Clinical Trials Register

Summary
EudraCT Number:	2010-024000-10
Sponsor's Protocol Code Number:	101MS206
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:	2011-05-26
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Spain - AEMPS

A.2

EudraCT number

2010-024000-10

A.3

Full title of the trial

A Randomized, Blinded, Parallel-Group, Phase 2 Study Exploring the Safety, Tolerability, and Efficacy of Multiple Regimens of Natalizumab in Adult Subjects With Relapsing Multiple Sclerosis

Estudio aleatorizado, ciego, con grupo paralelo en fase 2 en el que se explora la seguridad, tolerabilidad y eficacia de pautas posológicas múltiples de Natalizumab en sujetos adultos con esclerósis múltiple recidivante

A.3.1

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

A Study to Evaluate the Effect of Different Doses of TYSABRI on Safety and Efficacy in Relapsing Multiple Sclerosis

Estudio para evaluar para la seguridad y la eficacia en el efecto de diferentes dosis de TYSABRI en la esclerosis múltiple recidivante

A.3.2

Name or abbreviated title of the trial where available

REFINE

A.4.1

Sponsor's protocol code number

101MS206

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	Biogen Idec Limited
B.1.3.4	Country	United Kingdom
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	Biogen Idec Limited
B.4.2	Country	United Kingdom
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Biogen Idec Limited
B.5.2	Functional name of contact point	Not available
B.5.3	Address:
B.5.3.1	Street Address	Innovation House, 70 Norden Road
B.5.3.2	Town/ city	Maidenhead
B.5.3.3	Post code	SL6 4AY
B.5.3.4	Country	United Kingdom
B.5.6	E-mail	neurologyclinicaltrial@biogenidec.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	Yes
D.2.1.1.1	Trade name	TYSABRI
D.2.1.1.2	Name of the Marketing Authorisation holder	Elan Pharma International Ltd.
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Natalizumab for IV Infusion
D.3.2	Product code	AN100226; BG00002
D.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NATALIZUMAB
D.3.9.1	CAS number	189261-10-7
D.3.9.2	Current sponsor code	AN100226; BG00002
D.3.9.4	EV Substance Code	SUB22282
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	300
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	Natalizumab for Subcutaneous Injection
D.3.2	Product code	AN100226; BG00002
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NATALIZUMAB
D.3.9.1	CAS number	189261-10-7
D.3.9.2	Current sponsor code	AN100226; BG00002
D.3.9.4	EV Substance Code	SUB22282
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	150
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	Solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Relapsing-Remitting Multiple Sclerosis

Esclerósis múltiple recidivante

E.1.1.1

Medical condition in easily understood language

Multiple sclerosis characterized by unpredictable relapses followed by
periods with no new signs of disease activity

La esclerosis múltiple se caracteriza por recaídas imprevisibles, seguido de períodos sin nuevos signos de actividad de la enfermedad

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	13.1
E.1.2	Level	PT
E.1.2	Classification code	10063399
E.1.2	Term	Relapsing-remitting multiple sclerosis
E.1.2	System Organ Class	10029205 - Nervous system 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 explore the effects of multiple regimens of natalizumab on disease
activity and safety in subjects with relapsing remitting MS.

El objetivo principal de este estudio es explorar los efectos de múltiples pautas posológicas de natalizumab sobre la actividad de la enfermedad y la seguridad en sujetos con EM remitente recurrente

E.2.2

Secondary objectives of the trial

• To characterize and compare PK/PD profiles across multiple dose
regimens of natalizumab.
• To characterize effects on secondary brain MRI measures.
• To evaluate potential laboratory markers of immune function and
trafficking across the treatment regimens.
• To assess the safety and tolerability across multiple dose regimens

Caracterizar y comparar los perfiles FC/FD en múltiples pautas posológicas de natalizumab.
Caracterizar los efectos sobre las medidas secundarias de la RMN cerebral. Evaluar los posibles
marcadores de laboratorio de la actividad y el tráfico inmunitarios en las diferentes pautas posológicas.
Evaluar la seguridad y la tolerabilidad en múltiples pautas posológicas

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Ability to understand the purpose and risks of the study and provide
signed and dated informed consent and authorization to use protected
health information (PHI) in accordance with national and local subject
privacy regulations.
2. Aged 18 to 55 years old, inclusive, at the time of informed consent.
3. Subjects of childbearing potential must practice effective
contraception during the study.
4. A documented diagnosis of RRMS.
5. Free of MS relapse, as determined by the enrolling Investigator, for 12
months prior to randomization.
6. Treatment with natalizumab according to locally approved pescribing
information for a minimum of the 12 months immediately prior to
randomization. The subject must have received at least 11 doses of
natalizumab in the 12 months prior to randomization with no missed doses in the 3 months prior to randomization.
7. In the 12 months prior to the initiation of natalizumab, subject must
have experienced a minimal level of disease activity as defined by:
• 2 or more documented clinical relapses
OR
• 1 relapse and documented MRI activity, defined by the presence of at
least one Gd enhancing lesion on MRI, unrelated to the relapse

1. Capacidad de comprender el objetivo y los riesgos del estudio y de dar el consentimiento informado firmado y fechado y la autorización para usar información de salud protegida (Protected Health Information, PHI) de acuerdo con la normativa nacional y local sobre la privacidad de los sujetos. 2. Edad de entre 18 y 55 años, ambos inclusive, en el momento del consentimiento informado. 3. Los
sujetos en edad fértil deben tomar medidas anticonceptivas eficaces durante el estudio 4. Diagnóstico documentado de EMRR. 5. Ausencia de recaída de la EM, determinada por el investigador que realiza
la inscripción, en los 12 meses previos a la aleatorización. 6. Tratamiento con natalizumab de acuerdo con la información de la ficha técnica aprobada localmente al menos durante los 12 meses
inmediatamente anteriores a la aleatorización. El sujeto debe haber recibido un mínimo de 11 dosis de natalizumab en los 12 meses previos a la aleatorización y no haberse saltado ninguna en los 3 meses previos a la aleatorización. 7. En los 12 meses previos al inicio del natalizumab, el sujeto debe haber experimentado un grado mínimo de actividad de la enfermedad, definido por: 2 o más recaídas clínicas documentadas O 1 recaída y actividad documentada en la RMN, definida como la presencia de al menos una lesión potenciada con Gd en la RMN no relacionada con la recaída

E.4

Principal exclusion criteria

1. Known history of Human Immunodeficiency Virus (HIV).
2. Known history of hepatitis C (test for hepatitis C virus antibody [HCV
Ab]) or hepatitis B virus (test for Hepatitis B Surface Antigen [HBsAg]
and/or Hepatitis B Core Antibody [HBcAb]).
3. Positive for anti-natalizumab antibodies at Screening.
4. MRI positive for Gd-enhancing lesions at study entry.
5. Subjects for whom MRI is contraindicated, e.g., have a pacemaker or
other contraindicated implanted metal devices, have suffered, or are at
risk for, side effects from Gd, or have claustrophobia that cannot be
medically managed.
6. History of any clinically significant (as determined by the
Investigator) cardiac, endocrinologic, hematologic, hepatic,
immunologic, metabolic (including diabetes), urologic, pulmonary,
neurologic (except for RRMS), dermatologic, psychiatric, renal, or
other major disease that would preclude participation in a clinical study.
7. History of malignant disease, including solid tumors and hematologic
malignancies (with the exception of basal cell and squamous cell
carcinomas of the skin that have been completely excised and are
considered cured).
8. History of transplantation or any anti-rejection therapy.
9. History of severe allergic or anaphylactic reactions or known
hypersensitivity to any drug.
10. A clinically significant infectious illness (e.g., cellulitis, abscess,
pneumonia, septicemia) within 30 days prior to Screening, or PML or
other opportunistic infections at any time.
11. Signs or symptoms suggestive of any serious infection, based on
medical history, physical examination, or laboratory testing, as
determined by the Investigator.
MS Treatment History
12. Prior treatment with total lymphoid irradiation.
13. Prior treatment with cladribine, mitoxantrone, fingolimod, T-cell or
T-cell receptor vaccination, cyclophosphamide, cyclosporine,
azathioprine, methotrexate, mycophenolate mofetil, or any therapeutic
monoclonal antibody other than natalizumab within 24 months
prior to randomization.
14. Prior treatment with intravenous immunoglobulin (IVIg),
plasmapheresis, or cytapheresis within 12 months prior to
randomization.
15. Treatment with IV or oral corticosteroids (topical corticosteroids are
acceptable) or related products within 3 months prior to randomization.
Miscellaneous
16. Female subjects considering becoming pregnant while in the study.
17. Female subjects who are pregnant or currently breastfeeding.
18. History of drug or alcohol abuse within 2 years prior to entry per
Investigator judgment.
19. Unwillingness or inability to comply with the requirements of this
protocol including the presence of any condition (physical, mental, or
social) that is likely to affect the subject's ability to comply with the study protocol.
20. Receiving any other investigational treatment within the 12 months
prior to Screening or concurrent with this study.
21. Any pre-scheduled elective procedure during the study period that,
in the opinion of the Investigator, would interfere with study endpoints.
22. Any other condition, clinical finding, or reason that, in the opinion of
the Investigator and/or the Sponsor, makes the subject unsuitable for
enrollment into this study.
23. Previous participation in this study at randomization

1. Antecedentes conocidos de infección por el virus de la inmunodeficiencia humana (VIH). 2. Antecedentes conocidos de infección por el virus de la hepatitis C [prueba de detección de anticuerpos para el virus de la hepatitis C (Ac VHC)] o el virus de la hepatitis B [prueba de detección del antígeno de superficie del virus de la hepatitis B (HBsAg) y/o anticuerpos del antígeno nuclear del virus de la hepatitis B (HBcAb)]. 3. Anticuerpos anti-natalizumab positivos en la selección. 4. RMN positiva para lesiones potenciadas con Gd en la inscripción en el estudio. 5. Sujetos en los que la RMN está contraindicada, por ejemplo, que tienen un marcapasos u otros dispositivos metálicos implantados contraindicados, presentan riesgo de experimentar efectos secundarios del Gd o sufren claustrofobia que
no puede tratarse médicamente. 6. Antecedentes de cualquier enfermedad clínicamente significativa (según determine el investigador) cardíaca, endocrinológica, hematológica, hepática, inmunitaria, metabólica (incluyendo la diabetes), urológica, pulmonar, neurológica (excepto la EMRR), dermatológica, psiquiátrica, renal u otra enfermedad importante que impida participar en un estudio
clínico. 7. Antecedentes de cáncer, incluyendo tumores sólidos y cánceres hematológicos (con la excepción de los carcinomas basocelulares y de células escamosas de la piel que se han extirpado por completo y se consideran curados). 8. Antecedentes de trasplante o tratamiento antirrechazo. 9. Antecedentes de reacciones alérgicas o anafilácticas graves o hipersensibilidad conocida a cualquier
fármaco. 10. Enfermedad infecciosa clínicamente significativa (por ejemplo, celulitis, absceso, neumonía, septicemia) en los 30 días previos a la selección o LMP u otras infecciones oportunistas en
cualquier momento. 11. Signos o síntomas indicativos de cualquier infección grave, en base al historial médico, la exploración física o las pruebas de laboratorio, según determine el investigador. Tratamiento
previo de la EM 12. Tratamiento previo con irradiación linfoide total. 13. Tratamiento previo con cladribina, mitoxantrona, fingolimod, vacuna con células T o receptores de células T, ciclofosfamida,
ciclosporina, azatioprina, metotrexato, micofenolato mofetil o cualquier anticuerpo monoclonal terapéutico distinto del natalizumab en los 24 meses previos a la aleatorización 14. Tratamiento previo
con inmunoglobulina intravenosa (Ig i.v.), plasmaféresis o citaféresis en los 12 meses previos a la aleatorización. 15. Tratamiento con corticoesteroides i.v. u orales (los corticoesteroides tópicos son
aceptables) o productos relacionados en los tres meses previos a la aleatorización. Varios 16. Sujetos del sexo femenino que se estén planteando quedarse embarazadas durante el estudio. 17. Sujetos del sexo femenino que estén embarazadas o amamantando. 18. Antecedentes de abuso de drogas o alcohol en los dos años previos a la inscripción en el estudio, a criterio del investigador. 19. Falta de voluntad o incapacidad de cumplir con los requisitos de este protocolo, incluyendo la presencia de cualquier enfermedad (física, mental o social) que probablemente afecte la capacidad del sujeto de cumplir con el protocolo del estudio. 20. Recepción de cualquier otro tratamiento en investigación en los 12 meses previos a la selección o de manera simultánea a este estudio. 21. Cualquier intervención quirúrgica programada previamente durante el estudio que, en opinión del investigador, interferiría en los criterios de valoración del estudio. 22. Cualquier otra enfermedad, hallazgo clínico o motivo que, en opinión del investigador y/o el patrocinador, hace que el sujeto no sea apto para su inscripción en este estudio. 23. Participación previa en este estudio en la aleatorización.

E.5 End points

E.5.1

Primary end point(s)

Cumulative number of combined unique active lesions (sum of the
number of new Gd enhancing and new or newly enlarging T2
hyperintense lesions not associated with Gd enhancement on T1
weighted scans) based on brain MRI scans

Número acumulado de lesiones activas únicas nuevas (suma de lesiones Gd+ nuevas y de lesiones hiperintensas en T2 nuevas o de nuevo crecimiento no asociadas a imágenes en T1 potenciadas con Gd) según las RMN cerebrales

E.5.1.1

Timepoint(s) of evaluation of this end point

At weeks 12, 24, 36, 48 and 60.

En las semanas 12, 24, 36, 48 y 60.

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

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

Yes

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

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

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

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 end of study is last subject, last visit for final collection of data

El fin del estudio es la última visita del último sujeto para la obtención de datos final

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	No
F.1.1.1.1	Number of subjects for this age range:	0
F.1.1.2	Preterm newborn infants (up to gestational age < 37 weeks)	No
F.1.1.2.1	Number of subjects for this age range:	0
F.1.1.3	Newborns (0-27 days)	No
F.1.1.3.1	Number of subjects for this age range:	0
F.1.1.4	Infants and toddlers (28 days-23 months)	No
F.1.1.4.1	Number of subjects for this age range:	0
F.1.1.5	Children (2-11years)	No
F.1.1.5.1	Number of subjects for this age range:	0
F.1.1.6	Adolescents (12-17 years)	No
F.1.1.6.1	Number of subjects for this age range:	0
F.1.2	Adults (18-64 years)	Yes
F.1.2.1	Number of subjects for this age range:	300
F.1.3	Elderly (>=65 years)	No
F.1.3.1	Number of subjects for this age range:	0
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	50
F.4.2	For a multinational trial
F.4.2.1	In the EEA	300
F.4.2.2	In the whole clinical trial	300

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

2011-08-08

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-07-27

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2014-07-18

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