Clinical Trials Register

Summary
EudraCT Number:	2019-004375-40
Sponsor's Protocol Code Number:	NGAM-12
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-06-17
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2019-004375-40

A.3

Full title of the trial

Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients with Chronic Lymphocytic Leukemia ("PRO-SID" study)

Studio prospettico di fase III in doppio cieco, randomizzato, controllato con placebo, volto a valutare l’efficacia e la sicurezza di Panzyga nella profilassi delle infezioni primarie in pazienti affetti da leucemia linfatica cronica (studio “PRO-SID”)

A.3.1

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

STUDIO PROSPETTICO DI FASE III IN DOPPIO CIECO, RANDOMIZZATO, CONTROLLATO CON PLACEBO, VOLTO A VALUTARE L’EFFICACIA E LA SICUREZZA DI PANZYGA NELLA PROFILASSI DELLE INFEZIONI PRIMARIE IN PAZIENTI AFFETTI DA LEUCEMIA LINFATICA CRONICA (studio “PRO-SID”)

A.3.2

Name or abbreviated title of the trial where available

PRO-SID

A.4.1

Sponsor's protocol code number

NGAM-12

A.5.4

Other Identifiers

Name:

IND number

Number:

19414

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	OCTAPHARMA PHARMAZEUTIKA PRODUKTIONSGES.M.B.H.
B.1.3.4	Country	Austria
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	Octapharma Pharmazeutika Produktionsges.m.b.H.
B.4.2	Country	Austria
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Octapharma Pharmazeutika Produktionsges.m.b.H.
B.5.2	Functional name of contact point	Global Clinical Project Manager
B.5.3	Address:
B.5.3.1	Street Address	Oberlaaer Str. 235
B.5.3.2	Town/ city	Vienna
B.5.3.3	Post code	1100
B.5.3.4	Country	Austria
B.5.4	Telephone number	00431610321796
B.5.5	Fax number	00431610321796
B.5.6	E-mail	AT1ClinicalDepartment@octapharma.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	Panzyga
D.2.1.1.2	Name of the Marketing Authorisation holder	Octapharma - Marketing Authorisation Number 236803
D.2.1.2	Country which granted the Marketing Authorisation	Austria
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	Panzyga
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	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	Immunoglobulin G
D.3.9.1	CAS number	308067-58-5
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	HUMAN NORMAL IMMUNOGLOBULIN (IV)
D.3.9.4	EV Substance Code	SUB12041MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	Yes
D.3.11.7	Plasma derived medicinal product	Yes
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	Solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Primary infection prophylaxis in patients with chronic lymphocytic leukemia (CLL) and secondary hypogammaglobulinemia

Profilassi delle infezioni primarie in pazienti con leucemia linfatica cronica (LLC) e ipogammaglobulinemia secondaria

E.1.1.1

Medical condition in easily understood language

Prophylaxis to prevent infection in patients with a type of cancer that
starts in bone marrow and then go into the blood

Profilassi per prevenire l'infezione nei pazienti con un tipo di cancro che inizia nel midollo osseo e poi entra nel sangue

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10008976
E.1.2	Term	Chronic lymphocytic leukemia
E.1.2	System Organ Class	100000004864

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of this study is to demonstrate the benefit of
Panzyga administration compared with placebo as primary infection
prophylaxis in CLL patients with secondary immunodeficiency (SID)
undergoing CLL antineoplastic therapy.

L’obiettivo primario di questo studio è dimostrare il beneficio della somministrazione di Panzyga rispetto al placebo come profilassi primaria delle infezioni in pazienti affetti da LLC con immunodeficienza secondaria (IDS) sottoposti a terapia antineoplastica per LLC.

E.2.2

Secondary objectives of the trial

The secondary objectives of this study are to compare the following
aspects in CLL patients with SID treated with and without primary
Panzyga prophylaxis:
• Overall infection rate
• Frequency of prophylaxis with anti-infectives (antibacterials and
antivirals)
• Duration of prophylaxis with anti-infectives (antibacterials and
antivirals)

Gli obiettivi secondari di questo studio sono confrontare i seguenti aspetti nei pazienti affetti da LLC con IDS trattati con e senza profilassi primaria con Panzyga:
• Tasso d’infezione complessivo
• Frequenza della profilassi con antinfettivi (antibatterci e antivirali)
• Durata della profilassi con antinfettivi (antibatterci e antivirali)

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

Other types of substudies
Specify title, date and version of each substudy with relative objectives: Pharmacokinetic (PK) Sub-study:
Pharmacokinetic parameters will be determined in a subgroup of
patients. Blood samples for the PK profiles of total IgG will be taken.
The following parameters will be assessed for patients treated with
Panzyga.
• Half-life (T1/2)
• Area under the curve (AUC0-t; AUC0-infinity)
• Volume of distribution (Vd)
• Concentration maximum (Cmax; Tmax)
• Clearance (Cl)
For patients treated with Placebo, no PK parameters will be estimated,
as the underlying assumptions for exponential modelling are not
fulfilled.

Altre tipologie di sottostudi
specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Sotto-studio di farmacocinetica (PK):
I parametri farmacocinetici saranno determinati in un sottogruppo di
pazienti. Verranno prelevati campioni di sangue per i profili PK delle IgG totali.
I seguenti parametri saranno valutati per i pazienti trattati con
Panzyga.
• Emivita (T1 / 2)
• Area sotto la curva (AUC0-t; AUC0-infinito)
• Volume di distribuzione (Vd)
• Concentrazione massima (Cmax; Tmax)
• Clearance (Cl)
Per i pazienti trattati con Placebo, non verranno stimati parametri PK,
poichè le ipotesi di base per la modellazione esponenziale non sono
soddisfatte

E.3

Principal inclusion criteria

1. Treatment-naïve or relapsed/refractory CLL patients undergoing CLL
antineoplastic treatment. Diagnosis of B-cell CLL established according
to International Workshop on Chronic Lymphocytic Leukemia (iwCLL)
criteria and documented within medical records.
2. Hypogammaglobulinemia (IgG levels <5 g/L) as confirmed by the
Central Laboratory.
3. =18 years of age.
4. Voluntarily given, fully informed written and signed consent obtained
before any study-related procedures are conducted.

1. Pazienti con LLC naïve al trattamento o recidivante/refrattario sottoposti a trattamento antineoplastico per LLC. Diagnosi di LLC a cellule B stabilita secondo i criteri del Workshop internazionale sulla leucemia linfatica cronica (iwCLL) e documentata nelle cartelle cliniche.
2. Ipogammaglobulinemia (livelli di IgG <5 g/l) confermata dal laboratorio centrale.
3. Età =18 anni.
4. Acquisizione del consenso scritto e firmato fornito volontariamente e spiegato in ogni sua parte prima dello svolgimento di qualsiasi procedura correlata allo studio.

E.4

Principal exclusion criteria

1. IgG treatment within 3 months prior to Screening.
2. Antibiotic prophylaxis and/or treatment within 7 days prior to current
CLL treatment start (with the exception of trimethoprimsulfamethoxazole
[TMP/SMX] diaminodiphenyl sulfone [dapsone] and pentamidine inhalation).
3.
Current major infection or >1 major infection in the previous 6
months
before Baseline.
4.
History of anaphylaxis or severe systemic response to
immunoglobulin,
blood or plasma-derived products or any Panzyga
component.
5.
History of a non-CLL malignancy with life-expectancy of less than two
years.
6.
Severe liver disease, with signs of ascites and/or hepatic
encephalopathy.
7.
Severe kidney disease (as defined by estimated glomerular filtration
rate
[eGFR] <30 mL/min/1.73 m2).
8.
Body weight >140 kg.
9.
Eastern Cooperative Oncology Group (ECOG) performance score of >2
(Appendix
1).
10.
Female patients of childbearing potential unwilling to use a protocolrequired
method of contraception from the Screening Visit throughout
the
study treatment period and for 30 days following the last dose of
study
drug.
11.
Human immunodeficiency virus (HIV) infection at Screening (defined
for
the study as positive HIV antibody test).
12.
Patients found to be chronic carriers of hepatitis B virus (HBV),
defined
by positivesurface antigen (HBsAg), positive Hepatitis B core antibodies (HBcAb) and/or low HBV
titers,
who will not receive targeted antiviral therapy while undergoing
CLL
therapy, and patients with active HBV, defined as high HBV titers.
13.
Uncontrolled hepatitis C infection at Screening (defined for the study
as
positive hepatitis virus C [HCV] polymerase chain reaction [PCR]).
14.
Pregnant and lactating women.
15.
Subjects with a history of thromboembolic events (TEE) such as deep
vein
thrombosis, pulmonary embolism, myocardial infarction, ischemic
stroke,
transient ischemic attack, peripheral artery disease (Fontaine IV)
within
6 months before Baseline.
16.
Planned or ongoing immunosuppressive treatment (other than for
CLL
or corticosteroids) or other forbidden medication during the entire
study
duration after study enrollment.
17.
Participation in another interventional clinical trial that is either
blinded
or involves an investigational (not approved) product within 3
months
before Baseline or during the course of the clinical study.
Participation
in observational clinical trials or open-label trials involving
an
approved product may be permitted after consultation with the
medical
monitor.
18.
Known IgA deficiency with antibodies to IgA.
19.
Known blood hyperviscosity, or other hypercoagulable states.
20.
Patients unable or unwilling to understand or comply with the study
protocol.

Criteri di esclusione:
1. Trattamento con IgG nei 3 mesi precedenti lo screening.
2. Profilassi e/o trattamento con antibiotici nei 7 giorni precedenti l’inizio dell’attuale trattamento per LLC (ad eccezione di trimetoprim-sulfametoxazolo [(TMP/SMX], diaminodifenilsulfone [dapsone]) e pentamidina per inalazione).
3. Attuale infezione importante o >1 infezione importante nei 6 mesi precedenti al basale.
4. Anamnesi di anafilassi o risposta sistemica grave a immunoglobuline, prodotti ematici o plasmaderivati o a qualsiasi componente di Panzyga.
5. Anamnesi di tumore maligno non LLC con aspettativa di vita inferiore a due anni.
6. Malattia epatica grave, con segni di ascite e/o encefalopatia epatica.
7. Malattia renale grave (definita da una velocità di filtrazione glomerulare stimata [eGFR] <30 ml/min/1,73 m2).
8. Peso corporeo >140 kg.
9. Punteggio dello stato di validità secondo il Gruppo cooperativo orientale di oncologia (ECOG) >2 (Appendice 1).
10. Pazienti di sesso femminile in età fertile non disposte a utilizzare un metodo contraccettivo richiesto dal protocollo (come specificato nella sezione 7.3.9 b del protocollo) dalla Visita di screening per tutto il periodo di trattamento dello studio e per 30 giorni dopo l’ultima dose del farmaco sperimentale.
11. Infezione da virus dell’immunodeficienza umana (HIV) allo screening (definita, ai fini dello studio, come positività al test degli anticorpi anti-HIV).
12. Pazienti risultati portatori cronici del virus dell’epatite B (HBV), definiti come positivi all’antigene di superficie (HBsAg) nonché agli anticorpi anti-proteina core dell’epatite B (HBcAb) e/o da bassi titoli di HBV, che non riceveranno una terapia antivirale mirata durante la terapia per LLC e pazienti con HBV attivo, definiti da elevati titoli di HBV.
13. Infezione da epatite C non controllata allo screening (definita, ai fini dello studio, come positività della reazione a catena della polimerasi [PCR] per il virus dell’epatite C [HCV]).
14. Donne in stato di gravidanza e allattamento al seno.
15. Soggetti con anamnesi di eventi tromboembolici (TEE) come trombosi venosa profonda, embolia polmonare, infarto miocardico, ictus ischemico, attacco ischemico transitorio, arteriopatia periferica (Fontaine IV) nei 6 mesi precedenti al basale.
16. Trattamento immunosoppressivo pianificato o in corso (che non sia per LLC o diverso da corticosteroidi) o altri farmaci proibiti durante l’intera durata dello studio dopo l’arruolamento nello studio.
17. Partecipazione ad un’altra sperimentazione clinica interventistica che sia in cieco o implichi un prodotto sperimentale (non approvato) nei 3 mesi precedenti al basale o nel corso dello studio clinico. La partecipazione a sperimentazioni cliniche osservazionali o sperimentazioni in aperto che richiedono l’uso di un prodotto approvato può essere consentita dopo consultazione con il responsabile del monitoraggio medico.
18. Deficit noto di IgA con anticorpi anti-IgA.
19. Iperviscosità ematica nota o altri stati di ipercoagulabilità.
20. Pazienti incapaci o non disposti a comprendere o a rispettare il protocollo dello studio.

E.5 End points

E.5.1

Primary end point(s)

Since the study objective is to show the benefit of Panzyga
administration as primary infection prophylaxis, the primary endpoint is
not the major infection rate but occurrence of at least one major
infection in CLL patients with or without primary infection prophylaxis
with Panzyga. Therefore, although patients will stay in the study
regardless of the number of major infections, each patient will be
counted only once for the primary endpoint calculation.
Major infection for this trial is defined as:
• Bacterial and/or viral infections resulting in death
• Bacterial and/or viral infections, which are microbiologically
documented (MDI) or clinically documented (CDI) requiring treatment
with anti-infectives; upper respiratory tract infections, bronchitis, lower
urinary tract infections, bacterial skin infections and stomatitis (MDI or
CDI) are considered major only if they require treatment with antiinfectives
AND hospitalization or hospitalization prolongation.
•
FUO requiring hospitalization or hospitalization prolongation

Poiché l'obiettivo dello studio è mostrare i benefici della somministrazione di Panzyga come profilassi dell'infezione primaria, l'endpoint primario non è il tasso di infezione principale ma l'insorgenza di almeno una infezione maggiore nei pazienti con CLL con o senza profilassi dell'infezione primaria con Panzyga. Pertanto, sebbene i pazienti rimarranno nello studio indipendentemente dal numero di infezioni maggiori, ogni paziente verrà conteggiato una sola volta per il calcolo dell'endpoint primario.Ai fini della presente sperimentazione, un’infezione importante è definita come:
• Infezioni batteriche e/o virali che portano al decesso
• Infezioni batteriche e/o virali microbiologicamente documentate (MDI) o clinicamente documentate (CDI) che richiedono trattamento con antinfettivi; le infezioni delle vie respiratorie superiori, bronchite, infezioni del tratto urinario inferiore, infezioni batteriche della pelle e stomatite (MDI o CDI) sono considerate importanti solo se richiedono un trattamento con antinfettivi E il ricovero ospedaliero o il prolungamento del ricovero.
• Febbre di origine sconosciuta (FUO) che richiede il ricovero ospedaliero o il prolungamento del ricovero ospedaliero.

E.5.1.1

Timepoint(s) of evaluation of this end point

Timepoint of major infection.

Timepoint dell'infezione maggiore.

E.5.2

Secondary end point(s)

• Overall infection rate: infection rate for all infections.
• Frequency of prophylaxis with anti-infectives (antibacterials and
antivirals)
• Duration of prophylaxis with anti-infectives (antibacterials and
antivirals)

• Tasso d’infezione complessivo: tasso di infezione per tutte le infezioni.
• Frequenza della profilassi con antinfettivi (antibatterci e antivirali)
• Durata della profilassi con antinfettivi (antibatterci e antivirali)

E.5.2.1

Timepoint(s) of evaluation of this end point

• Infections throughout the study
• anti-infectives frequency throughout the study
• duration of prophylaxis throughout the study

• Infezioni durante lo studio
• frequenza anti-infettivi durante lo studio
• durata della profilassi durante lo studio

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

Yes

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

Yes

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

Yes

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

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

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

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Russian Federation

United States

Denmark

Germany

Hungary

Italy

Poland

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:

225

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

225

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

180

F.4.2.2

In the whole clinical trial

450

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)

None

Nessuno

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

2020-05-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-04-08

P. End of Trial
P.	End of Trial Status	Ongoing

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