Clinical Trials Register

Summary
EudraCT Number:	2022-001950-45
Sponsor's Protocol Code Number:	INS1009-212
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:	2022-11-17
Trial results

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

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2022-001950-45

A.3

Full title of the trial

An Open-Label Extension Study to Assess the Safety, Tolerability, and Effectiveness of the Long-Term use of Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Hypertension Associated with Interstitial Lung Disease.

Studio di estensione in aperto volto a valutare la sicurezza, la tollerabilità e l’efficacia dell’uso a lungo termine di Treprostinil Palmitil in polvere per inalazione in partecipanti affetti da ipertensione polmonare associata a malattia polmonare interstiziale

A.3.1

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

A study to evaluate the effects of Treprostinil Palmitil Inhalation Powder in participants with Pulmonary Hypertension with Interstitial Lung Disease

Uno studio volto a valutare gli effetti di Treprostinil Palmitil in polvere per inalazione in partecipanti affetti da ipertensione polmonare associata a malattia polmonare interstiziale

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

INS1009-212

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	INSMED INCORPORATED
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	Insmed Incorporated
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	Insmed Switzerland GmbH
B.5.2	Functional name of contact point	Regulatory Affairs Manager EU
B.5.3	Address:
B.5.3.1	Street Address	Grafenauweg 10
B.5.3.2	Town/ city	Zug
B.5.3.3	Post code	6300
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+41763823300
B.5.5	Fax number	000000
B.5.6	E-mail	urnell.greaves@insmed.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	Treprostinil Palmitil Inhalation Powder
D.3.2	Product code	[INS1009]
D.3.4	Pharmaceutical form	Inhalation powder
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Inhalation use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TREPROSTINIL PALMITIL
D.3.9.1	CAS number	1706528-83-7
D.3.9.2	Current sponsor code	INS1009
D.3.9.4	EV Substance Code	SUB21168
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	80
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	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.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	Treprostinil Palmitil Inhalation Powder
D.3.2	Product code	[INS1009]
D.3.4	Pharmaceutical form	Inhalation powder
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Inhalation use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TREPROSTINIL PALMITIL
D.3.9.1	CAS number	1706528-83-7
D.3.9.2	Current sponsor code	INS1009
D.3.9.4	EV Substance Code	SUB21168
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	160
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	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.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	Treprostinil Palmitil Inhalation Powder
D.3.2	Product code	[INS1009]
D.3.4	Pharmaceutical form	Inhalation powder
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Inhalation use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TREPROSTINIL PALMITIL
D.3.9.1	CAS number	1706528-83-7
D.3.9.2	Current sponsor code	INS1009
D.3.9.4	EV Substance Code	SUB21168
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	320
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	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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Inhalation powder
D.8.4	Route of administration of the placebo	Inhalation use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Pulmonary Hypertension Associated with Interstitial Lung Disease

Ipertensione polmonare associata a malattia polmonare interstiziale

E.1.1.1

Medical condition in easily understood language

Participants with high blood pressure in the arteries of the lungs

Partecipanti con pressione alta nelle arterie dei polmoni

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

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	10077731
E.1.2	Term	Pulmonary hypertension WHO functional class I
E.1.2	System Organ Class	100000004855

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	10077732
E.1.2	Term	Pulmonary hypertension WHO functional class II
E.1.2	System Organ Class	100000004855

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	10077733
E.1.2	Term	Pulmonary hypertension WHO functional class III
E.1.2	System Organ Class	100000004855

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	10077734
E.1.2	Term	Pulmonary hypertension WHO functional class IV
E.1.2	System Organ Class	100000004855

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 evaluate the safety and tolerability of the long-term use of TPIP in participants with PH-ILD from Study INS1009-211 and other lead-in studies of TPIP in participants with PH-ILD (pulmonary hypertension associated with interstitial lung disease).

Valutare la sicurezza e la tollerabilità dell’uso a lungo termine di TPIP in partecipanti affetti da PH-ILD provenienti dallo studio INS1009-211 e da altri studi di lead-in su TPIP in partecipanti affetti da PH-ILD (Ipertensione polmonare associata a malattia polmonare interstiziale ).

E.2.2

Secondary objectives of the trial

-To evaluate the effect of the long-term use of TPIP on exercise capacity in participants with PH-ILD.
-To evaluate the effect of the long-term use of TPIP on lung function in participants with PH-ILD.
-To evaluate the effects of the long-term use of TPIP on blood biomarkers of disease severity in participants with PH-ILD.
-To evaluate the effect of the long-term use of TPIP on the clinical worsening rate in participants with PH-ILD.
-To evaluate the effect of the long-term use of TPIP on the exacerbations of underlying lung disease in participants with PH-ILD.
-To assess the effect of the long-term use of TPIP on QOL (PRO measures) in participants with PH-ILD.
-To further evaluate the PK profile of the long-term use of TPIP in participants with PH-ILD.

- Valutare l’effetto dell’uso a lungo termine di TPIP sulla capacità di esercizio in partecipanti affetti da PH-ILD.
- Valutare l’effetto dell’uso a lungo termine di TPIP sulla funzione polmonare nei partecipanti affetti da PH-ILD
- Valutare gli effetti dell’uso a lungo termine di TPIP sui biomarcatori ematici di gravità della malattia nei partecipanti affetti da PH-ILD.
- Valutare l’effetto dell’uso a lungo termine di TPIP sul tasso di peggioramento clinico nei partecipanti affetti da PH-ILD
- Valutare l’effetto dell’uso a lungo termine di TPIP sulle esacerbazioni della malattia polmonare preesistente nei partecipanti affetti da PH-ILD.
- Valutare l’effetto dell’uso a lungo termine di TPIP sulla QOL (misurazioni PRO) nei partecipanti affetti da PH-ILD.
- Valutare ulteriormente il profilo PK dell’uso a lungo termine di TPIP nei partecipanti affetti da PH-ILD.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male and female participants who completed the end of treatment visit in Study INS1009-211, or any other lead-in PH-ILD TPIP study. Participants for whom the OLE study was not available at the time of their completion of the lead-in study are eligible for enrolment within one year of their lead-in end of treatment visit.
2. Complete baseline screening assessments to confirm eligibility to participate if more than 30 days have elapsed since the end of the study visit in Study INS1009-211, or any other lead-in PH-ILD TPIP study. Requirements for baseline screening assessments are located in Section 4.1.1.1. of the Protocol
3. Capable of giving signed informed consent that includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

1. Partecipanti di sesso maschile e femminile che hanno completato la visita di fine trattamento nello studio INS1009-211, o qualsiasi altro studio di lead-in su TPIP nella PH-ILD. I partecipanti per i quali lo studio OLE non era disponibile al momento del completamento dello studio di lead-in sono idonei per l’arruolamento entro un anno dalla loro visita di fine trattamento dello studio di lead-in
2. Aver completato le valutazioni di screening al Basale per confermare l’idoneità a partecipare se sono trascorsi più di 30 giorni dalla fine della visita dello studio nello studio INS1009-211, o in qualsiasi altro studio di lead-in su TPIP nella PH-ILD. I requisiti per le valutazioni di screening al Basale sono disponibili nella Sezione 4.1.1.1 del protocollo
3. Devono essere in grado di fornire un consenso informato firmato, che include la conformità ai requisiti e alle restrizioni elencati nel modulo di consenso informato (Informed consent form, [ICF]) e in questo protocollo

E.4

Principal exclusion criteria

1. Participants who experienced any hypersensitivity or adverse drug reaction or were withdrawn early/discontinued in a previous PH-ILD TPIP study, which in the opinion of the Investigator, could indicate that continued treatment with TPIP may present an unreasonable risk for the participant.
2. Initiation of parenteral administration of prostacyclin analogues (eg, TRE, epoprostenol) since the completion of Study INS1009-211 or other TPIP studies. Initiation of inhaled prostacyclin analogues (eg, TRE [Tyvaso] or iloprost) and oral prostacyclin analogues (eg, TRE [Orenitram]) or receptor agonists (eg, selexipag) are permitted if stopped 24 hours prior to the start of study drug administration.
3. Pregnant or breastfeeding. Male and female participants must use contraceptives that are consistent with local regulations regarding the methods of contraception for those participating in clinical studies (contraceptive guidance is located in Section 10.4). Female participants of childbearing potential must have a negative urine pregnancy test result at trial entry before the first dose of study drug. Additional requirements for pregnancy testing during and after study intervention are located in Section 8.2.4.2 and Section 8.3.5.
4. Any medical or psychological condition, including relevant laboratory abnormalities at screening that, in the opinion of the Investigator, suggest a new and/or insufficiently understood disease that may present an unreasonable risk to the study participant as a result of participation in the study.

1. I partecipanti che abbiano manifestato qualsiasi ipersensibilità o reazione avversa al farmaco o che siano stati ritirati anticipatamente/abbiano interrotto la partecipazione a un precedente studio su TPIP nella PH-ILD, cosa che a giudizio dello sperimentatore potrebbe indicare che proseguire il trattamento con TPIP può presentare un rischio eccessivo per il partecipante
2. Avvio della somministrazione parenterale di analoghi della prostaciclina (per es., TRE, epoprostenolo) dal momento del completamento dello studio INS1009-211 o di altri studi su TPIP. L’avvio di trattamento con analoghi della prostaciclina per via inalatoria (per es., TRE [Tyvaso] o iloprost) e analoghi della prostaciclina per via orale (per es., TRE [Orenitram]) o agonisti del recettore (per es., selexipag) è consentito se interrotto 24 ore prima dell’inizio della somministrazione del farmaco in studio.
3. Gravidanza o allattamento al seno. I partecipanti di sesso maschile e femminile dovranno utilizzare contraccettivi conformi alle normative locali riguardanti i metodi contraccettivi per coloro che partecipano a studi clinici (le linee guida sulla contraccezione sono disponibili nella Sezione 10.4 del Protocollo). Le partecipanti potenzialmente fertili devono presentare un risultato negativo al test di gravidanza sulle urine al momento dell’ingresso nella sperimentazione prima della prima dose del farmaco in studio. Ulteriori requisiti per il test di gravidanza durante e dopo il trattamento dello studio sono disponibili nella Sezione 8.2.4.2 e nella Sezione 8.3.5 del Protocollo
4. Qualsiasi condizione medica o psicologica, comprese anomalie di laboratorio rilevanti allo screening che, a giudizio dello sperimentatore, suggeriscano una malattia nuova e/o non conosciuta in modo sufficiente che potrebbe presentare un rischio eccessivo per il partecipante allo studio a seguito della partecipazione allo studio stesso

E.5 End points

E.5.1

Primary end point(s)

Frequency and severity of TEAEs during the study.

Frequenza e gravità dei TEAE durante lo studio.

E.5.1.1

Timepoint(s) of evaluation of this end point

Month 6, Month 12, Month 18, and Month 24

Mese 6, Mese 12, Mese 18, e Mese 24

E.5.2

Secondary end point(s)

- Change from pre-OLE baseline* to Month 6, Month 12, Month 18, and Month 24 in 6MWD (absolute and relative).
- Change from pre-OLE baseline* to Month 6, Month 12, Month 18, and Month 24 in FVC, FVC%, FEV1, FEV1%, and FEF25-75% (absolute and relative).
- Change from pre-OLE baseline* to Month 12 and Month 24 in DLCO (absolute and relative).
- Change from pre-OLE baseline* to Month 6, Month 12, Month 18, and Month 24 in the concentration of NT-proBNP in blood.
- Annualized rate of clinical worsening events. Clinical worsening is defined as one of the following:
---Hospitalization due to a cardiopulmonary indication
---Lung transplantation
---Death from any cause
---Decrease in 6MWD = 15% from baseline, directly related to disease under study, at 2 consecutive visits at least 24 hours apart
---Need for additional PH therapy.
- Annualized clinical worsening event rate defined as the total number of clinical worsening events that occurred during the treatment period divided by the total number of participant-years during the treatment period.
- Annualized rate of occurrence of AE-ILDs.
- Change from OLE baseline in the K-BILD and EQ-5D-5L questionnaire scores at Month 6, Month 12, Month 18, and Month 24.
- Plasma concentration levels of TP and TRE.

- Variazione dal Basale* pre-OLE al Mese 6, Mese 12, Mese 18 e Mese 24 nella 6MWD (assoluta e relativa).
- Variazione dal Basale* pre-OLE al Mese 6, Mese 12, Mese 18 e Mese 24 a livello di FVC, FVC%, FEV1, FEV1%, and FEF25-75% (assoluta e relativa).
- Variazione dal Basale* pre-OLE al Mese 12 e al Mese 24 a livello di DLCO (assoluta e relativa).
- Variazione dal Basale* pre-OLE al Mese 6, Mese 12, Mese 18 e Mese 24 a livello di concentrazione di NT-proBNP nel sangue.
- Tasso annualizzato di eventi di peggioramento clinico.Il peggioramento clinico è definito come uno dei seguenti eventi:
• Ricovero ospedaliero a causa di un’indicazione cardiopolmonare
• Trapianto di polmone
• Morte per qualsiasi causa
• Riduzione della 6MWD = 15% dal Basale, direttamente collegata alla malattia in studio, in 2 visite consecutive a distanza di almeno 24 ore
• Necessità di una terapia aggiuntiva per la PH
- Tasso annualizzato di eventi di peggioramento clinico, definito come il numero totale di eventi di peggioramento clinico che si sono verificati durante il periodo di trattamento diviso per il numero totale di anni-partecipante durante il periodo di trattamento.
- Tasso annualizzato di insorgenza di AE-ILDs.
- Variazione rispetto al Basale dell’OLE nei punteggi dei questionari K-BILD ed EQ-5D-5L al Mese 6, al Mese 12, al Mese 18 e al Mese 24.
- Livelli di concentrazione plasmatica di TP e TRE.

E.5.2.1

Timepoint(s) of evaluation of this end point

Month 6, Month 12, Month 18, and Month 24

Mese 6, Mese 12, Mese 18, e Mese 24

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

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)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

E.8.1.2

Open

Yes

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

3-week titration period (Open-label TPIP titration and Blinded titration)

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

Argentina

United States

Spain

Germany

Italy

Belgium

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

End of Study is defined as the date of the last Follow-up telephone call or visit for the last participant in the study.
Participants will be considered to have completed the study if they have completed all visits of the study including the Follow-up telephone call or visit.

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)

Following completion of the open label study, subjects will be discontinued from study medication and continue their care under their physician

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

2023-02-03

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-02-14

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

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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