Clinical Trials Register

Summary
EudraCT Number:	2014-003076-22
Sponsor's Protocol Code Number:	PSY-201401_ESPRIT
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2015-11-27
Trial 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

Germany - BfArM

A.2

EudraCT number

2014-003076-22

A.3

Full title of the trial

Multimodal Prevention of First Psychotic Episode – a 2x2-Factorial Randomized Trial investigating the efficacy of Acetylcysteine (ACC) and Integrated Preventive Psychological Intervention (IPPI) in Subjects Clinically at High Risk for Psychosis

Multimodale Prävention des ersten psychotischen Schubs- eine 2x2 faktorial randomisierte Untersuchung der Effektivität von Acetylcystein (ACC) und einer integrierten vorbeugenden psychologischen Intervention (IPPI) bei Personen mit einem erhöhten Risiko für Psychosen- Die ESPRIT B1 Studie

A.3.1

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

A.3.2

Name or abbreviated title of the trial where available

ESPRIT B1

A.4.1

Sponsor's protocol code number

PSY-201401_ESPRIT

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	Central Institute of Mental Health Mannheim (ZI)
B.1.3.4	Country	Germany
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Federal Ministry of Education and Research
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Studienzentrale des Studienzentrum Bonn (SZB)
B.5.2	Functional name of contact point	Dr. rer. nat. Christine Fuhrmann
B.5.3	Address:
B.5.3.1	Street Address	Venusberg-Campus 1
B.5.3.2	Town/ city	Bonn
B.5.3.3	Post code	53127
B.5.3.4	Country	Germany
B.5.4	Telephone number	004922816046
B.5.5	Fax number	004922816039
B.5.6	E-mail	Studienzentrale-SZB@ukbonn.de

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	Information not present in EudraCT
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	Acetylcysteine 500 mg capsules
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	ACETYLCYSTEINE
D.3.9.3	Other descriptive name	N-Acetylcystein (NAC)
D.3.9.4	EV Substance Code	SUB05229MIG
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	500
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Clinical High Risk state for developing a first psychotic episode

Menschen mit einem erhöhten Risiko an einem ersten psychotischen Schub zu erkranken.

E.1.1.1

Medical condition in easily understood language

Subjects Clinically at High Risk for Psychosis

Personen mit einem erhöhten Risiko für Psychosen

E.1.1.2

Therapeutic area

Psychiatry and Psychology [F] - Psychological processes [F02]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	SOC
E.1.2	Classification code	10037175
E.1.2	Term	Psychiatric disorders
E.1.2	System Organ Class	10037175 - Psychiatric disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10061920
E.1.2	Term	Psychotic disorder
E.1.2	System Organ Class	10037175 - Psychiatric 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 evaluate individual and combined preventive effects of a pharmaceutical intervention with glutamatergic, neuroprotective and anti-inflammatory capabili-ties (ACC) and an integrated preventive psychologcial intervention (IPPI) on transition rates to psychosis, on progression of symptoms and on improvement of social functioning.

Es sollen die individuellen und kombinierten präventiven Effekte von einer pharmazeutischen Intervention mit glutamatergen, neuroprotektiven und anti-inflammatorischen Eigenschaften (ACC) und einer integrierten vorbeugenden psychologischen Intervention (IPPI) auf die Entstehung von Psychosen, das Fortschreiten der Symptome und auf die Verbesserung der Gesellschaftsfähigkeit untersucht werden.

E.2.2

Secondary objectives of the trial

To evaluate individual and combined preventive effects of a pharmaceutical intervention with glutamatergic, neuroprotective and anti-inflammatory capabili-ties (ACC) and an integrated preventive psychological intervention (IPPI) on improvement of symptoms social cognition and neuropsychological perfor-mance. Tolerability will also be evaluated.

Es sollen die individuellen und kombinierten präventiven Effekte von einer pharmazeutischen Intervention mit glutamatergen, neuroprotektiven und anti-inflammatorischen Eigenschaften (ACC) und einer integrierten vorbeugenden psychologischen Intervention (IPPI) auf die Verbesserung von Symptomen, sozialer Kognition und neuropsychologischer Leistungsfähigkeit untersucht werden.

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

MRI
MRS
Experience sampling (EMA)
Health Economy Analysis (D1, workpackage)
RDoC
Mikrobiom
MRS/haemotology
Genetics
All sub-studies do not involve an IMP and their related objectives are integrated into the Trial protocol (Version 1.0, 24.11.2015)

E.3

Principal inclusion criteria

•Age 18 – 40 years
•Subjects with the ability to follow study instructions and likely to attend and complete all required visits
•Written informed consent of the subject
•Subjects are able to speak, write and understand the German lan-guage sufficiently well (at the investigators discretion) to complete all required study procedures
•Subjects show impaired social functioning skills as measured by the Global Assessment of Functioning-Scale (Social) (GFS ≤ 7)
•Clinical High Risk Criteria
Ultra-high risk criteria (Attenuated Positive Symptoms and/or Brief Limited Intermittend Psychotic Symptoms and/or a com-bination of familial risk or schizotypal disorder with a significant loss of functioning; assessed by the Structured Interview for Prodromal Syndromes, SIPS 5.0)

and/or

The Basic Symptom Criterion 'Cognitive Disturbances, COGDIS' (2/9 cognitive-perceptive basic symptoms; assessed by the Schizophrenia Proneness Instrument – Adult Version, SPI-A)

•Patienten müssen ≥ 18 Jahre und < 40 Jahre alt sein
•Schriftlich dokumentierte Einwilligungserklärung und Einwilligung zur Teilnahme an der Studie.
•Patienten, die in der Lage sind, die Studienanweisungen zu befolgen und die wahrscheinlich alle erforderlichen Studienvisiten einhalten werden. (Compliance)
•Ausreichende Deutschkenntnisse müssen vorhanden sein.
•Die Patienten zeigen eine Beeinträchtigung der Sozialfunktion, gemessen anhand der GFS-Skala (Global Functioning Social (GFS ≤ 7).
•Frauen in gebärfähigem Alter müssen während der Behandlung eine zuverlässige Verhütungsmethode anwenden.
•Hohes Risiko an einer Psychose zu erkranken:
Ultra-High-Risk-(UHR)-Kriterien (Abgeschwächte positive Symptome und/oder kurze limitierte psychotische Symptome und/oder eine Kombination aus familiärem Risiko oder schizotypischen Erkrankungen mit einer signifikanten Funktionsstörung; bemessen an SIPS 5.0)

und/or

Basissymptomkriterium (Cognitive Disturbances (COGDIS), 2/9 cognitive-perceptive basic symptoms (COPER); bewertet mithilfe des „Schizophrenia Proneness Instrument“ – Erwachsenen Version, SPI-A)

Folgende Organ- und Laborparameter müssen erfüllt sein:
•Negativer Schwangerschaftstest (ß-HCG Test im Urin)

E.4

Principal exclusion criteria

1. Known history of hypersensitivity to the investigational drug or to drugs with a similar chemical structure
2. Simultaneously participation in another clinical trial involving admin-istration of an investigational medicinal product within 30 days prior to clinical trial beginning. The simultaneous participation in a non-interventional clinical trial is permitted in case the subject is neverthe-less able and willing to attend and complete all required visits and in case there are no other contraindications.
3. Subjects with a physical or psychiatric condition which at the investigator’s discretion may put the subject at other clinically significant risks than those that are defined as outcome of this study (development of a first psychotic episode, functional deterioration), may confound the trial results, or may interfere with the subject’s per protocol participation in this clinical trial.
4. Suicidality in terms of subjects, scoring higher than 0 on the CDSS item 8 'Suicidality'.
5. Known substance abuse or dependency within the last month accord-ing to DSM-IV-TR. Patients at least have to be in Early Partial Remis-sion in order to participate (Patients have met one or more Substance Abuse or Dependency Diagnosis criteria for at least 1 month but less than 12 months. However, the patient has not met all criteria for a dependence or abuse diagnosis)
6. Patients with hepatic or renal failure
7. Patients with known problems of galactose intolerance, clinically significant lactase deficiency or glucose-galactose malabsorption or histamine-intolerance
8. Subjects with known asthma bronchiale
9. Subjects with a history of gastrointestinal ulcer
10. Intake of antitussives (cough-relieving agents)
11. Intake of nitroglycerin
Exclusion criteria regarding special restrictions for females:
12. Current pregnancy or pregnancy planned within 9 months after start of medication or nursing women
13. Females of childbearing potential, who are not using and not willing to use medically reliable methods of contraception for the entire study duration (such as oral, injectable, or implantable contraceptives, or intrauterine contraceptive devices) unless they are surgically sterilized / hysterectomized or there are any other criteria considered sufficiently reliable by the investigator in individual cases
Indication specific exclusion criteria:
14. Having had a psychotic episode for > 1 week (according to SIPS 5.0);
15. Having symptoms relevant for inclusion potentially arising from a known general medical disorder
16. Life time antipsychotic medication for more than 30 days (cumulative number of days) at or above minimum dosage of the '1st episode psychosis' range of DGPPN S3 Guidelines (Exception: maximum dosage for aripiprazole 5 mg/d) (Deutsche Gesellschaft für Psychiatrie, Psychotherapie und Nervenheilkunde, 2006).
17. Any intake of antipsychotic medication (i.e., independent of duration of intake) within the past 3 months before psychopathological baseline assessments (including self-ratings and screening assessments) at or above minimum dosage of the '1st episode psychosis' range of DGPPN S3 Guidelines (Exception: maximum dosage for aripiprazole 5 mg/d) (Deutsche Gesellschaft für Psychiatrie, Psychotherapie und Nervenheilkunde, 2006).
18. Any intake of mood stabilizers (lithium, valproate, carbamazepine, ox-cabazepine, lamotrigine) > 30 days (cumulative number of days) dur-ing the past three months or any intake during the month before psy-chopathological baseline assessments (including self-ratings and screening assessments).
19. Any past psychotherapeutic treatment targeting specifically psychotic symptoms or its prevention.

Generelle Ausschlusskriterien:
• Patienten, die jünger als 18 Jahre oder älter als 40Jahre sind.
• Patient willigt nicht in die Studie ein
• Patient ist nicht in der Lage, den Umfang, die Bedeutung und die Konsequenzen dieser klinischen Prüfung zu verstehen
• Unfähigkeit des Patienten, den Studienablauf zu befolgen
• Bekannte Allergie gegen das Prüfpräparat oder gegen Präparate mit ähnlicher chemischer Struktur
• Gleichzeitige Teilnahme an einer anderen klinischen Prüfung oder Teilnahme an einer klinischen Prüfung mit Einnahme eines Prüfpräparats bis zu 30 Tage vor Teilnahme an dieser klinischen Prüfung
• Bekannter Drogenmissbrauch oder Abhängigkeit innerhalb des letzten Monats nach DSM-IV-TR. Um teilnehmen zu können müssen sich die Patienten mindestens in einer frühen partiellen Remission befinden (die Patienten haben mindestens 1 Monat aber weniger als 12 Monate lang ein oder mehrere Kriterien für die Diagnose von Substanzmissbrauch oder Abhängigkeit erfüllt. Die Patienten haben jedoch nicht alle Kriterien für eine Abhängigkeits- oder Missbrauchsdiagnose erfüllt).
• Vorliegende oder geplante Schwangerschaft oder Stillzeit
• Verfehlen der laborchemischen Einschlusskriterien

Indikationsspezifische Ausschlusskriterien:
• Ein physischer oder psychiatrischer Zustand, welcher nach Einschätzung des Prüfarztes für den Patienten bei Teilnahme an dieser klinischen Studie ein klinisch signifikantes Risiko darstellt (außer Entwicklung eines ersten psychotischen Schubs oder Funktionsstörungen)
• Lebensmüdigkeit (höher als 0 auf der Calgary Depression Skala für Schizophrenie (CDSS)
• Leber- oder Nierenschaden/-insuffizienz
• Milchzuckerunverträglichkeit/-allergie,Traubenzucker-Milchzucker Malabsorption oder Histamin-Intoleranz
• Chronische Entzündung der Atemwege ( Asthma Bronchiale)
• Magengeschwüre ( Ulcusanamnese)
• Einnahme von Hustenmitteln
• Einnahme von Nitroglycerin
• Psychotischer Schub vor > 1 Woche (gemäß SIPS 5.0)
• Symptome die relevant für die Einschließung sind und wahrscheinlich von einer bekannten Erkrankung herrühren
• Antipsychotische Arzneimittelanwendung mit einer kumulativen Dosis von > 30 mal über der minimalen Dosis gemäß S3 Richtlinie Schizophrenie (S3-Guideline Schizophrenie: Deutsche Gesellschaft für Psychiatrie und Psychotherapie, Psychosomatik und Nervenheilkunde, 2018)
• Antipsychotische Arzneimittelanwendung (unabhängig von der Dauer der Einnahme) innerhalb der letzten 30 Tage vor der psychopathologischen Ausgangsbewertung (Selbst-Bewertung und Screeningverfahren inbegriffen) ≥ der minimalen Dosis gemäß S3-Guideline Schizophrenia [Exception: quetiapine (see 18.)]. (S3-Guideline Schizophrenie: Deutsche Gesellschaft für Psychiatrie undPsychotherapie, Psychosomatik und Nervenheilkunde, 2018).
• Einnahme von > 300 mg Quetiapine (kumulative Dosis) oder für einen Zeitraum von > 7 Tagen innerhalb des letzten Monats.
• Jede Einnahme von Stimmungs-stabilizsierenden Mitteln (Lithium, Valproinsäure, Carbamazepin, Ox-Cabazepin, Lamotrigin) > 30 Tage (kumulative Tagesanzahl) während der letzten drei Monate oder jede Einnahme während des letzten Monats vor der psychopathologischen Ausgangsbewertung (including self-ratings and screening assessments)
• Jegliches psychotherapeutisches Training die spezifisch auf beeinträchtigte soziale Kognition verbunden mit psychotischen Symptomen oder deren Prävention abzielte.

E.5 End points

E.5.1

Primary end point(s)

•Transition to psychosis within up to 18 months, defined (according to EPOS1) as the presence of at least one psychotic symptom for at least one week (assessed by the SIPS).
•Improvement of psychosocial functioning after 18 months (assessed by the SOFAS and the FROGS).

•Übergang in eine Psychose innerhalb von bis zu 18 Monaten (EPOS1) durch das Auftreten von mindestens einem psychotischen Symptom für mindestens eine Woche (bewertet durch SIPS)
•Verbesserung der psychosozialen Funktion nach 18 Monaten (bewertet durch SOFAS und FROGS)

E.5.1.1

Timepoint(s) of evaluation of this end point

An interim Analysis of the end points is done when 75% of the planned number of patients (i.e. 98 out of 130) have been randomized. The timepoint cannot be defined, as randomization may vary in the course of the study.

Data will be analyzed after Termination of the Trial.

E.5.2

Secondary end point(s)

•Remission of symptomatic CHR criteria (APS/BLIPS and/or COGIDS); decrease of positive, negative and disorganization symptoms (as-sessed by the SIPS); improvement of social cognition (SAT-MC I & II, PoFA)
•Assessment of safety and tolerability: Neurologic and general exami-nation (medical history, weight, - adverse events (assessed by UKU), Calgary depression rating scale items 1,2,8,9 (CDSS), Laboratory as-sessments

•Rückbildung von symptomatischen CHR Kriterien (APS/BLIPS und/oder COGIDS); Rückgang von Desorganisationssymptomen (bewertet durch SIPS); Verbesserung der sozialen Kognition (SAT-MC I & II, PoFA)
•Bewertung der Sicherheit: Neurologische und generelle Untersuchung (medizinische Vorgeschichte, Gewicht, Größe, unerwünschte Ereignisse (AEs, bewertet durch UKU), Calgary Depressionsbewertungsskala (CDSS)
•Laboratoruntersuchungen: Haematologie, Urin-Schwangerschaftstest, Urin-Drogentest

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

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Blinded ratings of the additional psychotherapy

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

Yes

E.8.2.3.1

Comparator description

additional psychotherapy (standard counseling vs. IPPI)

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

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

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

130

F.1.3

Elderly (>=65 years)

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

130

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)

After completing the study, no psychological intervention according to the study conditions is planned. If patients still show indicators of risk according to the inclusion criteria at this time, observational follow-up by the partciapting centers will be offered to allow for an early detection of a progression to psychosis. In case of a first psychotic episode, treatment by the participating University Departments of Psychiatry will be offered.

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

2016-04-05

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-07-07

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-01-26

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