Clinical Trials Register

Summary
EudraCT Number:	2021-003294-66
Sponsor's Protocol Code Number:	INS1009-211
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:	2022-03-24
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

2021-003294-66

A.3

Full title of the trial

A Phase 2, Randomized, Double-Blind, Multicenter, Placebo-Controlled Study to Evaluate the Safety and Tolerability of Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Hypertension Associated with Interstitial Lung Disease

Studio di fase 2, randomizzato, in doppio cieco, multicentrico, controllato con placebo volto a valutare la sicurezza e la tollerabilità 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 con malattia polmonare interstiziale

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

INS1009-211

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT05176951

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.5.6	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.3.9.3	Other descriptive name	Treprostinil palmitil inhalation powder - 320 microgram
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.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.3.9.3	Other descriptive name	Treprostinil palmitil inhalation powder - 80 microgram
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: 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.3.9.3	Other descriptive name	Treprostinil palmitil inhalation powder - 160 microgram
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.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	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	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	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

The primary objective of this study (INS1009-211) is to evaluate safety and tolerability of TPIP compared with placebo in participants with PHILD.

L'obiettivo primario di questo studio (INS1009-211) è valutare la sicurezza e la tollerabilità di TPIP rispetto al placebo in partecipanti con ipertensione polmonare associata a malattia polmonare interstiziale (PH-ILD)

E.2.2

Secondary objectives of the trial

The secondary objective is to evaluate PK of TP and TRE in plasma

L'obiettivo secondario è valutare la PK di TP e TRE nel plasma

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Males and females must be = 18 to = 75 years of age at the time of signing the ICF.
2. Diagnosis of PH associated with ILD (including IIP, IPF, CTD, sarcoidosis) at least 6 months prior to Screening.
3. Uncorrected DLCO < 50% of predicted
4. ppFVC = 40% and = 70%
5. PH confirmed by RHC at Screening or within 90 days prior to Screening, with the following hemodynamic findings:
a. Pulmonary vascular resistance (PVR) = 4 Wood Units (WU) and
b. Pulmonary capillary wedge pressure (PCWP) of = 15 mmHg and
c. Mean pulmonary arterial pressure (mPAP) = 25 mmHg.
6. 6MWD at Screening = 100 meters
7. Male and female participants must use contraceptives that are consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
8. Capable of giving signed informed consent that includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
9. Able to understand and comply with protocol requirements, restrictions, and instructions, and likely to complete the study as planned, as judged by the Investigator

1. I soggetti di sesso maschile e femminile devono avere un'età compresa tra = 18 e = 75 anni al momento della firma dell'ICF.
2. Diagnosi di PH associata a ILD (comprese IIP, IPF, CTD, sarcoidosi) almeno 6 mesi prima dello Screening.
3. DLCO non corretta < 50% del valore previsto
4. ppFVC = 40% e = 70%
5. PH confermata da RHC allo Screening o nei 90 giorni precedenti lo Screening, con i seguenti risultati emodinamici:
a. Resistenza vascolare polmonare (PVR) = 4 unità Wood (WU) e
b. Pressione capillare polmonare di incuneamento (PCWP) di = 15 mmHg e
c. Pressione arteriosa polmonare media (mPAP) = 25 mmHg
6. 6MWD allo Screening = 100 metri
7. I partecipanti di sesso maschile e femminile devono usare metodi contraccettivi coerenti con le normative locali relative ai metodi contraccettivi per coloro che partecipano a studi clinici
8. Essere in grado di fornire un consenso informato firmato che include il rispetto dei requisiti e delle limitazioni elencati nel modulo di consenso informato e in questo protocollo.
9. Capacità di comprendere e rispettare i requisiti, le restrizioni e le istruzioni del protocollo e presumibile capacità di completare lo studio come previsto, secondo il giudizio dello Sperimentatore

E.4

Principal exclusion criteria

1. Primary diagnosis of chronic obstructive pulmonary disease (COPD).
2. Allergy, or documented hypersensitivity or contraindication to TPIP or TRE or mannitol (an excipient of the TPIP formulation).
3. Received or currently treated with riociguat, endothelial receptor antagonists, selexipag, phosphodiesterase 5 (PDE5) inhibitors and/or prostacyclin analogues within 30 days prior to Screening.
4. Started therapy with pirfenidone or nintedanib < 90 days prior to Screening, OR, if already receiving either medication, there is a dose change within 30 days of Screening Visit
5. QTcF interval > 480 ms on resting ECG at Screening.
6. Any known ventricular or supraventricular tachyarrhythmia (except for paroxysmal atrial fibrillation), and/or any symptomatic bradycardia.
7. History of heart disease including left ventricular ejection fraction (LVEF) = 40% or clinically significant valvular, constrictive, or symptomatic atherosclerotic heart disease (eg, stable angina, myocardial infarction, etc).
8. Systolic BP < 90 mmHg at Screening.
9. Participation in a cardiopulmonary rehabilitation program within 30 days of the first Screening Visit.
10. Evidence of multisegment or bilateral thromboembolic disease as assessed by VQ scan, pulmonary angiography, or pulmonary CT scan.
11. Acutely decompensated heart failure within 30 days of Screening Visit.
12. Moderate and severe hepatic impairment (Child-Pugh category 2 and 3) at Screening.
13. Active liver disease or hepatic dysfunction manifested as:
a. Elevated liver function test results (ALT or AST >2 × ULN)
b. Bilirubin >1.5 × ULN (isolated bilirubin >1.5 × ULN is acceptable if bilirubin is fractionated and direct bilirubin <35%)
c. Known hepatic or biliary abnormalities, not including Gilbert's syndrome or asymptomatic gallstones
14. History of HIV infection or positive HIV serology at Screening.
15. Established diagnosis of hepatitis B viral infection, or positive for HBsAg at the time of Screening
a. Participants who have gained immunity for hepatitis B virus infection after vaccination (ie, participants who are HBsAg-negative, HBsAb positive, and HBcAb-negative) are eligible for the study
b. Participants with positive HBcAbs are eligible for the study only if the hepatitis B virus DNA level is undetectable
16. Established diagnosis of hepatitis C viral infection at the time of Screening. Participants positive for hepatitis C antibody are eligible for the study only if hepatitis C virus RNA is negative.
17. Active and current symptomatic COVID-19 and/or previous diagnosis of moderate to severe disease, or hospitalization due to COVID-19.
18. History of organ transplantation.
19. Use of live attenuated vaccines within 30 days of the Screening Visit.
20. Supplemental oxygen requirement > 10 L/min at Screening.
21. Exacerbation of underlying lung disease or active pulmonary or upper respiratory infection within 30 days of the first dose of study drug (may be rescreened at appropriate time).
22. Current or recent (past 30 days) lower respiratory tract infection (may be rescreened at appropriate time).
23. Any form of congenital heart disease or congenital heart defect (repaired or unrepaired) other than a patent foramen ovale.
24. The participant has anemia as defined by a screening hemoglobin value < 9.0 g/dL, active infection, or any other condition that in the opinion of the Investigator would interfere with the interpretation of study assessments.
For the full list of exclusion criteria pleas refer to the protocol

1. Diagnosi primaria di malattia polmonare ostruttiva cronica (BPCO)
2. Allergia o ipersensibilità documentata o controindicazione a TPIP o TRE o mannitolo (un eccipiente della formulazione di TPIP).
3. Trattamento pregresso o attuale con riociguat, antagonisti del recettore dell'endotelina (ERA), selexipag, inibitori della fosfodiesterasi 5 (PDE5) e/o analoghi della prostaciclina nei 30 giorni precedenti lo Screening.
4. Inizio di una terapia con pirfenidone o nintedanib < 90 giorni prima dello Screening, OPPURE, se è già stato somministrato uno dei due farmaci, modifica della dose nei 30 giorni precedenti la Visita di screening
5. Intervallo QTcF > 480 ms all'ECG a riposo allo Screening.
6. Qualsiasi tachiaritmia ventricolare o sopraventricolare (eccetto fibrillazione atriale parossistica) e/o bradicardia sintomatica nota.
7. Anamnesi di malattia cardiaca, compresi valori di frazione di eiezione ventricolare sinistra (LVEF) = 40% o malattia cardiaca valvolare, costrittiva o aterosclerotica sintomatica clinicamente significativa (per es. angina stabile, infarto del miocardio, ecc.).
8. BP sistolica < 90 mmHg allo Screening.
9. Partecipazione a un programma di riabilitazione cardiopolmonare nei 30 giorni precedenti la prima Visita di screening.
10. Evidenza di malattia tromboembolica multisegmento o bilaterale valutata mediante scansione VQ, angiografia polmonare o TC polmonare.
11. Insufficienza cardiaca acutamente scompensata nei 30 giorni precedenti la Visita di screening
12. Compromissione epatica moderata e grave (categoria Child-Pugh 2 e 3) allo Screening
13. Malattia epatica attiva o disfunzione epatica che si manifesta come:
a. Aumento nei risultati dei test di funzionalità epatica (ALT o AST > 2 × ULN)
b. Bilirubina > 1,5 × ULN (la bilirubina isolata > 1,5 × ULN è accettabile qualora si tratti di bilirubina frazionata e la bilirubina diretta sia < 35%)
c. Anomalie epatiche o biliari note, esclusa la sindrome di Gilbert o i calcoli biliari asintomatici
14. Anamnesi di infezione da HIV o sierologia HIV positiva allo Screening.
15. Diagnosi accertata di infezione virale da epatite B o positività per HBsAg allo Screening
a. I partecipanti che hanno sviluppato immunità all'infezione da virus dell'epatite B dopo la vaccinazione (ovvero, i partecipanti HBsAg negativi, HBsAb positivi e HBcAb negativi) sono eleggibili per lo studio
b. I partecipanti con risultati positivi per HBcAb sono eleggibili per lo studio solo se il livello di DNA del virus dell'epatite B non è rilevabile
16. Diagnosi accertata di infezione virale da epatite C allo Screening. I partecipanti positivi agli anticorpi dell'epatite C sono eleggibili per lo studio solo se il test dell'RNA del virus dell'epatite C è negativo.
17. COVID-19 attiva e attualmente sintomatica e/o precedente diagnosi di malattia da moderata a grave oppure ricovero ospedaliero dovuto alla COVID-19.
18. Anamnesi di trapianto di organo.
19. Uso di vaccini vivi attenuati nei 30 giorni precedenti la Visita di screening.
20. Fabbisogno di ossigeno supplementare > 10 l/min allo Screening
21. Esacerbazione della malattia polmonare preesistente o infezione polmonare o delle vie respiratorie superiori nei 30 giorni precedenti la prima dose di farmaco in studio (potrà essere eseguito il re-screening al momento opportuno).
22. Infezione attuale o recente (ultimi 30 giorni) delle vie respiratorie inferiori (potrà essere eseguito il re-screening al momento opportuno).
23. Qualsiasi forma di malattia cardiaca congenita o difetto cardiaco congenito (riparato o non riparato) diverso da un forame ovale pervio.
24. Il partecipante presenta anemia definita da un valore di emoglobina allo Screening < 9,0 g/dl, un'infezione attiva o qualsiasi altra condizione che, a giudizio dello Sperimentatore, potrebbe interferire con l'interpretazione delle valutazioni dello studio.
Per la lista completa dei criteri di esclusione si prega di far riferimento al protocollo

E.5 End points

E.5.1

Primary end point(s)

Frequency and severity of TEAEs and SAEs during the study. Change in oxygenation, as measured by pulse oximetry, pre-, during, and post- 6MWT at Baseline, Week 5, Week10, and Week 16

Frequenza e gravità dei TEAE e SAE durante lo studio. Variazione a livello di ossigenazione, misurata tramite pulsossimetria, prima, durante e dopo il 6MWT al Basale, alla Settimana 5, alla Settimana 10 e alla Settimana 16.

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 5, Week10, and Week 16

E.5.2

Secondary end point(s)

Plasma PK parameters of TP and TRE, such as Cmax, Tmax, AUC24, CL/F, t½, and Vd/F

Parametri PK plasmatici di TP e TRE, quali Cmax, Tmax, AUC24, CL/F, t½ e Vd/F

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 10

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

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

Argentina

Mexico

United States

Belgium

France

Germany

Italy

United Kingdom

Spain

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)

Continued access to study drug will be contingent upon availability of an OLE study. Patients who have stopped study drug after completing EOT may enroll into the OLE at a later date. Patients who will immediately continue into the OLE will remain on blinded study drug for an additional 30 days after V7 in an end of study treatment extension (ESTE) to facilitate individual subject unblinding. At the completion of the ESTE, such participants may be screened and consent to enter the OLE study.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2022-07-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-05-24

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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