Clinical Trials Register

Summary
EudraCT Number:	2006-000880-27
Sponsor's Protocol Code Number:	CLBH589B2201
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-04-13
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

Spain - AEMPS

A.2

EudraCT number

2006-000880-27

A.3

Full title of the trial

A phase II study of oral LBH589 in adult patients with refractory cutaneous T-Cell lymphoma

Estudio fase II de LBH589 oral, en pacientes adultos con linfoma cutáneo de células T refractario

A.3.1

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

Study of Oral LBH589 in Adult Patients With Refractory Cutaneous T-Cell Lymphoma

Estudio de LBH589 en pacientes adultos con linfoma cutáneo de células T refractario

A.4.1

Sponsor's protocol code number

CLBH589B2201

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT00425555

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 Farmacéutica, S.A.
B.1.3.4	Country	Spain
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 Farmacéutica, S.A.
B.5.2	Functional name of contact point	Javier Malpesa
B.5.3	Address:
B.5.3.1	Street Address	ran Via de les Corts Catalanes 764
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08013
B.5.3.4	Country	Spain
B.5.4	Telephone number	+34933064464
B.5.5	Fax number	+34933064290
B.5.6	E-mail	eecc.novartis@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.3.1	Product name	panobinostat
D.3.2	Product code	LBH589
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	panobinostat
D.3.9.1	CAS number	404950-80-7
D.3.9.2	Current sponsor code	LBH589
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	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.3.1	Product name	panobinostat
D.3.2	Product code	LBH589
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	panobinostat
D.3.9.1	CAS number	404950-80-7
D.3.9.2	Current sponsor code	LBH589
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	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

Adult patients with stages IB to IVA mycosis fungoides (MF) or Sézary syndrome (SS). Patients with any history of visceral involvement of their CTCL will not be eligible for this study.
Patients with Sézary syndrome and bone marrow involvement are eligible. All patients must have received at least two prior systemic treatment regimens for their MF or SS.

Pacientes adultos con estadíos IB a IVA de micosis fungoide (MF) o síndrome de Sézary (SS). Los pacientes con algún antecedente de afectación visceral de su LCCT no serán elegibles para este estudio. Los pacientes con síndrome de Sézary y afectación de la médula ósea son elegibles. Todos los pacientes deberán haber recibido por lo menos dos regímenes sistémicos de tratamientos previos para su MF o SS

E.1.1.1

Medical condition in easily understood language

Refractory cutaneous T-Cell Lymphoma

Linfomas cutáneos de células T refractarios

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	14.1
E.1.2	Level	LLT
E.1.2	Classification code	10011679
E.1.2	Term	Cutaneous T-cell lymphoma refractory
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

To determine the response rate of patients with refractory CTCL treated with oral LBH589 as determined by using the modified Severity-Weighted Assessment Tool (mSWAT) to assess skin disease and the combined evaluation of disease in the viscera, and lymph nodes.

The primary objectives listed above have been met and results have been reported. The primary purpose of the study as of amendment 6 is to provide treatment to and to monitor and assess safety in the remaining ongoing patients in the trial who in the opinion of the investigator are continuing to derive clinical benefit.

Determinar la tasa de respuesta de pacientes con LCCT refractario tratados con LBH589 oral, determinada utilizando la herramienta de evaluación ponderada de la severidad modificada (mSWAT) para evaluar la enfermedad cutánea y la evaluación combinada de la enfermedad en vísceras y ganglios linfáticos.
Los objetivos principales anteriores, fueron conseguidos y los resultados publicados. El objetivo principal del estudio tras la enmienda 6, es proporcionar tratamiento y monitorizar y evaluar la seguridad de los pacientes que continuan en el estudio, que ne la opinión del investigador, siguen obteniendo beneficio clínico.

E.2.2

Secondary objectives of the trial

Response rate using the modified Severity-Weighted Assessment Tool (mSWAT); Response rate using a composite of mSWAT to assess skin disease and the combined evaluation of disease in the viscera, lymph nodes and blood (circulating SS cells); Response rate using the Physician?s Global Assessment of Clinical Condition (PGA); Responses in index lesions by lesion measurements and with photographic supporting documentation; To determine the overall response rate of patients with resistant CTCL treated with oral LBH589 by using the Physician?s Global Assessment (PGA) to assess skin disease and the evaluation of disease in the viscera, lymph nodes and blood (circulating SS cells); Improvement in CTCL-related symptoms and patient-reported outcomes; Duration of response; Time to response; Progression-free survival; Safety and tolerability; Pharmacokinetic (PK) profile

Determinar en pacientes con LCCT refractario, la(s):Tasa de respuesta utilizando la herramienta de evaluación ponderada de la severidad modificada (mSWAT); Tasa de respuesta utilizando un compuesto de la mSWAT para evaluar la enfermedad cutánea y la evaluación combinada de la enfermedad en vísceras, ganglios linfáticos y sangre (células SS circulantes); Tasa de respuesta utilizando la evaluación global por parte del médico de la condición clínica (PGA); Respuestas de las lesiones índice con medición de la lesión y con documentación fotográfica de apoyo; Determinar la tasa de respuesta global de pacientes con LCCT refractario tratados con LBH589 oral, determinada utilizando la evaluación global por parte del médico (PGA) para evaluar la enfermedad cutánea y la evaluación de la enfermedad en las vísceras, ganglios linfáticos y sangre (células SS circulantes); Mejoría de los síntomas relacionados con el LCCT y los resultados notificados por el paciente;

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent obtained prior to any screening procedures
2. Age ? 18 years old
3. Patients with biopsy-confirmed stages IB-IVA mycosis fungoides or Sézary syndrome. Patients with SS who have bone marrow involvement are also eligible. Patients with transformed CTCL are eligible. Disease stage for eligibility is based on the stage at time of study enrollment. NOTE: patients with any history of visceral involvement due to CTCL (i.e. stage IVB disease) are not eligible for this study.
4. Patients must have received at least two prior systemic therapy regimens. Systemic regimens include oral bexarotene, PUVA, photophoresis, chemotherapy such as methotrexate, and interferon. Topical steroids alone are not considered as a treatment regimen.
5. Patients must have had disease progression on or following their most recent treatment regimen. Patients are also eligible if they had an inadequate response to their most recent treatment regimen defined as stable disease as the best response after at least 3 months of therapy.
6. Patients will be accrued to one of two groups:
Group 1: Patients previously treated with oral bexarotene. This group includes patients who had:
? Disease progression on following treatment oral bexarotene, OR
? An inadequate response to oral bexarotene treatment defined as stable disease as the best response after at least 3 months of treatment, OR
? Intolerance of oral bexarotene defined as patients who discontinued oral bexarotene treatment due to adverse events.
Group 2: Patients who have not had prior oral bexarotene treatment.

1. Consentimiento informado por escrito obtenido antes de cualquier procedimiento de selección
2. Edad ? 18 años
3. Pacientes con micosis fungoide o síndrome de Sézary en estadío IB-IVA confirmado con biopsia. Los pacientes con SS que presenten afectación de la médula ósea también son elegibles. Los pacientes con LCCT transformado son elegibles. El estadío de la enfermedad para la elegibilidad se basará en el estadío en el momento de la inclusión en el estudio. NOTA: Los pacientes con cualquier antecedente de afectación visceral debido al LCCT (es decir, enfermedad en estadío IVB) no son elegibles para este estudio.
4. Los pacientes deberán haber recibido por lo menos dos regímenes de terapia sistémica. Los regímenes sistémicos incluyen bexaroteno oral, PUVA, fotoféresis, quimioterapia como metotrexato e interferón. Los esteroides tópicos en monoterapia no se consideran un régimen de tratamiento.
5. Los pacientes deberán haber presentado progresión de la enfermedad durante o tras su régimen de tratamiento más reciente. Los pacientes también son elegibles si presentaron una respuesta insuficiente a su régimen de tratamiento más reciente, definido como enfermedad estable como la mejor respuesta después de por lo menos 3 meses de terapia.
6. Los pacientes se incluirán en uno de los dos siguientes grupos:
?Grupo 1: Pacientes tratados previamente con bexaroteno oral. Este grupo incluye pacientes que presentaron:
?Progresión de la enfermedad durante o después del tratamiento con bexaroteno oral, O
?Una respuesta insuficiente al tratamiento con bexaroteno oral, definida como enfermedad estable como la mejor respuesta después de por lo menos 3 meses de tratamiento, O
?Intolerancia al bexaroteno oral, definido como pacientes que suspendieron el tratamiento oral con bexaroteno debido a acontecimientos adversos.
Grupo 2: Pacientes que no hayan recibido tratamiento previo con bexaroteno oral.

E.4

Principal exclusion criteria

1. Prior treatment with an HDAC inhibitor for CTCL.
2. Patients with visceral disease including CNS involvement (i.e. stage IVB CTCL). Note; Patients with SS who have bone marrow involvement are eligible.
3. Patients who have been previously treated with LBH589.
4. Impaired cardiac function
5. Concomitant use of drugs with a risk of causing torsades de pointes
6. Patients who have received chemotherapy or any investigational drug or undergone major surgery ?3 weeks prior to starting study drug or who have not recovered from side effects of such therapy
7. Less than 3 months since prior electron beam therapy
8. Women who are pregnant or breast feeding, or women of childbearing potential (WOCBP) not willing to use a double method of contraception during the study and 3 months after the end of treatment. One of these methods of contraception must be a barrier method. WOCBP are defined as sexually mature women who have not undergone a hysterectomy or who have not been naturally postmenopausal for at least 12 consecutive months (i.e., who has had menses any time in the preceding 12 consecutive months). Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at baseline.
9. Male patients whose sexual partners are WOCBP not using a double method of contraception during the study and 3 months after the end of treatment. One of these methods of contraception must be a condom.

1. Tratamiento previo con un inhibidor de HDAC para el LCCT.
2. Pacientes con enfermedad visceral incluyendo afectación del SNC (es decir, LCCT en estadío IVB). Nota: los pacientes con SS que presenten afectación de la médula ósea son elegibles.
3. Pacientes que hayan sido tratados previamente con LBH589.
4. Deterioro de la función cardíaca.
5. Uso concomitante de fármacos con un riesgo de producir torsades de pointes
6. Pacientes que hayan recibido quimioterapia o cualquier fármaco en investigación o que hayan sido sometidos a cirugía mayor ? 3 semanas antes del inicio de la medicación del estudio o que no se hayan recuperado de los efectos secundarios de dicha terapia.
7. Menos de 3 meses desde la terapia previa con haz de electrones
8. Mujeres embarazadas o en periodo de lactancia, o mujeres físicamente fértiles (WOCBP) que no deseen utilizar un método anticonceptivo doble durante el estudio y 3 meses después del final del tratamiento. Uno de estos métodos anticonceptivos deberá ser un método de barrera. Las WOCBP se definen como mujeres sexualmente maduras que no hayan sido sometidas a histerectomía o que no sean postmenopáusicas durante por lo menos 12 meses consecutivos (es decir, sin ninguna menstruación en los 12 meses precedentes consecutivos). Las mujeres físicamente fértiles (WOCBP) deberán presentar una prueba de embarazo en suero con resultados negativos en la visita basal
9. Pacientes varones cuyas parejas sexuales sean WOCBP, que no utilicen un método anticonceptivo doble durante el estudio y 3 meses después del final del tratamiento. Uno de estos métodos anticonceptivos deberá ser un preservativo.

E.5 End points

E.5.1

Primary end point(s)

- mSWAT to assess skin disease
- The evaluation of disease in the viscera or lymph nodes by CT scan
- Blood analysis for Sézary cells
- Patient reported outcomes.

As of this amendment, there will be no formal efficacy assessment requirements or analyses. Investigators will follow and assess efficacy as per SOC for patients with CTCL.

SWAT para evaluar la enfermedad cutánea
La evaluación de la enfermedad en las vísceras o ganglios linfáticos con TAC
Evaluación sanguínea de las células de Sézary
Resultados notificados por el paciente
Tras esta enmienda, no habrá una evaluación formal de los requerimientos o análisis de la eficacia. Los investigadores seguiran y evaluaran la eficacia en función del estandar de tratamiento de los pacientes con LCCT.

E.5.1.1

Timepoint(s) of evaluation of this end point

Investigators will follow and assess efficacy as per SOC for patients with CTCL. Investigators will note if disease progression or death on study treatment occurs on the mSWAT assessment and EOT pages respectively

Los investigadores seguiran y evaluaran la eficacia en función del estandar de tratamiento de los pacientes con LCCT.
Los investigadores anotaran la progresión de la enfermedad o muerte durante el tratamiento del estudio, en las páginas de evaluación de SWAT o EOT respectivamente.

E.5.2

Secondary end point(s)

As of this amendment, laboratory and other safety assessments will be reduced to that which is appropriate to adequately monitor and protect patient safety. Thyroid function and coagulation will be assessed per SOC. Assessment of ECGs will be performed locally only. Please also refer to Section 7 of the protocol for an updated safety assessment schedule.

Tras esta enmienda, las evaluaciones de laboratorio y otras evaluaciones de seguridad, se reducirán a aquellas que son necesarias para monitorizar y proteger la seguridad del paciente adecuadamente. La función tiroidea y coagulación serán evaluadas segun el estandar de tratamiento. La evaluación de los ECGs se hará sólo localmente. Por favor, referir a la sección 7 del protocolo para una actualización del esquema de la evaluación de seguridad.

E.5.2.1

Timepoint(s) of evaluation of this end point

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

Yes

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

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

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)

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.8.3

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

Argentina

Australia

Belgium

Canada

Finland

France

Germany

Hungary

Italy

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

Under this amendment patients who are still on treatment and are demonstrating clinical
benefit with good tolerability of panobinostat will continue to receive treatment until their disease progresses, or they develop intolerability to treatment, withdraw consent or alternative drug access is available.

Tras esta enmienda, los pacientes que sigan en tratamiento y que estén demostrando un beneficio clínico y una buena tolerancia al panobinostat, continuarán recibiendo el tratamiento hasta que su enfermedad progrese, o desarrollen intolerancia, o retiren el consentimiento o tengan acceso a una terapia alternativa.

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

F.4.2.2

In the whole clinical trial

139

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)

see protocol

Ver protocolo

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

2007-02-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2006-11-09

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

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