Clinical Trials Register

Summary
EudraCT Number:	2022-002301-24
Sponsor's Protocol Code Number:	NMD670-02-0001
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:	2023-08-21
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

2022-002301-24

A.3

Full title of the trial

A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy

Studio di fase 2, randomizzato, in doppio cieco, controllato con placebo, crossover a 2 vie per valutare l’efficacia, la sicurezza e la tollerabilità di NMD670 negli adulti deambulanti con atrofia muscolare spinale di tipo 3

A.3.1

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

A study to evaluate the efficacy, safety, and tolerability of oral NMD670 compared with placebo in ambulatory adults with spinal muscular atrophy

Studio per valutare l’efficacia, la sicurezza e la tollerabilità di NMD670 orale rispetto al placebo negli adulti deambulanti con atrofia muscolare spinale

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

NMD670-02-0001

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	NMD Pharma A/S
B.1.3.4	Country	Denmark
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	NMD Pharma A/S
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Parexel International
B.5.2	Functional name of contact point	Clinical Trials Department
B.5.3	Address:
B.5.3.1	Street Address	70 Sir John Rogerson's Quay
B.5.3.2	Town/ city	Dublin
B.5.3.3	Post code	2
B.5.3.4	Country	Ireland
B.5.6	E-mail	clinicaltrial.enquiries@parexel.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	-
D.3.2	Product code	[NMD670]
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	-
D.3.9.1	CAS number	2354321-33-6
D.3.9.2	Current sponsor code	NMD670
D.3.9.3	Other descriptive name	NMD670
D.3.9.4	EV Substance Code	SUB208279
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	300
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.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	-
D.3.2	Product code	[NMD670]
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	-
D.3.9.1	CAS number	2354321-33-6
D.3.9.2	Current sponsor code	NMD670
D.3.9.3	Other descriptive name	NMD670
D.3.9.4	EV Substance Code	SUB208279
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	100
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	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated 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

Type 3 spinal muscular atrophy

Atrofia muscolare spinale di tipo 3

E.1.1.1

Medical condition in easily understood language

Spinal muscular atrophy - an inherited condition that makes the muscles weaker and causes problems with movement.

Atrofia muscolare spinale - una condizione ereditata che rende i muscoli più deboli e causa problemi di movimento.

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	10079415
E.1.2	Term	Spinal muscular atrophy type III
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

To assess changes in muscle strength and function after NMD670 400 mg bid for 21 days, compared with placebo, in participants with SMA

Valutare i cambiamenti nella forza e nella funzione muscolare dopo la somministrazione di NMD670 400 mg due volte al giorno per 21 giorni, rispetto al placebo, nei partecipanti con SMA

E.2.2

Secondary objectives of the trial

- To assess changes in muscle strength after NMD670 400 mg bid for 21 days, compared with placebo, in participants with SMA
- To further assess changes in muscle strength and function after NMD670 400 mg bid for 21 days, compared with placebo, in participants with SMA
- To assess changes in neuromuscular junction transmission after NDM670 400 mg bid for 21 days, compared with placebo, in participants with SMA
- To assess the safety and tolerability of NMD670, compared with placebo, over 21-day dosing, in participants with SMA
Exploratory objectives:
- To assess changes in patient-reported quality of life and fatigue severity after 21-day dosing of NMD670, compared with placebo, in participants with SMA
- To explore drug exposure after NMD670 400 mg bid for 21 days in participants with SMA

- Valutare i cambiamenti nella forza muscolare dopo la somministrazione di NMD670 400 mg due volte al giorno per 21 giorni, rispetto al placebo, nei partecipanti con SMA
- Valutare ulteriormente i cambiamenti nella forza e nella funzione muscolare dopo la somministrazione di NMD670 400 mg due volte al giorno per 21 giorni, rispetto al placebo, nei partecipanti con SMA
- Valutare le variazioni nella trasmissione della giunzione neuromuscolare dopo la somministrazione di NDM670 400 mg due volte al giorno per 21 giorni, rispetto al placebo, nei partecipanti con SMA
- Valutare la sicurezza e la tollerabilità di NMD670, rispetto al placebo, nell’arco di 21 giorni di somministrazione, in partecipanti affetti da SMA
Obiettivi esplorativi:
- Valutare le variazioni nella qualità della vita riferita dal paziente e nella gravità della fatica dopo la somministrazione di NMD670 per 21 giorni, rispetto al placebo, nei partecipanti con SMA
- Esplorare l’esposizione al farmaco dopo (v. protocollo)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Participant must be 18 to 60 years of age inclusive, at the time of signing the informed consent.
2. Participants who are with a clinical diagnosis of Type 3 SMA.
3. Participants who are ambulatory, defined as being able to walk at least 50 metres without walking aids.
4. Participant with genetic confirmation of diagnosis (i.e., homozygous deletion of survival of motor neuron 1 gene [SMN1]).
5. Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
6. Participants with a MFM-32 dimension 1 score <80% at screening.
7. Participants with >=7% CMAP amplitude decrement at screening (recorded from the trapezius muscle during repeated nerve stimulation [RNS]).
8. Participant has a body mass index (BMI) within the range 19-30 kg/m2 (inclusive).
9. Participant is male or female.
10. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Male Participants:
• A male participant must agree to use a highly effective contraception as detailed in Appendix 4 of this protocol during the intervention period and for at least 14 days after the last dose of study intervention (corresponding to time needed to eliminate study intervention) and refrain from donating sperm during this period.
Female Participants:
• A female participant is eligible to participate if she is not pregnant (see Appendix 4), not breastfeeding, and at least one of the following conditions applies:
o Not a woman of childbearing potential (WOCBP) as defined in Appendix 4.
OR
o A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 during the intervention period and for at least 14 days after the last dose of study intervention (corresponding to time needed to eliminate study intervention).
11. Participant is capable of giving signed informed consent as described in Appendix 1, Section 10.1.3 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

1. Il partecipante deve avere dai 18 ai 60 anni inclusi, al momento della firma del consenso informato.
2. Partecipanti con diagnosi clinica di SMA di tipo 3.
3. Partecipanti che sono deambulanti, definiti come in grado di camminare per almeno 50 metri senza ausili per la deambulazione.
4. Partecipante con conferma genetica della diagnosi (ovvero, delezione omozigote del gene della sopravvivenza del motoneurone 1 [SMN1]).
5. Partecipante con 3-5 copie del gene della sopravvivenza del motoneurone 2 [SMN2].
6. Partecipanti con un punteggio di dimensione 1 MFM-32 <80% allo screening.
7. Partecipanti con decremento dell'ampiezza CMAP >=7% allo screening (registrato dal muscolo trapezio durante la stimolazione nervosa ripetuta [RNS]).
8. Il partecipante ha un indice di massa corporea (BMI) compreso tra 19 e 30 kg/m2 (inclusi).
9. Il partecipante è maschio o femmina.
10. L'uso di contraccettivi da parte di uomini e donne deve essere coerente con le normative locali relative ai metodi di contraccezione per coloro che partecipano a studi clinici.
Partecipanti maschi:
• Un partecipante di sesso maschile deve accettare di utilizzare una contraccezione altamente efficace come dettagliato nell'Appendice 4 del presente protocollo durante il periodo di trattamento e per almeno 14 giorni dopo l'ultima dose di trattamento dello studio (corrispondente al tempo necessario per eliminare il trattamento dello studio) e astenersi dal donare sperma durante questo periodo.
Partecipanti femminili:
• Una partecipante di sesso femminile è idonea a partecipare se non è incinta (vedi Appendice 4), non sta allattando e si applica almeno una delle seguenti condizioni:
o Non una donna in età fertile (WOCBP) come definito nell'Appendice 4.
OPPURE
o Un WOCBP che accetta di seguire la guida contraccettiva nell'Appendice 4 durante il periodo di trattamento e per almeno 14 giorni dopo l'ultima dose di trattamento dello studio (corrispondente al tempo necessario per eliminare il trattamento dello studio).
11. Il partecipante è in grado di fornire il consenso informato firmato come descritto nell'Appendice 1, Sezione 10.1.3 che include la conformità ai requisiti e alle restrizioni elencate nel modulo di consenso informato (ICF) e nel presente protocollo.

E.4

Principal exclusion criteria

1. Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
2. Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
3. Participant with a clinical diagnosis of gout, or with serum uric acid >6.5 mg/dL at screening.
4. Participant with clinically significant ECG abnormalities at screening including PR interval >=220 msec, irregular rhythms (other than sinus arrhythmia or occasional, rare supraventricular or rare ventricular ectopic beats) in the judgement of the Investigator, or T-wave configurations are not of sufficient quality for assessing QT interval duration.
5. Participant with any of the following abnormalities in QT interval corrected for heart rate using Fridericia's correction (QTcF) at screening:
a. QTcF >450 msec for male participants without bundle branch block.
b. QTcF >470 msec for female participants without bundle branch block.
c. QTcF >480 msec in participants with bundle branch block.
6. Participant with any of the following:
a. Abnormal liver function test defined as total bilirubin >1.5×ULN (participants with Gilbert's syndrome can be included with total bilirubin >1.5×ULN if direct bilirubin is <=1.5×ULN and <=35% of total bilirubin).
b. Current or chronic history of liver disease including (but is not limited to) hepatitis virus infections, drug- or alcohol-related liver disease, nonalcoholic steatohepatitis, autoimmune hepatitis, hemochromatosis, Wilson's disease, a-1 antitrypsin deficiency, primary biliary cholangitis, primary sclerosing cholangitis, or any other liver disease considered clinically significant by the Investigator.
c. Known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones).
7. Participant with clinically significant laboratory test abnormalities at screening.
8. Participant with breast cancer within the past 10 years or lymphoma, leukaemia, or any malignancy within the past 5 years. An exception of this 5-year requirement is basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 5 years.
9. Participants with ongoing significant psychiatric disorder (e.g., uncontrolled depression, anxiety).
10. Participants with positive drug screen (cocaine, heroin, ketamine [unless medically prescribed], opiates) at screening.
11. Participants with positive human immunodeficiency virus (HIV) antibody test.
12. Participants with presence of hepatitis B surface antigen (HBsAg) [or hepatitis B core antibody (HBcAb)] at screening or within 3 months prior to first dose of investigational intervention.
13. Participants with positive hepatitis C antibody or ribonucleic acid (RNA) test result at screening or within 3 months prior to Day 1.

1. Partecipanti con precedente intervento chirurgico o deformità fissa (scoliosi, contratture) che limiterebbe la capacità di svolgere attività correlate allo studio.
2. Partecipanti con altre malattie significative che possono interferire con l'interpretazione dei dati dello studio (ad esempio, altre malattie neuromuscolari o muscolari).
3. Partecipante con diagnosi clinica di gotta o con acido urico sierico > 6,5 mg / dL allo screening.
4. Partecipante con anomalie ECG clinicamente significative allo screening incluso intervallo PR >= 220 msec, ritmi irregolari (diversi dall'aritmia sinusale o occasionali, rari battiti ectopici sopraventricolari o rari) a giudizio dello sperimentatore, o le configurazioni dell'onda T non sono di qualità sufficiente per valutare la durata dell'intervallo QT.
5. Partecipante con una delle seguenti anomalie nell'intervallo QT corretto per la frequenza cardiaca utilizzando la correzione di Fridericia (QTcF) allo screening:
a. QTcF >450 msec per partecipanti di sesso maschile senza blocco di branca.
b. QTcF >470 msec per partecipanti di sesso femminile senza blocco di branca.
c. QTcF >480 msec nei partecipanti con blocco di branca.
6. Partecipante con uno dei seguenti:
a. Test di funzionalità epatica anormale definito come bilirubina totale > 1,5 × ULN (i partecipanti con la sindrome di Gilbert possono essere inclusi con bilirubina totale >1,5×ULN se la bilirubina diretta è <=1,5×ULN e <=35% della bilirubina totale).
b. Anamnesi attuale o cronica di malattia epatica incluse (ma non limitate a) infezioni da virus dell'epatite, malattia epatica correlata a farmaci o alcol, steatoepatite non alcolica, epatite autoimmune, emocromatosi, malattia di Wilson, deficit di a-1 antitripsina, colangite biliare primitiva, colangite sclerosante o qualsiasi altra malattia del fegato considerata clinicamente significativa dallo sperimentatore.
c. Anomalie epatiche o biliari note (ad eccezione della sindrome di Gilbert o calcoli biliari asintomatici).
7. Partecipante con anomalie dei test di laboratorio clinicamente significative allo screening.
8. Partecipante con cancro al seno negli ultimi 10 anni o linfoma, leucemia o qualsiasi tumore maligno negli ultimi 5 anni. Un'eccezione a questo requisito di 5 anni sono i carcinomi epiteliali a cellule basali o squamose della pelle che sono stati resecati senza evidenza di malattia metastatica per 5 anni.
9. Partecipanti con disturbo psichiatrico significativo in corso (ad es. Depressione incontrollata, ansia).
10. Partecipanti con test antidroga positivo (cocaina, eroina, ketamina [se non prescritto dal medico], oppiacei) allo screening.
11. Partecipanti con test anticorpale positivo per il virus dell'immunodeficienza umana (HIV).
12. Partecipanti con presenza di antigene di superficie dell'epatite B (HBsAg) [o anticorpo centrale dell'epatite B (HBcAb)] allo screening o entro 3 mesi prima della prima dose dell'intervento sperimentale.
13. Partecipanti con risultato positivo del test per l'anticorpo dell'epatite C o l'acido ribonucleico (RNA) allo screening o entro 3 mesi prima del giorno 1.

E.5 End points

E.5.1

Primary end point(s)

Change from baseline in 6MWT (total distance) after 21-day dosing

Variazione rispetto al basale nel test 6MWT (distanza totale) dopo una somministrazione di 21 giorni

E.5.1.1

Timepoint(s) of evaluation of this end point

After 21 day dosing

Dopo 21 giorni di somministrazione

E.5.2

Secondary end point(s)

Change from baseline in individual muscle groups maximum strength (measured with a dynamometer) after 21-day dosing
Change from baseline after 21-day dosing in:
• 6MWT (fatigue index)
• revised Hammersmith scale score
• ESNHP
Change from baseline in jitter and blocking measured via sfEMG after 21-day dosing
AEs, physical examinations, clinical laboratory parameters, vital signs, ECG, C-SSRS
Exploratory:
Change from baseline after 21-day dosing in:
• INQoL score
• FSS
Plasma levels of NMD670 and its metabolite NMD1190

Variazione rispetto al basale nella forza massima dei singoli gruppi muscolari (misurata con un dinamometro) dopo una somministrazione di 21 giorni
Variazione rispetto al basale dopo una somministrazione di 21 giorni in:
• 6MWT (indice di affaticamento)
• punteggio sulla scala di Hammersmith revisionata
• ESNHP
Variazione rispetto al basale nel tremore e nel blocco misurato mediante sfEMG dopo una somministrazione di 21 giorni
EA, esami obiettivi, parametri clinici di laboratorio, segni vitali, ECG, C-SSRS
Esplorativi:
Variazione rispetto al basale dopo una somministrazione di 21 giorni in:
• Punteggio INQoL
• FSS
Livelli plasmatici di NMD670 e del suo metabolita NMD1190

E.5.2.1

Timepoint(s) of evaluation of this end point

After 21 day dosing
During study participation

Dopo 21 giorni di somministrazione
Durante la partecipazione allo studio

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

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

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

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

Yes

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

Belgium

Canada

Denmark

Germany

Italy

Netherlands

Spain

United States

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

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)

Standard of care

Terapia standard

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

2023-07-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-04-05

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

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IMP with orphan designation in the indication
Orphan Designation Number:
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