Clinical Trials Register

Summary
EudraCT Number:	2020-004427-16
Sponsor's Protocol Code Number:	GR40549
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:	2022-04-29
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

2020-004427-16

A.3

Full title of the trial

A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY AND TOLERABILITY OF THE PORT DELIVERY SYSTEM WITH RANIBIZUMAB IN PATIENTS WITH NEOVASCULAR AGE-RELATED MACULAR DEGENERATION (PORTAL)

STUDIO DI ESTENSIONE IN APERTO E MULTICENTRICO,
VOLTO A VALUTARE LA SICUREZZA E LA TOLLERABILITÀ A
LUNGO TERMINE DELL’IMPIANTO INTRAVITREALE DI
SOMMINISTRAZIONE A RILASCIO GRADUALE (PDS) DI
RANIBIZUMAB IN PAZIENTI AFFETTI DA DEGENERAZIONE
MACULARE LEGATA ALL’ETÀ DI TIPO NEOVASCOLARE

A.3.1

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

A Study to Evaluate Long-Term Safety and Tolerability of the Port Delivery System with Ranibizumab in Patients with Neovascular Age-Related Macular Degeneration (Portal)

Uno studio per valutare la sicurezza e la tollerabilità a lungo termine del sistema di somministrazione portuale con ranibizumab in pazienti con degenerazione maculare neovascolare correlata all'età (Portal)

A.3.2

Name or abbreviated title of the trial where available

PORTAL

A.4.1

Sponsor's protocol code number

GR40549

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	F. HOFFMANN - LA ROCHE LTD.
B.1.3.4	Country	Switzerland
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	F.Hoffman-La Roche Ltd
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	F. Hoffman-La Roche Ltd
B.5.2	Functional name of contact point	Trial Information Support Line-TISL
B.5.3	Address:
B.5.3.1	Street Address	Grenzacherstrasse 124
B.5.3.2	Town/ city	Basel
B.5.3.3	Post code	4070
B.5.3.4	Country	Switzerland
B.5.6	E-mail	global.rochegenentechtrials@roche.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	Lucentis
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis
D.2.1.2	Country which granted the Marketing Authorisation	United States
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	Ranibizumab
D.3.2	Product code	[RO4893594]
D.3.4	Pharmaceutical form	Solution for injection in pre-filled syringe
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravitreal use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RANIBIZUMAB
D.3.9.1	CAS number	347396-82-1
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB22314
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	up to
D.3.10.3	Concentration number	1
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)	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	RANIBIZUMAB
D.3.2	Product code	[RO4893594]
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	Ocular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RANIBIZUMAB
D.3.9.1	CAS number	347396-82-1
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB22314
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	Yes
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

Neovascular Age-Related Macular Degeneration (nAMD)

Degenerazione maculare neovascolare correlata all'età (nAMD)

E.1.1.1

Medical condition in easily understood language

AMD is an eye disease that impacts the central area of the retina in the eye. Neovascular AMD, is a serious type of AMD that causes vision loss due to abnormal blood vessel growth.

L'AMD è una malattia che colpisce l'area centrale della retina. L'AMD neovascolare, è un tipo grave di AMD che causa la perdita della vista a causa della crescita anormale dei vasi sanguigni.

E.1.1.2

Therapeutic area

Diseases [C] - Eye Diseases [C11]

MedDRA Classification

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	10071129
E.1.2	Term	Neovascular age-related macular degeneration
E.1.2	System Organ Class	10015919 - Eye 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 the long-term safety and tolerability of ranibizumab delivered via the Port Delivery System with ranibizumab (PDS) every 24 weeks (Q24W) or every 36 weeks (Q36W) with the 100 mg/mL formulation

Valutare la sicurezza e la tollerabilità a lungo termine di ranibizumab, nella formulazione da 100 mg/mL, erogato mediante PDS in regime Q24W o Q36W

E.2.2

Secondary objectives of the trial

- To evaluate the efficacy of ranibizumab delivered via the PDS Q24W or Q36W with the 100 mg/mL formulation, as assessed by visual acuity
- To evaluate the efficacy of ranibizumab, delivered via the PDS Q24W or Q36W with the 100-mg/mL formulation, as assessed by center point thickness (CPT) on optical coherence tomography (OCT)
- To evaluate the proportion of patients who undergo supplemental treatment with intravitreal ranibizumab 0.5 mg

-Valutare l’efficacia di ranibizumab, nella formulazione da 100 mg/mL, erogato mediante PDS in regime Q24W o Q36W, in base all’acuità visiva
-Valutare l’efficacia di ranibizumab, nella formulazione da 100 mg/mL, erogato mediante PDS in regime Q24W o Q36W, in base a CPT all’OCT
-Valutare la percentuale di pazienti sottoposti a trattamento supplementare con iniezione intravitreale di ranibizumab 0,5 mg

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Previous enrollment in and completion of Study GX28228 (Ladder) or Study GR40548 (Archway), without early treatment or study discontinuation in either study (monthly intravitreal ranibizumab 0.5 mg or implant arms) OR Previous enrollment in Study WR42221 (Velodrome) and either not eligible to be randomized in Study WR42221 at Week 24 or completed the study (from the Q24W or Q36W arm)
- Ability and willingness to undertake all scheduled visits and assessments
- For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures during the treatment period and for at least 28 days after the last intravitreal injection of ranibizumab or 1 year after the last implant refill-exchange of ranibizumab

-Precedente arruolamento nello studio GX28228 (Ladder) o GR40548 (Archway) e completamento dello stesso, senza interruzione anticipata del trattamento o della sperimentazione nell’ambito dell’uno o dell’altro (braccio trattato con iniezioni intravitreali mensili di ranibizumab 0,5 mg o braccio trattato con PDS). OPPURE Precedente arruolamento nello studio WR42221 (Velodrome) e inidoneità alla randomizzazione alla Settimana 24 nell’ambito dello stesso oppure completamento della sperimentazione (braccio in regime Q24W o Q36W).
-Capacità e volontà di sottoporsi a tutte le visite e le valutazioni in programma
-Per le pazienti in età fertile: consenso a praticare l’astinenza dai rapporti eterosessuali o a fare uso di
metodi contraccettivi,durante il periodo di trattamento e per almeno 28 giorni dopo l'ultima iniezione intravitreale di ranibizumab o 1 anno dopo l'ultimo ricambio di impianto di ranibizumab

E.4

Principal exclusion criteria

- Pregnant or breastfeeding, or intending to become pregnant during the treatment period and for at least 28 days after the last intravitreal injection of ranibizumab or 1 year after the last implant refill-exchange of ranibizumab
- History of other ocular diseases that give reasonable suspicion of a disease or condition that contraindicates the use of ranibizumab, that might affect interpretation of the results of the study or that renders the patient at high risk for treatment complications
- History of other diseases, metabolic dysfunction, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of ranibizumab or placement of the implant and that might affect interpretation of the results of the study or that renders the patient at high risk of treatment complications
- Requirement for continuous use of any medications or treatments indicated in the "Prohibited Therapy"

-Gravidanza o allattamento o intenzione di iniziare una gravidanza durante il periodo di trattamento e
almeno nei 28 giorni successivi all’ultima iniezione intravitreale di ranibizumab o nell’anno successivo
all’ultima procedura di ricarica del dispositivo con ranibizumab.
¿ Le pazienti in età fertile, comprese quelle sottopostesi a un intervento di chiusura delle tube, dovranno
eseguire un test di gravidanza sulle urine in occasione della visita di arruolamento e in occasione di
specifiche visite successive. L’eventuale risultato positivo al test di gravidanza sulle urine dovrà essere
confermato da un test di gravidanza su siero.
¿ Positività anamnestica per altra patologia oculare, che ponga il ragionevole sospetto di una malattia o
condizione rappresentante una controindicazione all’uso di ranibizumab, che possa interferire con
l’interpretazione dei risultati dello studio o che esponga il paziente ad alto rischio di complicanze correlate
al trattamento.
¿ Positività anamnestica per altra malattia, disfunzione metabolica o referto di laboratorio, che ponga il
ragionevole sospetto di una malattia o condizione rappresentante una controindicazione all’uso di
ranibizumab o all’inserimento dell’impianto intravitreale, che possa interferire con l’interpretazione dei
risultati dello studio o che esponga il paziente ad alto rischio di complicanze correlate al trattamento.
¿ Necessità continuativa di qualsiasi farmaco o trattamento indicato nel paragrafo “Terapie non consentite”
del protocollo.

E.5 End points

E.5.1

Primary end point(s)

1. Incidence and severity of ocular and systemic (non-ocular) adverse events (AEs)
2. Incidence, severity, and duration of adverse event of special interest (AESIs)
3. Incidence, severity, and duration of ocular AESIs during the postoperative period (up to 37 days of initial implantation) and follow-up period (>37 days after implantation surgery) for patients who receive the implant in the study
4. Incidence and severity of adverse device effects
5. Incidence, causality, severity, and duration of anticipated serious adverse device effects

1. Incidenza e gravità degli eventi avversi (EA) oculari e sistemici (non oculari)
2. Incidenza, gravità e durata dell'evento avverso di particolare interesse (AESI)
3. Incidenza, gravità e durata degli AESI oculari durante il periodo postoperatorio (fino a 37 giorni dall'impianto iniziale) e il periodo di follow-up (>37 giorni dopo l'intervento chirurgico di impianto) per i pazienti che ricevono l'impianto nello studio
4. Incidenza e gravità degli effetti avversi del dispositivo
5. Incidenza, causalità, gravità e durata degli effetti avversi gravi previsti dal dispositivo

E.5.1.1

Timepoint(s) of evaluation of this end point

1-5 for cohorts 1-4: up to week 240; for cohorts 5-7: up to week 144

1-5 per le coorti 1-4: fino alla settimana 240; per le coorti 5-7: fino alla settimana 144

E.5.2

Secondary end point(s)

1. Change in best-corrected visual acuity (BCVA) score from baseline over time, as assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart at a starting distance of 4 meters
2. Percentage of patients who lose < 15, < 10, or < 5 letters in BCVA score from baseline over time
3. Percentage of patients with BCVA score of 38 letters (of 20/200 approximate Snellen equivalent) or worse over time
4. Percentage of patients with BCVA score of 69 letters (20/40 approximate Snellen equivalent) or better over time
5. Change from baseline in CPT over time
6. Percentage of patients who undergo supplemental treatment with intravitreal ranibizumab 0.5 mg during each refill-exchange interval

1. Modifica nel tempo del punteggio dell'acuità visiva (BCVA) con la migliore correzione rispetto al basale, come valutato utilizzando il diagramma dell'acuità visiva dello studio sulla retinopatia diabetica con trattamento precoce (ETDRS) a una distanza iniziale di 4 metri
2. Percentuale di pazienti che perdono < 15, < 10 o < 5 lettere nel punteggio BCVA rispetto al basale nel tempo
3. Percentuale di pazienti con punteggio BCVA di 38 lettere (di 20/200 equivalente a Snellen approssimativo) o peggiore nel tempo
4. Percentuale di pazienti con punteggio BCVA di 69 lettere (20/40 equivalente a Snellen approssimativo) o migliore nel tempo
5. Modifica dal valore di base in CPT nel tempo
6. Percentuale di pazienti sottoposti a trattamento supplementare con ranibizumab intravitreale 0,5 mg durante ciascun intervallo di ricarica e scambio

E.5.2.1

Timepoint(s) of evaluation of this end point

1. for cohorts 1-4: Baseline to Week 240; for cohorts 5-7: baseline to week 144
2-4. for cohorts 1-4:Up to Week 240; for cohorts 5-7: Up to Week 144
5. for cohorts 1-4:Baseline to Week 240; for cohorts 5-7:Baseline to Week 144
6. for cohorts 1-4:baseline to week 240; for cohorts 5-7: baseline to week 144

1. per le coorti 1-4: basale alla settimana 240; per le coorti 5-7: basale alla settimana 144
2-4. per le coorti 1-4: fino alla settimana 240; per le coorti 5-7: fino alla settimana 144
5. per le coorti 1-4: basale alla settimana 240; per le coorti 5-7: dal basale alla settimana 144
6. per le coorti 1-4: basale alla settimana 240; per le coorti 5-7: basale alla settimana 144

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

Yes

E.6.13.1

Other scope of the trial description

Tolerability, Biomarkers, exploratory patient experience

Tollerabilità, biomarcatori, esperienza esplorativa del paziente

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

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

in aperto, I pazienti hanno completato la Fase II GX28228, la Fase III GR40548, la Fase IIIb WR42221

Patients completed Phase II GX28228 , Phase III GR40548, Phase IIIb WR42221 or completed W24

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

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

Argentina

Australia

Brazil

Canada

Israel

Korea, Republic of

Singapore

Taiwan

United States

Austria

Belgium

France

United Kingdom

Spain

Switzerland

Germany

Italy

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:

145

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

890

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

216

F.4.2.2

In the whole clinical trial

1035

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)

The sponsor will terminate the trial once Ranibizumab becomes commercially available. Currently, the Sponsor does not have any plans to provide Ranibizumab to patients who have completed the study. The Sponsor may evaluate whether to continue providing Ranibizumab in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product is available at the following website: http://www.roche.com/policy_continued_access_to_investigational_ medicines.pdf

Lo sponsor interromperà la sperimentazione una volta che Ranibizumab sarà disponibile in commercio. Attualmente, lo sponsor non ha in programma di fornire Ranibizumab ai pazienti che hanno completato lo studio. Lo sponsor può valutare se continuare a fornire Ranibizumab in conformità con la Roche Global Policy on Continued Access to Investigational Medicinal Product è disponibile sul seguente sito Web: http://www.roche.com/policy_continued_access_to_investigational_ medicines.pdf

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

2022-07-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-07-05

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

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