Clinical Trials Register

Summary
EudraCT Number:	2019-001075-36
Sponsor's Protocol Code Number:	CaboCHILD
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2020-02-13
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

2019-001075-36

A.3

Full title of the trial

A phase II study to evaluate the safety, tolerability and efficacy of Cabozantinib in patients with hepatocellular carcinoma (HCC) and impaired liver function (Child-Pugh score B7-8) - CABOCHILD -

Phase II Studie zur Evaluierung der Sicherheit, Verträglichkeit und Wirksamkeit von Cabozantinib in Patienten mit hepatozellulärem Karzinom (HCC) und eingeschränkter Leberfunktion (Child-Pugh Wert B7-8) - CABOCHILD -

A.3.1

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

Study to evaluate the safety, tolerability and efficacy of Cabozantinib in patients with hepatocellular carcinoma and impaired liver function.

Studie zur Evaluierung der Sicherheit, Verträglichkeit und Wirksamkeit von Cabozantinib in Patienten mit Leberkarzinom und eingeschränkter Leberfunktion.

A.3.2

Name or abbreviated title of the trial where available

CaboCHILD

A.4.1

Sponsor's protocol code number

CaboCHILD

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	Universitätsmedizin Mainz
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	Ipsen Pharma GmbH
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	Universitätsmedizin Mainz
B.5.2	Functional name of contact point	PD Dr. med. Arndt Weinmann
B.5.3	Address:
B.5.3.1	Street Address	Langenbeckstr. 1
B.5.3.2	Town/ city	Mainz
B.5.3.3	Post code	55131
B.5.3.4	Country	Germany
B.5.4	Telephone number	+496131172669
B.5.5	Fax number	+49613117472669
B.5.6	E-mail	arndt.weinmann@unimedizin-mainz.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	Yes
D.2.1.1.1	Trade name	Cabometyx 20 mg Filmtabletten
D.2.1.1.2	Name of the Marketing Authorisation holder	Ipsen Pharma GmbH
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.3.4	Pharmaceutical form	Film-coated tablet
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	Cabozantinib
D.3.9.1	CAS number	849217-68-1
D.3.9.4	EV Substance Code	SUB93452
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.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	Yes
D.2.1.1.1	Trade name	Cabometyx 40 mg Filmtabletten
D.2.1.1.2	Name of the Marketing Authorisation holder	Ipsen Pharma GmbH
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.3.4	Pharmaceutical form	Film-coated tablet
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	Cabozantinib
D.3.9.1	CAS number	849217-68-1
D.3.9.4	EV Substance Code	SUB93452
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	40
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

Hepatocellular carcinoma (HCC)

Hepatozelluläres Karzinom (HCC)

E.1.1.1

Medical condition in easily understood language

Liver carcinoma

Leberkarzinom

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

The objective of this study is to evaluate the safety, tolerability and efficacy of Cabozantinib as a second-line therapy (after one prior systemic therapy) in patients with intermediate to advanced HCC (BCLC B/C) and concomitant impaired liver function CP score B7-8.

Das Ziel der Studie ist die Untersuchung der Sicherheit, Verträglichkeit und Wirksamkeit von Cabozantinib als second line Therapie (nach einer vorherigen systemischen Therapie) bei Patienten mit mittlerem bis fortgeschrittenem HCC (BCLC B/C) und gleichzeitiger Einschränkung der Leberfunktion (CP-Score B7-8).

E.2.2

Secondary objectives of the trial

Not applicable

unzutreffend

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent
2. Age 18
3. Histological/cytological or non-invasive (according to EASL/AASLD guidelines) diagnosis of HCC
4. Availability of a recent (up to 28 days old) CT/MRI images of thorax and abdomen
5. Subject’s HCC is not amenable to a curative treatment approach (e.g., transplant, surgery, radiofrequency ablation) corresponding to BCLC
classification B/C.
6. Progression or toxicities following one prior systemic therapy for HCC
7. Recovery to ≤ grade 1 from toxicities related to any prior treatments, unless the adverse events are clinically non-significant and/or stable on
supportive therapy
8. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
9. Adequate hematologic function, based upon meeting the following laboratory criteria within 7 days before enrollment:
• absolute neutrophil count (ANC) ≥ 1200/mm3 (≥ 1.2 x 109/L)
• platelets ≥ 60,000/mm3 (≥ 60 x 109/L)
• hemoglobin ≥ 8 g/dL (≥ 80 g/L)
10. Adequate renal function, based upon meeting the following laboratory criteria within 7 days before enrollment: serum creatinine ≤ 1.5 × upper limit of normal or calculated creatinine clearance ≥ 40 mL/min (using the Cockcroft-Gault equation)
11. Liver function Child-Pugh (CP) score B7-8
12. ALBI (albumin-bilirubin) grade 1-2
13. Alanine aminotransferase (ALT) and aspartate amino-transferase (AST) < 7.0 × upper limit of normal (ULN) within 7 days before enrollment
14. Antiviral therapy per local standard of care if active hepatitis B (HBV) infection
15. Capability to understand and comply with the protocol requirements (e.g. sufficient knowledge of German language to answer the questionnaires ability to swallow intact tablets).

1. Schriftliche Zustimmung nach Aufklärung
2. Alter ≥18
3. Histologisch/zytologisch oder nicht-invasiv (nach EASL/AASLD-Richtlinien) gesichertes HCC
4. Verfügbarkeit einer aktuellen (bis zu 28 Tage alten) CT/MRT-Aufnahme von Thorax und Abdomen
5. Das HCC des Patienten ist für einen kurativen Behandlungsansatz (z.B. Transplantation, Operation, Radiofrequenzablation), der der BCLC-
Klassifikation B/C entspricht, nicht geeignet.
6. Progression oder Toxizität nach einer vorherigen systemischen Therapie bei HCC
7. Wiederherstellung bis zur Baseline oder ≤ Grad 1 CTCAE v.5.0 von Toxizitäten im Zusammenhang mit früheren Behandlungen, es sei denn, die Nebenwirkungen sind klinisch nicht signifikant und/oder stabil bei unterstützender Therapie.
8. ECOG Leistungsstatus (ECOG) 0-2
9. Ausreichende hämatologische Funktion
10. Ausreichende Nierenfunktion
11. Leberfunktion Child-Pugh (CP) Score B7-8
12. ALBI (Albumin-Bilirubin) Grad 1-2
13. Alanin-Aminotransferase (ALT) und Aspartat-Aminotransferase (AST) < 7,0 × obere Normwertgrenze (ULN) innerhalb von 7 Tagen vor Einschluss
14. Antivirale Therapie nach lokalem Behandlungsstandard bei aktiver Hepatitis B (HBV)-Infektion
15. Fähigkeit, die Anforderungen des Protokolls zu verstehen und zu erfüllen (z.B. ausreichende Deutschkenntnisse zur Beantwortung der Fragebögen, Fähigkeit, intakte Tabletten zu schlucken).

E.4

Principal exclusion criteria

1. Fibrolamellar carcinoma or mixed hepatocellular cholangiocarcinoma
2. Receipt of more than 1 prior systemic therapy for advanced HCC. Additional prior systemic therapies used as adjuvant or local therapy are allowed.
3. Any type of anti-cancer agent (including investigational) within 2 weeks before enrollment
4. Radiation therapy within 4 weeks (2 weeks for radiation for bone metastases) or radionuclide treatment (e.g., I-131 or Y-90) within 6 weeks of enrollment. Subject cannot be enrolled if there are any clinically relevant ongoing complications from prior radiation therapy.
5. Prior Cabozantinib treatment
6. Known brain metastases or cranial epidural disease unless adequately treated with radiotherapy and/or surgery (including radiosurgery) and stable for at least 3 months before enrollment. Eligible subjects must be without corticosteroid treatment at the time of enrollment.
7. Concomitant anticoagulation, at therapeutic doses, with anticoagulants such as warfarin or warfarin-related agents, low molecular weight Heparin (LMWH), thrombin or activated coagulation factor X (FXa) inhibitors, or antiplatelet agents (e.g., clopidogrel). Low-dose aspirin for cardioprotection (per local applicable guidelines), low-dose warfarin (≤ 1 mg/day), and low-dose LMWH are permitted.
8. The subject has uncontrolled, significant intercurrent or recent illness.
9. Subjects with untreated or incompletely treated varices with bleeding or high risk for bleeding are excluded with the following clarification: subjects with history of prior variceal bleeding must have been treated with adequate endoscopic therapy without any evidence of recurrent bleeding for at least 6 months prior to study entry and must be stable on optimal medical management (e.g. non-selective beta blocker, proton pump inhibitor) at study entry.
10. Women who are pregnant, nursing, or who plan to become pregnant while in the Trial.
11. Women of child-bearing potential (WOCBP) and men who are able to father a child, unwilling to be abstinent or use highly effective methods of birth control that result in a low failure rate of less than 1% per year when used consistently and correctly beginning at informed consent, for the duration of study participation and for at least 4 months after last dose of the study drug. Because oral contraceptives might possibly not be considered as "effective Methods of contraception" during the Treatment with cabozantinib, they should be used together with another method, such as a barrier method.
12. Currently receiving any other investigational agent or received an investigational agent within 30 days (or within 5 times the half-life of this agent or its relevant Metabolits, the longer period apply) before the first dose of Cabozantinib.
13. Hepatic encephalopathy Grad I-IV according to CP classification (≥ 2 points) and West Haven Criteria.
14. Moderate or severe ascites according to CP classification (≥ 3 points).
15. Corrected QT interval calculated by the Fridericia formula (QTcF) > 500 ms within 7 days before enrollment.

1. Fibrolamelläres Karzinom oder gemischtes hepatozelluläres Cholangiokarzinom.
2. Erhalt von mehr als 1 vorherigen systemischen Therapie bei fortgeschrittenem HCC.
3. Jede Art von Anti-Krebs-Medikamenten innerhalb von 2 Wochen vor Einschluss.
4. Bestrahlungstherapie innerhalb von 4 Wochen (2 Wochen bei Bestrahlung von Knochenmetastasen) oder Radionuklidbehandlung (z.B. I-131 oder Y-90) innerhalb von 6 Wochen vor Einschluss.
5. Vorherige Cabozantinib-Behandlung.
6. Bekannte Hirnmetastasen oder eine kraniale Epiduralerkrankung.
7. Begleitende Antikoagulation, in therapeutischen Dosen.
8. Patient hat eine unkontrollierte, signifikante interkurrente oder kürzlich aufgetretene Krankheit.
9. Frauen, die schwanger sind, stillen oder planen, während der Studie schwanger zu werden.
10. Frauen im gebärfähigen Alter (WOCBP) oder Männer, die ein Kind zeugen können und nicht bereit sind, abstinent zu sein oder hochwirksame Methoden der Geburtenkontrolle zu verwenden. Da orale Kontrazeptiva wahrscheinlich nicht ausreichend sicher wirksam sind, sollten sie zusammen mit einer anderen Empfängnisverhütungsmethode, wie beispielsweise einer Barrieremethode, angewendet werden.
11. Gleichzeitig oder in den letzten 30 Tagen vor dem Einschluss sich einer medikamentösen Behandlung im Rahmen einer klinischen Prüfung unterzogen haben.
12. Hepatische Enzephalopathie Grad I-IV gemäß CP- Klassifikation (≥ 2 Punkte) und West Haven-Kriterien
13. Mäßiger oder schwerer Aszites nach der CP-Klassifikation (≥ 3 Punkte)
14. Korrigiertes QT-Intervall, berechnet nach der Fridericia-Formel (QTcF) > 500 ms innerhalb von 7 Tagen vor Einschluss

E.5 End points

E.5.1

Primary end point(s)

Safety and tolerability of Cabozantinib administration in HCC patients with impaired liver function CP score B7-8, assessed by adverse events, laboratory values, vital signs, Child-Pugh class/score, ALBI score and ECOG performance Status.

Sicherheit und Verträglichkeit von Cabozantinib bei HCC-Patienten mit eingeschränkter Leberfunktion, bewertet durch die Analyse von unerwünschten Ereignissen, Laborwerten, Vitalparametern, Child-Pugh-Score, ALBI-Score und ECOG.

E.5.1.1

Timepoint(s) of evaluation of this end point

Through study completion, up to approximately 2 years.

Bis zum Studienende (bis zu ca. 2 Jahren).

E.5.2

Secondary end point(s)

- Overall survival (OS)
- Progression-free survival (PFS) per RECIST 1.1
- Objective response rate (ORR) per RECIST 1.1

Additional outcomes:
- Pharmacokinetics (PK) of Cabozantinib administration in HCC patients with impaired liver function CP score B7-8.
- Health-related quality of life (HRQOL) as assessed by the validated German version of the Chronic Liver Disease Questionnaire (CLDQ-D).
- Evaluation of ALBI grade 1 and 2 in comparison to CP score classification.

- Gesamtüberleben (OS)
- progressionsfreies Überleben (PFS)
- Objektive Ansprechrate (ORR)

zusätzliche Endpunkte:
- Pharmakokinetik (PK)
- Gesundheitsbezogene Lebensqualität (HRQOL)
- Bewertung von Albumin-Bilirubin (ALBI) im Vergleich zur CP-Score- Klassifizierung

E.5.2.1

Timepoint(s) of evaluation of this end point

Time Frame: Through study completion, up to approximately 2 years.

Additional outcomes:
- Pharmacokinetics (PK) of Cabozantinib administration in HCC patients with impaired liver function CP score B7-8
Timepoints: 6 weeks

- Health-related quality of life (HRQOL) as assessed by the validated German version of the Chronic Liver Disease Questionnaire (CLDQ-D)
Timepoints: Through study completion, up to approximately 2 years

- Evaluation of ALBI grade 1 and 2 in comparison to CP score classification
Timepoints: Through study completion, up to approximately 2 years

Zeitrahmen: Bis zum Studienende (bis zu ca. 2 Jahren).

zusätzliche Endpunkte:
- Pharmakokinetik (PK): Zeitpunkt 6 Wochen
- Gesundheitsbezogene Lebensqualität (HRQOL): Zeitpunkt: Bis zum Studienende (bis zu ca. 2 Jahren).
- Bewertung von Albumin-Bilirubin (ALBI) im Vergleich zur CP-Score- Klassifizierung: Zeitpunkt: Bis zum Studienende (bis zu ca. 2 Jahren).

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

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)

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

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

Yes

E.8.1.7.1

Other trial design description

prospektiv, einarmig

prospective, single arm

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

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

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

last visit or last scheduled procedure for the last patient

letzter Besuch oder letzte geplante Prozedur für den letzten Patienten

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

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.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 the end of the treatment, the study drug will be discontinued. The patients will be treated according to best clinical practice.

Am Ende der Studie werden die Patienten durch den Prüfer über mögliche Weiterbehandlungs- und
Weiterbetreuungsmöglichkeiten informiert. Die Patienten werden gemäß "best clinical practice" weiterbehandelt.

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

2020-04-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-03-16

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2022-04-12

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