this end point

At protocol defined timepoints (PK, PD and preliminary anti-tumor activity end point until the end of the study

Ai timepoints definiti dal protocollo (PK, PD e l'attivit¿ antitumorale
preliminare fino alla 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

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)

Yes

E.7.1.1

First administration to humans

Yes

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)

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

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)

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

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

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

Canada

France

Germany

Italy

Japan

Netherlands

Singapore

Spain

Switzerland

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 end when the treatment period and 30-day safety follow-up have been completed for all patients, when survival follow-up (for expansion patients only) has additionally has been completed, or when the study is terminated early.

Lo studio terminer¿ quando il periodo di trattamento e i 30 giorni di sicurezza di follow-up saranno terminati per tutti i pazienti e quando sar¿ completato il follow up di sopravvivenza (solo per i pazienti della fase di espansione) o quando lo studio ¿ terminato in anticipo.

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

Yes

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

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

Yes

F.3.3.2

Women of child-bearing potential using contraception

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

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)

not applicable

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

2016-09-06

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-07-21

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

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Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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