Clinical Trials Register

Summary
EudraCT Number:	2022-001292-14
Sponsor's Protocol Code Number:	CV029-009
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:	2022-11-18
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2022-001292-14

A.3

Full title of the trial

A Phase 2a, Open-label, Pilot Study to Evaluate Efficacy, Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of MYK-224 in Participants with Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

Studio pilota di fase 2a, in aperto per valutare l’efficacia, la farmacocinetica, la farmacodinamica, la sicurezza e la tollerabilità di MYK-224 in partecipanti con cardiomiopatia ipertrofica sintomatica e ostruzione del tratto di efflusso ventricolare sinistro

A.3.1

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

Multiple Dose Study to Evaluate Efficacy, Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of MYK-224 in Participants with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Studio a dosi multiple per valutare l’efficacia, la farmacocinetica, la farmacodinamica, la sicurezza e la tollerabilità di MYK-224 in partecipanti con cardiomiopatia ipertrofica ostruttiva sintomatica

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

CV029-009

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1276-3555

A.5.4

Other Identifiers

Name:

IND

Number:

146425

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	MYOKARDIA, 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	MyoKardia, 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	Bristol-Myers Squibb International Corporation
B.5.2	Functional name of contact point	GSM-CT
B.5.3	Address:
B.5.3.1	Street Address	Parc de l'Aliance - Avenue de Finlande 4
B.5.3.2	Town/ city	Braine-l'Alleud
B.5.3.3	Post code	1420
B.5.3.4	Country	Belgium
B.5.6	E-mail	clinical.trials@bms.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	MYK-224 (5 mg per 1 Tablet)
D.3.2	Product code	[BMS-986435]
D.3.4	Pharmaceutical form	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	MYK-224
D.3.9.2	Current sponsor code	BMS-986435
D.3.9.3	Other descriptive name	BMS-986435
D.3.9.4	EV Substance Code	SUB263294
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	5
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	MYK-224 (10 mg per 1 Tablet)
D.3.2	Product code	[BMS-986435]
D.3.4	Pharmaceutical form	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	MYK-224
D.3.9.2	Current sponsor code	BMS-986435
D.3.9.3	Other descriptive name	BMS-986435
D.3.9.4	EV Substance Code	SUB263294
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	10
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: 3
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	MYK-224 (2.5 mg per 1 Tablet)
D.3.2	Product code	[BMS-986435]
D.3.4	Pharmaceutical form	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	MYK-224
D.3.9.2	Current sponsor code	BMS-986435
D.3.9.3	Other descriptive name	BMS-986435
D.3.9.4	EV Substance Code	SUB263294
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	2.5
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

Cardiomiopatia ipertrofica sintomatica e ostruzione del tratto di efflusso ventricolare sinistro

E.1.1.1

Medical condition in easily understood language

A kind of heart disease with enlarging of the heart and the obstruction of a cardiac tract

Un tipo di cardiopatia con ingrossamento del cuore e ostruzione di un tratto cardiaco

E.1.1.2

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

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	10020871
E.1.2	Term	Hypertrophic cardiomyopathy
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

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 the safety and tolerability of MYK-224 in participants with oHCM (obstructive hypertrophic cardiomyopathy)

Valutare la sicurezza e la tollerabilità di MYK-224 in partecipanti con oHCM (cardiomiopatia ipertrofica ostruttiva)

E.2.2

Secondary objectives of the trial

- To evaluate the effect of MYK-224 on LVOT gradient in participants with oHCM
- To assess the PK/PD relationship of MYK-224
- To assess the PK of MYK-224 in participants with oHCM

- Valutare l’effetto di MYK-224 sul gradiente del tratto di efflusso del ventricolo sinistro (LVOT) in partecipanti con oHCM
- Valutare la relazione PK/PD di MYK-224
- Valutare la PK di MYK-224 in partecipanti con oHCM

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Has adequate acoustic windows, to enable accurate TTEs as determined by the echocardiography core laboratory.
- Men or women diagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines, satisfying both of the following criteria:
-- Has unexplained left ventricular (LV) hypertrophy with nondilated ventricular chambers in the absence of other cardiac (eg, hypertension, aortic stenosis) or systemic disease and with maximal LV wall thickness >= 15 millimeter (mm) (or >= 13 mm with positive family history of hypertrophic cardiomyopathy or with a known disease-causing mutation), as determined by core laboratory interpretation.
AND
-- Has a LVOT peak gradient during screening as assessed by echocardiography of >= 50 millimeters of mercury (mm Hg) at rest, or >= 30 mm Hg at rest and >= 50 mm Hg after Valsalva maneuver (confirmed by echocardiography core laboratory interpretation).
- Has documented LVEF >= 60% at the Screening visit as determined by echocardiography core laboratory.
- Has a valid measurement of LVOT post-exercise peak gradient at screening as determined by echocardiography core laboratory.
- NYHA functional class II or III symptoms at screening.

- Dispone di finestre acustiche adeguate, per consentire ecocardiogrammi transtoracici (TTE) accurati, come determinato dal laboratorio centrale di ecocardiografia.
- Uomini o donne con diagnosi di cardiomiopatia ipertrofica ostruttiva (oHCM) coerente con le attuali linee guida dell’American College of Cardiology Foundation/American Heart Association e della European Society of Cardiology che soddisfano entrambi i seguenti criteri:
-- Presenta ipertrofia ventricolare sinistra (VS) inspiegabile con camere ventricolari non dilatate in assenza di altre patologie cardiache (ad es. ipertensione, stenosi aortica) o sistemiche e con spessore massimo della parete VS >= 15 millimetri (mm) (o >= 13 mm con anamnesi familiare positiva di cardiomiopatia ipertrofica o con una mutazione nota che causa la malattia), come determinato dall’interpretazione del laboratorio centrale.
E
-- Presenta un gradiente di picco del tratto di efflusso ventricolare sinistro (LVOT) durante lo screening valutato mediante ecocardiografia di >= 50 millimetri di mercurio (mm Hg) a riposo o >= 30 mm Hg a riposo e >= 50 mm Hg dopo la manovra di Valsalva (confermata dall’interpretazione dell’ecocardiografia del laboratorio centrale).
- Presenta una FEVS (frazione di eiezione ventricolare sinistra) documentata >= 60% alla visita di screening, come determinato dal laboratorio centrale di ecocardiografia.
- Presenta una misurazione valida del gradiente di picco LVOT post-esercizio allo screening, come determinato dal laboratorio centrale di ecocardiografia.
- Sintomi di classe funzionale della New York Heart Association (NYHA) II o III allo screening.

E.4

Principal exclusion criteria

- Presence of any medical condition that precludes exercise stress testing.
- History of syncope or sustained ventricular tachyarrhythmia within 6 months prior to screening.
- Known infiltrative or storage disorder causing cardiac hypertrophy that mimics HCM, such as Fabry disease, amyloidosis, or Noonan syndrome with left ventricular hypertrophy.
- Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the study (Note: Individuals with an unsuccessful myectomy or percutaneous ASA procedure performed > 6 months prior to Screening may be enrolled if study eligibility criteria for LVOT gradient criteria are met).
- Implantable cardioverter-defibrillator (ICD) placement or pulse generator change within 2 months prior to screening or planned new ICD placement during the study (pulse generator changes, if needed during the study are allowed).
- Has a history of resuscitated sudden cardiac arrest (any time) or known history of appropriate implantable cardioverter-defibrillator (ICD discharge for life-threatening ventricular arrhythmia within 6 months prior to screening.
- Has paroxysmal, intermittent atrial fibrillation with atrial fibrillation present per the Investigator's evaluation of the subject's ECG at the time of Screening.
- Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening.
- Has QT interval with Fridericia correction (QTcF) > 500 msec when QRS interval < 120 msec or QTcF > 520 msec when QRS >= 120 msec if participant has left bundle branch block or any other 12-lead ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II).
- Has known moderate or severe (per investigator's judgment) aortic valve stenosis at screening.
- History of LV systolic dysfunction (LVEF < 45%) at any time during their clinical course.
- Has pulmonary disease that limits exercise capacity.
- History of obstructive coronary artery disease (stenosis of > 70% of luminal diameter in one or more coronary arteries).
- Prior treatment with mavacamten or aficamten. An exception may be made in cases where myosin inhibitor use was not within 4 months of the Screening visit, and with the agreement of both the Investigator and the Sponsor Medical Monitor.
- Prior treatment with cardiotoxic agents such as anthracyclines (eg, doxorubicin) or similar.

- Presenza di qualsiasi condizione medica che precluda il test sotto sforzo.
- Anamnesi di sincope o tachiaritmia ventricolare sostenuta nei 6 mesi precedenti lo screening.
- Noto disturbo infiltrativo o da accumulo che causa ipertrofia cardiaca simile all’HCM, come la malattia di Fabry, l’amiloidosi o la sindrome di Noonan con ipertrofia ventricolare sinistra.
- È stato trattato con successo con riduzione invasiva del setto (miectomia chirurgica o ablazione alcolica del setto [ASA] percutanea) nei 6 mesi precedenti lo screening o prevede di sottoporsi a uno di questi trattamenti durante lo studio (Nota: i soggetti con una procedura di miectomia o ASA percutanea non riuscita eseguita > 6 mesi prima dello screening possono essere arruolati se soddisfano i criteri di idoneità dello studio per i criteri di gradiente LVOT).
- Posizionamento di un cardiovertitore-defibrillatore impiantabile (ICD) o cambio del generatore di impulsi nei 2 mesi precedenti lo screening o nuovo impianto di ICD pianificato durante lo studio (sono consentite cambi del generatore di impulsi, se necessari durante lo studio).
- Ha un’anamnesi di arresto cardiaco improvviso rianimato (in qualsiasi momento) o anamnesi nota di cardiovertitore-defibrillatore impiantabile appropriato (dimissione con ICD per aritmia ventricolare potenzialmente letale nei 6 mesi precedenti lo screening.
- Presenta fibrillazione atriale parossistica intermittente con fibrillazione atriale presente secondo la valutazione dello sperimentatore dell’ECG del soggetto al momento dello screening.
- Presenta fibrillazione atriale persistente o permanente senza terapia anticoagulante da almeno 4 settimane prima dello screening e/o con frequenza non adeguatamente controllata entro 6 mesi prima dello screening.
- Presenta intervallo QT con correzione di Fridericia (QTcF) > 500 msec quando l’intervallo QRS < 120 msec o QTcF > 520 msec quando il QRS è >=120 msec se il partecipante ha un blocco di branca sinistro o qualsiasi altra anomalia dell’ECG a 12 derivazioni considerata dallo sperimentatore come un rischio per la sicurezza del partecipante (ad es. blocco atrioventricolare di secondo grado di tipo II).
- Presenta nota stenosi della valvola aortica, moderata o grave (secondo il giudizio dello sperimentatore) allo screening.
- Anamnesi di disfunzione sistolica VS (FEVS < 45%) in qualsiasi momento durante il decorso clinico.
- Malattia polmonare che limita la capacità di esercizio.
- Anamnesi di coronaropatia ostruttiva (stenosi > 70% del diametro luminale in una o più arterie coronarie).
- Precedente trattamento con mavacamten o aficamten. È possibile fare un’eccezione nei casi in cui l’uso dell’inibitore della miosina non sia avvenuto entro 4 mesi dalla visita di screening e con il consenso sia dello sperimentatore sia del responsabile del monitoraggio medico dello Sponsor.
- Trattamento precedente con agenti cardiotossici come le antracicline (ad es. doxorubicina) o simili.

E.5 End points

E.5.1

Primary end point(s)

- Incidence, severity, and causality of AEs and SAEs
- Incidence of symptomatic LVEF < 50% by TTE
- Incidence of LVEF <= 30% by TTE
- Incidence of MACE, including cardiovascular death, nonfatal stroke, nonfatal myocardial infarction
- Incidence of hospitalizations (due to cardiovascular and noncardiovascular events)
- Incidence of HF events (including hospitalizations and urgent emergency room/outpatient visits for HF)
- Incidence of atrial fibrillation/flutter (new from screening and recurrent)
- Incidence of appropriate implantable cardioverter defibrillator therapy and resuscitated cardiac arrest
- Incidence of ventricular tachyarrhythmias (includes ventricular tachycardia, ventricular fibrillation, and Torsades de Pointes)
- Results of vital signs, physical exams, 12-lead ECG assessments (including HR), and clinical laboratory tests

- Incidenza, gravità e causalità di AE e SAE
- Incidenza di LVEF sintomatica < 50% per TTE (ecocardiogramma transtoracico)
- Incidenza di LVEF <= 30% per TTE
- Incidenza di MACE, inclusi morte cardiovascolare, ictus non fatale, infarto del miocardio non fatale
- Incidenza dei ricoveri (a causa di eventi cardiovascolari e non cardiovascolari)
- Incidenza di eventi HF (inclusi ricoveri e visite urgenti al pronto soccorso/ambulatoriali per HF)
- Incidenza di fibrillazione/flutter atriale (insorti dopo lo screening e ricorrenti)
- Incidenza della terapia appropriata con defibrillatore cardioverter impiantabile e rianimazione da arresto cardiaco
- Incidenza di tachiaritmie ventricolari (include tachicardia ventricolare, fibrillazione ventricolare e torsioni di punta)
- Risultati di segni vitali, esami obiettivi, valutazioni ECG a 12 derivazioni (inclusa frequenza cardiaca [HR]) e test clinici di laboratorio

E.5.1.1

Timepoint(s) of evaluation of this end point

- Up to 30 days after EOT
- Up to EOS
- Up to EOS
- Up to 30 days after EOT
- Up to 30 days after EOT
- Up to EOS
- Up to EOS
- Up to EOS
- Up to EOS
- Up to EOS

- Fino a 30 giorno dopo EOT (fine trattamento)
- Fino a EOS (fine studio)
- Fino a EOS
- Fino a 30 giorno dopo EOT
- Fino a 30 giorno dopo EOT
- Fino a EOS
- Fino a EOS
- Fino a EOS
- Fino a EOS
- Fino a EOS

E.5.2

Secondary end point(s)

- Effect on LVOT gradient
- PK/PD relationship
- PK concentrations

- Effetto sul gradiente del LVOT (tratto di efflusso del ventricolo sinistro)
- Relazione PK/PD
- Concentrazioni farmacocinetiche

E.5.2.1

Timepoint(s) of evaluation of this end point

- Up to EOT
- Up to EOS
- Up to EOS

- Fino a EOT
- Fino a EOS
- Fino a EOS

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

Yes

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

Tollerabilità

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

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

Studio pilota in aperto

Open-label, Pilot Study

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

United States

Poland

Spain

Italy

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

End of trial is defined as the last participant last visit or scheduled procedure as shown as in the Schedule of Activities.

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)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.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)

The subjects can continue with standard approved medication for this condition.

I soggetti possono continuare con i farmaci standard approvati per questa condizione.

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

2022-12-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-04-18

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

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials