Clinical Trials Register

Summary
EudraCT Number:	2021-003294-66
Sponsor's Protocol Code Number:	INS1009-211
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-03-04
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

Spain - AEMPS

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

Estudio de fase 2, aleatorizado, doble ciego, multicéntrico y controlado con placebo para evaluar la seguridad y tolerabilidad de treprostinil palmitilo en polvo para inhalación en pacientes con hipertensión pulmonar asociada a enfermedad pulmonar intersticial

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

Estudio para evaluar los efectos de treprostinil palmitilo en polvo para inhalación en pacientes con hipertensión pulmonar asociada a enfermedad pulmonar intersticial

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	+34900834223
B.5.6	E-mail	RegistroEspanolDeEstudiosClinicos@druginfo.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 - 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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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 - 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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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 - 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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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

Hipertensión pulmonar asociada a enfermedad pulmonar intersticial

E.1.1.1

Medical condition in easily understood language

Participants with high blood pressure in the arteries of the lungs

Pacientes con presión alta en las arterias pulmonares

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

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

El objetivo principal de este estudio(INS1009-211) es evaluar la seguridad y la tolerabilidad de TPPI en comparación con placebo en pacientes con hipertensión pulmonar asociada a enfermedad pulmonar
intersticial.

E.2.2

Secondary objectives of the trial

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

El objetivo secundario es Evaluar la FC de TP y TRE en 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. Tanto los varones como las mujeres deberán tener entre ≥18 y ≤75 años en el momento de firmar el DCI.
2. Diagnóstico de HP asociada a EPI (como NII, FPI, ETC, sarcoidosis) a menos 6 meses antes de la selección.
3. DLCO no corregida <50% del valor previsto.
4. ppFVC≥40% y ≤70%.
5. HP confirmada mediante CCD en la selección o en los 90 días previos a la selección, con los siguientes hallazgos hemodinámicos:
a. Resistencia vascular pulmonar (RVP) ≥4 unidades Wood (UW) y
b. Presión de enclavamiento capilar pulmonar (PECP) ≤15mmHg y
c. Presión arterial pulmonar media (PAPm) ≥25mmHg.
6. DR6M ≥100 metros en la selección.
7. Los participantes de ambos sexos deben utilizar anticonceptivos compatibles con las normas locales sobre métodos anticonceptivos para los participantesen estudios clínicos.
8. Capacidad para otorgar el consentimiento informado firmado, lo que incluye el cumplimiento de los requisitos y restricciones que se recogen en el DCI y en este protocolo.
9. Capacidad para comprender y cumplir los requisitos, restricciones e instrucciones del protocolo y probabilidad de completar el estudio según lo previsto, según el criterio del investigador.

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.
25. Body Mass Index ≥40 kg/m2 or ≤ 18 kg/m2.
26. Reasonable likelihood of receiving lung transplant within the 16-week study period, as determined by Investigator.
27. Any physical limitation that would impair the participant from using the inhaler device or inability to generate a peak inspiratory flow of greater than 60 L/min, as determined by Investigator.

1. Diagnóstico principal de enfermedad pulmonar obstructiva crónica (EPOC).
2. Alergia o hipersensibilidad documentada o contraindicación a TPPI, TRE o manitol (un excipiente de la formulación de TPPI).
3. Recepción o tratamiento actual con riociguat, antagonistas de los receptores de la endotelina (ARE), selexipag, inhibidores de la fosfodiesterasa 5 (PDE5) y/o análogos de la prostaciclina en los 30 días previos a la selección.
4. Inicio del tratamiento con pirfenidona o nintedanib <90 días antes de la selección O, si ya está recibiendo alguno de los medicamentos, modificación de la dosis en los 30 días previos a la visita de selección.
5. Intervalo QTcF >480ms en el ECG en reposo en la selección.
6. Taquiarritmia ventricular o supraventricular conocida (excepto fibrilación auricular paroxística) y/o bradicardia sintomática.
7. Antecedentes de cardiopatía, como fracción de eyección del ventrículo izquierdo (FEVI) ≤40% o valvulopatía, constricción o cardiopatía aterosclerótica sintomática clínicamente significativa (por ejemplo, angina estable, infarto de miocardio, etc.).
8. PA sistólica <90 mmHg en la selección.
9. Participación en un programa de rehabilitación cardiopulmonar en los 30 días previos a la primera visita de selección.
10. Signos de enfermedad tromboembólica multisegmentaria o bilateral, evaluada mediante gammagrafía pulmonar de ventilación y perfusión, angiografía pulmonar o TC pulmonar.
11. Insuficiencia cardíaca descompensada aguda en los 30 días previos a la visita de selección.
12. Insuficiencia hepática moderada y grave (categoría 2 y 3 de Child-Pugh) en la selección.
13. Enfermedad hepática activa o disfunción hepática que se manifiesta como:
a. Resultados elevados de las pruebas de función hepática (ALT o AST >2 × LSN)
b. Bilirrubina >1,5 × LSN (bilirrubina aislada >1,5 × LSN es aceptable si la bilirrubina está fraccionada y la bilirrubina directa <35%)
c. Anomalías hepáticas o biliares conocidas, sin incluir el síndrome de Gilbert o los cálculos biliares asintomáticos
14. Antecedentes de infección por VIH o serología positiva para VIH en la selección.
15. Diagnóstico establecido de infección viral por hepatitis B, o positivo para HBsAg en la selección.
a. Los participantes que han ganado inmunidad para la infección por el virus de la hepatitis B después de la vacunación (por ejemplo, los participantes que son HBsAg negativos, HBsAb positivos, y HBcAb
negativos) son elegibles para el estudio.
b. Los participantes con HBcAbs positivos son elegibles para el estudio solo si el nivel de ADN del virus de la hepatitis B es indetectable.
16. Diagnóstico establecido de la infección viral por hepatitis C en el momento de la selección. Los participantes positivos para anticuerpos
contra la hepatitis C son elegibles para el estudio solo si el ARN del virus de la hepatitis C es negativo.
17. COVID-19 sintomática activa y actual, y/o diagnóstico previo de enfermedad moderada o grave u hospitalización por COVID-19.
18. Antecedentes de transplante de órganos.
19. Uso de vacunas vivas atenuadas en un plazo de 30 días de la visita de selección.
20. Necesidad de oxígeno suplementario >10l/min en la selección.
21. Exacerbación de una enfermedad pulmonar subyacente o infección pulmonar o respiratoria alta activa en los 30 días previos a la primera dosis del fármaco del estudio (podrá repetirse el proceso de selección en el momento adecuado).
22. Infección actual o reciente (últimos 30 días) de las vías respiratorias inferiores (puede repetirse el proceso de selección en el momento adecuado).
23. Cualquier forma de cardiopatía congénita o defecto cardíaco congénito (reparado o no reparado) distinto de un agujero oval permeable.
24. El participante tiene anemia, definida por una hemoglobina en la selección de valor < 9,0 g/dL, infección activa, o cualquier otra condición que en opinión del investigador interferiría con la interpretación de evaluaciones del estudio.
25. Índice de masa corporal ≥40 kg/m2 o ≤ 18 kg/m2.
26. Probabilidad razonable de recibir trasplante de pulmón dentro del período de estudio de 16 semanas, según lo determinado por el investigador.
27. Cualquier limitación física que impida al participante el uso del dispositivo inhalador o incapacidad para generar un flujo inspiratorio máximo de más de 60 L / min, según lo determinado por el investigador.

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

El criterio de valoración principal es la frecuencia y la intensidad de los acontecimientos adversos aparecidos durante el tratamiento (AAAT) y los AAG durante el estudio. Otros criterios de valoración de la seguridad son la variación de la oxigenación, determinada mediante pulsioximetría, antes, durante y después de la PM6M en el momento basal y las semanas 5, 10 y 16.

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 5, Week10, and Week 16

Semanas 5, 10 y 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

Los parámetros FC individuales de TRE y TP como los siguientes parámetros: Cmáx, Tmáx, AUC24, CL/F, t½ y Vd/F

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 10

Semana 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

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

Spain

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

El final del ensayo se define como la última llamada o visita de seguimiento del último participante en el estudio.
Se considera que un participante ha finalizado el ensayo si completa todas las visitas del estudio incluída la llamada o visita de seguimiento.

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.

El acceso continuado al fármaco del estudio (FE) dependerá de disponer de un estudio abierto de extensión (OLE). Los pacientes que han dejado el FE tras completar la visita de fin de tratamiento podrán incluirse en OLE en fecha posterior. Los que deseen continuar inmediatamente en OLE seguirán con FE ciego durante 30 días tras V7 como tratamiento de fin de estudio de extensión (TFEE) para facilitar el desenmasamiento para cada uno. Cuando finalice TFEE, se seleccionarán y consentirán para OLE.

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

2022-06-01

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-05-25

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

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