Clinical Trials Register

Summary
EudraCT Number:	2014-005432-33
Sponsor's Protocol Code Number:	UX001-CL301
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-01-19
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2014-005432-33

A.3

Full title of the trial

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Studio di fase 3, randomizzato, in doppio cieco, controllato verso placebo, per valutare l¿efficacia e la sicurezza di compresse a rilascio prolungato di acido sialico in pazienti affetti da miopatia da mutazione del gene GNE (glucosamina (UDP-N-acetil)-2-epimerasi/N-acetilmannosamina chinasi) (GNE Miopathy, GNEM) o miopatia ereditaria a corpi inclusi (Hereditary Inclusion Body Myopathy, HIBM)

A.3.1

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

not available

non disponibile

A.3.2

Name or abbreviated title of the trial where available

not applicable

non applicabile

A.4.1

Sponsor's protocol code number

UX001-CL301

A.5.4

Other Identifiers

Name:

non disponibile

Number:

non applicabile

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	ULTRAGENYX PHARMACEUTICAL INC.
B.1.3.4	Country	United States
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	Ultragenyx Pharmaceutical Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Ultragenyx Pharmaceutical Inc.
B.5.2	Functional name of contact point	Clinical Operations
B.5.3	Address:
B.5.3.1	Street Address	60 Leveroni Court
B.5.3.2	Town/ city	Novato, CA
B.5.3.3	Post code	94949
B.5.3.4	Country	United States
B.5.4	Telephone number	0014154838800
B.5.5	Fax number	0014154838820
B.5.6	E-mail	UX001ClinOps@ultragenyx.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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/12/972
D.3 Description of the IMP
D.3.1	Product name	Acido Sialico - Sialic acid (INN: aceneuramic acid)
D.3.2	Product code	UX001
D.3.4	Pharmaceutical form	Prolonged-release tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
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	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	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	Prolonged-release tablet
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

GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka's disease, or quadriceps sparing myopathy (QSM)

miopatia da mutazione del gene GNE, miopatia ereditaria a corpi inclusi (HIBM), miopatia distale con vacuoli cerchiati (Distal Myopathy With Rimmed Vacuoles, DMRV), malattia di Nonaka o miopatia che risparmia i quadricipiti (QSM

E.1.1.1

Medical condition in easily understood language

Myopathy

Miopatia

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10075048
E.1.2	Term	Hereditary inclusion body myopathy
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

Evaluate the effect of 6 g/day SA-ER treatment of subjects with GNEM on
upper extremity muscle strength (UEC score) as measured by
dynamometry

Valutare l¿effetto del trattamento con 6 g/die di SA-ER di soggetti affetti da GNEM sulla forza muscolare degli arti superiori (punteggio UEC) misurato mediante dinamometria.

E.2.2

Secondary objectives of the trial

Key Secondary Objectives: Evaluate the effect of 6 g/day of SA-ER treatment of subjects
with GNEM on:
¿ Lower extremity composite muscle strength score (LEC score) as measured by
dynamometry
¿ Muscle strength in the knee extensors as measured by dynamometry
¿ Physical functioning as measured using the GNEM-FAS mobility domain score
Other Secondary Objectives: Evaluate the effect of 6 g/day of SA-ER treatment of subjects with
GNEM on:
¿ Physical functioning as measured using the GNEM-FAS upper extremity domain score
¿ Lower extremity function as measured by a timed sit-to-stand test
¿ Upper extremity function as measured by a timed weighted arm lift test
¿ Lower extremity function as measured by distance walked in the 6MWT

Obiettivi Secondari Chiave: valutazione degli effetti di 6g/die di SA-ER in soggetti con GNEM su:
¿ Punteggio composito della forza muscolare degli arti inferiori (punteggio LEC) misurato con dinamometro
¿ Forza muscolare negli estensori del ginocchio misurato tramite dinamometro
¿ Funzionalit¿ fisica misurata tramite punteggio del dominio della mobilit¿ della scala GNEM-FAS
Altri Obiettivi secondari: valutare l¿effetto di SA-ER g/die in soggetti con GNEM su:
¿ Funzionalit¿ fisica misurata tramite punteggio del dominio di mobilit¿ della scala GNEM-FAS degli arti superiori
¿ Funzionalit¿ degli arti inferiori misurata tramite test sit-to stand
¿ Funzionalit¿ degli arti superiori misurata tramite test del sollevamento del braccio on carico
¿ Funzionalit¿ degli arti inferiori misurata tramite test della distanza dei 6 minuti (6MWT)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1) Male or female, aged 18 - 50 years, inclusive
2) Willing and able to provide written, signed informed consent after the
nature of the study has been explained, and before any research-related
procedures are conducted
3) Have a documented diagnosis of GNEM, HIBM, DMRV, or Nonaka
disease due to previously demonstrated mutations in the gene encoding
the GNE/MNK enzyme (genotyping will not be conducted in this study)
4) Able to provide reproducible force in elbow flexors (i.e. two
dynamometry force values with no more than 15% variability in the
dominant arm) at Screening
5) Able to walk a minimum of 200 meters during the 6MWT at Screening
without the use of assistive devices, including a cane, crutch(es),
walker, wheelchair or scooter (AFOs are permitted)
6) Willing and able to comply with all study procedures
7) Participants of child-bearing potential or with partners of childbearing
potential who have not undergone a bilateral salpingooophorectomy
and are sexually active must consent to use an effective
method of contraception as determined by the site investigator (i.e. oral
hormonal contraceptives, patch
hormonal contraceptives, vaginal ring, intrauterine device, physical
double-barrier methods, surgical hysterectomy, vasectomy, tubal
ligation, or true abstinence) from the period following the signing of the
informed consent through 3 months after last dose of study drug
8) Females of childbearing potential must have a negative pregnancy
test at Screening and be willing to have additional pregnancy tests
during the study. Females considered not of childbearing potential
include those who have been in menopause
for at least two years, have had tubal ligation at least one year prior to
Screening, or who have had a total hysterectomy or bilateral salpingooophorectomy

1. Soggetti di sesso maschile e femminile, età 18 ¿ 50 anni (compresi)
2. Disponibilità e capacità a fornire per iscritto il consenso informato firmato dopo avere ricevuto spiegazioni sulla natura dello studio e prima che siano espletate le procedure riguardanti lo studio
3. Avere una diagnosi documentata di GNEM, HIBM, miopatia distale con vacuoli cerchiati (Distal Myopathy With Rimmed Vacuoles, DMRV) o malattia di Nonaka dovuta a mutazioni precedentemente dimostrate nel gene di codifica dell’enzima GNE/MNK (ManNAc chinasi) (la genotipizzazione non sarà condotta in questo studio)
4. Capacità di applicare una forza riproducibile nei flessori del gomito (ossia, due valori di forza dinamometrica con variabilità non superiore al 15% nel braccio dominante) allo Screening
5. Capacità di camminare almeno 200 metri durante il test 6MWT allo Screening senza l’ausilio di dispositivi di assistenza, tra cui bastone, stampella/e, deambulatore, sedia a rotelle o scooter (le ortesi di piede e caviglia [Ankle-Foot Orthosis, AFO] sono consentite)
6. Disponibilità e capacità di rispettare tutte le procedure dello studio
7. I partecipanti in età fertile o con partner in età fertile che non abbiano subito una salpingo-ovariectomia e che siano sessualmente attivi devono acconsentire ad utilizzare un metodo contraccettivo altamente efficace come determinato dallo sperimentatore del centro (ossia, contraccettivi ormonali orali, cerotto a rilascio transdermico di contraccettivi ormonali, anello vaginale, dispositivo intrauterino, metodi di doppia barriera fisica, isterectomia chirurgica, vasectomia, legatura delle tube o astinenza totale[quando questo è
in linea con lo stile di vita preferito e abituale del soggetto], il che significa non fare sesso perché
il soggetto sceglie di non farlo) a partire dal periodo successivo alla firma del consenso informato fino a 3 mesi dopo l’ultima dose di farmaco in studio
8. I soggetti di sesso femminile in età fertile devono presentare un test di gravidanza negativo allo Screening ed essere disposti a sottoporsi a test di gravidanza supplementari nel corso dello studio. I soggetti di sesso femminile considerati non in età fertile includono coloro che sono in menopausa da almeno due anni, che sono stati sottoposti a intervento di legatura delle tube almeno un anno prima dello Screening o che hanno subito un’isterectomia totale o una salpingo-ovariectomia bilaterale

E.4

Principal exclusion criteria

1) Ingestion of N-acetyl-D-mannosamine (ManNAc), SA, or related
metabolites;
intravenous immunoglobulin (IVIG); or anything that can be
metabolized to produce
SA in the body within 60 days prior to the Screening Visit
2) History of more than 30 days treatment with SA-ER and/or SA-IR in
prior clinical trials in the past year
3) Has had any hypersensitivity to SA or its excipients that, in the
judgment of the investigator, places the subject at increased risk for
adverse effects
4) Has serum transaminase (i.e. aspartate aminotransferase [AST] or
gamma-glutamyl transpeptidase [GGT]) levels greater than 3X the upper
limit of normal (ULN) for age/gender, or serum creatinine of greater
than 2X ULN at Screening
5) Pregnant or breastfeeding at Screening or planning to become
pregnant (self or partner) at any time during the study
6) Use of any investigational product or investigational medical device
within 30 days prior to Screening, or anticipated requirement for any
investigational agent prior to completion of all scheduled study
assessments
7) Has a condition of such severity and acuity, in the opinion of the
investigator, that it warrants immediate surgical intervention or other
treatment or may not allow safe participation in the study
8) Has a concurrent disease, active suicidal ideation, or other condition
that, in the view of the investigator, places the subject at high risk of
poor treatment compliance or of not completing the study, or would
interfere with study participation or would affect safety

1. Assunzione di N-acetil-D-mannosammina (ManNAc), SA o metaboliti correlati, immunoglobulina per via endovenosa (Intravenous Immunoglobulin, IVIG) o tutto ciò che possa essere metabolizzato per produrre SA nell’organismo nei 60 giorni precedenti la Visita di screening
2. Anamnesi di trattamento superiore a 30 giorni con SA-ER e/o SA-IR in precedenti sperimentazioni cliniche nel corso dell’ultimo anno
3. Pregressa ipersensibilità all’SA o ai suoi eccipienti che, a giudizio dello sperimentatore, esponga il soggetto a un rischio maggiore di effetti avversi
4. Livelli di transaminasi sieriche (ossia, aspartato aminotransferasi [AST] o gamma-glutammiltransferasi [GGT]) 3 volte superiori al limite normale superiore (Upper Limit of Normal, ULN) per età/sesso o creatinina sierica 2 volte superiore l’ULN allo Screening
5. Soggetti di sesso femminile in gravidanza o allattamento al momento dello Screening o che stanno pianificando una gravidanza (il soggetto stesso o il partner) in qualsiasi momento nel corso dello studio
6. Utilizzo di qualsiasi prodotto sperimentale o dispositivo medico sperimentale nei 30 giorni precedenti lo Screening o prevista richiesta di qualsiasi farmaco sperimentale prima del completamento di tutte le valutazioni di studio in programma
7. Una condizione di gravità e acutezza tali che, a giudizio dello sperimentatore, richieda un immediato intervento chirurgico o altri trattamenti o che non possa consentire la partecipazione sicura allo studio
8. Una malattia concomitante, idee di suicidio attive o altra condizione che, a giudizio dello sperimentatore, esponga il soggetto a un rischio elevato di scarsa aderenza al trattamento o di mancato completamento dello studio o che interferirebbe con la partecipazione allo studio o ne altererebbe la sicurezza

E.5 End points

E.5.1

Primary end point(s)

The primary clinical efficacy analysis will be the change from baseline in UEC score for the
SA-ER group compared with placebo based on bilateral strength recorded in the following
muscle groups using a dynamometer: gross grip, shoulder abductors, elbow flexors, and
elbow extensors. The UEC is derived from the sum of the muscle groups. Each muscle
group value will represent the average of the right and left total values (measured in kg).
The comparison of the two treatment groups will be based on the change from baseline in
mean UEC score between SA-ER and placebo using GEE repeated measures analysis with
baseline, gender, and geographic region as covariates.

L’endpoint di efficacia primario è la variazione rispetto al basale del punteggio della forza muscolare composita degli arti superiori (UEC) tra il gruppo trattato con SA-ER verso placebo, basato sulla forza bilaterale misurata con un dinamometro nei seguenti gruppi di muscoli: forza di presa abduttori, delle spalle, dei flessori del gomito e degli estensori del gomito. Il punteggio UEC è derivato dalla somma dei gruppi di muscoli. Il valore di ciascun gruppo di muscoli è dato dalla media tra i valori totali a destra e a sinistra (misurati in kg). Il confronto tra i due gruppi di trattamento è, utilizzando l’equazione generalizzata di stima sulle misure ripetute, con basale, genere e regione geografica come covariate.

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline, Week 8, 16, 24, 32, 40, 48

Basale, Settimane 8, 16, 24, 32, 40, 48

E.5.2

Secondary end point(s)

Key Secondary Endpoints: the key secondary clinical efficacy analyses will assess the change from baseline for the SA-ER group compared with the placebo group using GEE for the following variables: ¿ LEC score based on a sum of the mean bilateral strength recorded in the following muscle groups: knee flexors, hip flexors, hip extensors, hip abductors and hip adductors
¿ Muscle strength in the knee extensors: bilateral total force (measured in kg)
¿ GNEM-FAS mobility domain score

Other Secondary Endpoints: the analysis of other secondary endpoints will assess the change from baseline for the SA-ER group compared to the placebo group using GEE for the following variables:
¿ GNEM-FAS upper extremity domain score
¿ Sit-to-stand score calculated as the number of times a subject can rise from a sitting to a standing position in a 30-second period
¿ Weighted arm lift score calculated as the number of times a subject can raise a 1 kg weight overhead in a 30-second period
¿ Walking ability as measured by the 6MWT, which will be reported as distance in meters and percent predicted based on normative data for age and gender.

Endpoint secondari chiave: gli endpoint secondari chiave dell¿analisi di efficacia clinica valutano le variazioni rispetto al basale tra il gruppi di trattamento con SA-ER e con placebo usando l¿equazione generalizzata di stima GEE sulle seguenti variabili:
¿ Punteggio compositio delle estremit¿ inferiori LEC basato sulla somma della forza media bilaterale misurata nei seguenti gruppi di muscoli: flessori del ginocchio, flessori dell¿anca, estensori dell¿anca, adduttori ed abduttori dell¿anca.
¿ Forza muscolare degli estensori del ginocchio: forza totale bilaterale (misurata in kg)
¿ Punteggio del dominio della mobilit¿ della scala GNEM- FAS

Altri endpoint secondari: l¿analisi degli altri obiettivi secondari valuta la variazione rispetto al basale dei gruppi di trattamento con SA-ER e placebo, usando l¿equazione generalizzata di stima GEE sulle seguenti variabili:
¿ Punteggio del dominio della scala GNEM-Fas degli arti superiori
¿ Punteggio del test sit-to-stand, calcolato come numero di volte in cui un soggetto passa da una posizione seduta ad una posizione eretta in 30 secondi
¿ Punteggio del test del tempo di sollevamento del braccio sotto carico, calcolato come numero di volte in cui un soggetto pu¿ sollevare 1 kg di peso in 30 secondi
¿ Capacit¿ di deambulazione misurata tramite test del cammino dei 6 minuti (6MWT), riportato come distanza in metri e percentuale dei valori predetti per et¿ e genere

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline, Week 8, 16, 24, 32, 40, 48

Basale, Settimane 8, 16, 24, 32, 40, 48

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

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

Canada

Israel

United States

Bulgaria

France

Italy

United Kingdom

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:

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

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

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)

An open-label study, UX001-CL302, will be available to subjects that complete the 48-week treatment period in this study.

Uno studio in aperto, UX001-CL302, sar¿ disponibile per pazienti che completino il trattamento di 48 settimane nello studio

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

2015-12-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-06-30

P. End of Trial
P.	End of Trial Status	Completed

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IMP with orphan designation in the indication
Orphan Designation Number:
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