Clinical Trials Register

Summary
EudraCT Number:	2019-001734-34
Sponsor's Protocol Code Number:	CLNP023D12201
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2019-09-13
Trial results	View 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

France - ANSM

A.2

EudraCT number

2019-001734-34

A.3

Full title of the trial

A randomized, treatment open-label, dose-blinded, parallel group, three arm, proof-of-concept clinical trial to investigate the efficacy and safety of LNP023 compared with rituximab in the treatment of subjects with idiopathic membranous nephropathy.

Etude clinique de preuve de concept randomisée, à 3 groupes parallèles, avec traitement en ouvert et posologies en aveugle, visant à étudier l'efficacité et la sécurité d’emploi du LNP023 par rapport au rituximab dans le traitement des patients atteints de glomérulonéphrite extra-membraneuse idiopathique.

A.3.1

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

Efficacy and safety of LNP023 compared with rituximab in subjects with idiopathic membranous nephropathy.

Efficacité et innocuité de LNP023 par rapport au rituximab chez les sujets atteints de glomérulonéphrite extra-membraneuse idiopathique.

A.4.1

Sponsor's protocol code number

CLNP023D12201

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	Novartis Pharma AG
B.1.3.4	Country	Switzerland
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	Novartis Pharma AG
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novartis Pharma S.A.S
B.5.2	Functional name of contact point	Information&Communication Médicales
B.5.3	Address:
B.5.3.1	Street Address	8/10 rue Henry Sainte Claire Deville, CS 40150
B.5.3.2	Town/ city	Rueil-Malmaison
B.5.3.3	Post code	92563
B.5.3.4	Country	France
B.5.4	Telephone number	+3315547 6600
B.5.5	Fax number	+3315547 6100
B.5.6	E-mail	icm.phfr@novartis.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.3.2	Product code	LNP023
D.3.4	Pharmaceutical form	Capsule, hard
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	not yet established
D.3.9.2	Current sponsor code	LNP023
D.3.9.3	Other descriptive name	LNP023 HYDROCHLORIDE SALT
D.3.9.4	EV Substance Code	SUB180361
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	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	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.3.2	Product code	LNP023
D.3.4	Pharmaceutical form	Capsule, hard
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	not yet established
D.3.9.2	Current sponsor code	LNP023
D.3.9.3	Other descriptive name	LNP023 HYDROCHLORIDE SALT
D.3.9.4	EV Substance Code	SUB180361
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	25
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	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.3.2	Product code	LNP023
D.3.4	Pharmaceutical form	Capsule, hard
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	not yet established
D.3.9.2	Current sponsor code	LNP023
D.3.9.3	Other descriptive name	LNP023 HYDROCHLORIDE SALT
D.3.9.4	EV Substance Code	SUB180361
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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	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: 4
D.1.2 and D.1.3	IMP Role	Comparator
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	Yes
D.2.1.1.1	Trade name	MabThera 100 mg concentrate for solution for infusion
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration GmbH
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RITUXIMAB
D.3.9.1	CAS number	174722-31-7
D.3.9.4	EV Substance Code	SUB12570MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
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	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Idiopathic Membranous nephropathy

Néphropathie membraneuse idiopathique

E.1.1.1

Medical condition in easily understood language

Kidney disease

Maladie rénale

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10027170
E.1.2	Term	Membranous nephropathy
E.1.2	System Organ Class	100000004857

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the efficacy of high dose LNP023 compared with rituximab

Évaluer l'efficacité de LNP023 à forte dose par rapport au rituximab

E.2.2

Secondary objectives of the trial

- To assess the safety and tolerability of low and high dose of LNP023
- To assess the relationship between LNP023 systemic drug exposure and pharmacodynamics, mode-of-action markers and clinical efficacy
- To assess the difference in response between low (regimen A) and high (regimen B) dose of LNP023
- To assess the effect of LNP023 compared with rituximab on proteinuria remission and renal function
- To assess the pharmacokinetics of LNP023

- Évaluer la relation entre l'exposition systémique au médicament LNP023 et la pharmacodynamique, les marqueurs de mode d'action et l'efficacité clinique
- Évaluer la différence de réponse entre les doses faible (régime A) et élevée (régime B) de LNP023
- Évaluer l'effet du LNP023 par rapport au rituximab sur la rémission de la protéinurie et la fonction rénale
- Évaluer la pharmacocinétique de LNP023

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Female or male adult (≥18 years) subjects at screening visit with a diagnosis of idiopathic (primary) MN confirmed by renal biopsy within 24 months prior to screening. A renal biopsy may be taken at any time during the run-in period to confirm the diagnosis of MN and facilitate subject eligibility, if the most recent biopsy was performed greater than 24 months prior to the screening visit.
• Anti-PLA2R antibody titer of ≥ 100 RU/mL at screening visit (based on the EuroImmun ELISA test)
• Urine protein ≥ 3.5 g/24h at run-in and baseline visits
• ≤50% reduction in both anti-PLA2R level and 24h urine protein between first measurement at screening or run-in visit and baseline
• Estimated GFR (using the CKD-EPI formula) ≥ 45 mL/min per 1.73 m2 at run-in visit
• Receiving stable dose at the maximum recommended dose according to local guidelines or maximum tolerated dose of ACEi and/or ARB and/or statins and/or diuretics for at least 8 weeks prior to Day 1
• Vaccination against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae (in accordance with local guidelines) at least 28 days prior to Day 1 and no longer than 5 years prior to Day 1

• Sujets femmes ou hommes adultes (≥ 18 ans) à la visite de dépistage avec un diagnostic de MN idiopathique (primaire) confirmé par biopsie rénale dans les 24 mois précédant le dépistage. Une biopsie rénale peut être pratiquée à tout moment pendant la période de rodage pour confirmer le diagnostic de MN et faciliter l’éligibilité du sujet, si la biopsie la plus récente a été réalisée plus de 24 mois avant la visite de sélection.
• Titre en anticorps anti-PLA2R ≥ 100 RU / mL lors de la visite de dépistage (basé sur le test ELISA EuroImmun)
• Protéine urinaire ≥ 3,5 g / 24h lors des visites de rodage et de référence
• Réduction ≤50% du taux d’anticorps anti-PLA2R et de la protéine urinaire sur 24 heures entre la première mesure à la visite de dépistage ou de rodage et la référence
• DFG estimé (en utilisant la formule CKD-EPI) ≥ 45 mL / min par 1,73 m2 lors de la visite de rodage
• Recevoir la dose stable à la dose maximale recommandée conformément aux directives locales ou à la dose maximale tolérée d’ACEI et / ou de BRA et / ou de statines et / ou de diurétiques pendant au moins 8 semaines avant le jour 1;
• Vaccination contre Neisseria meningitidis, Streptococcus pneumoniae et Haemophilus influenzae (conformément aux directives locales) au moins 28 jours avant le jour 1 et au maximum 5 ans avant le jour 1

E.4

Principal exclusion criteria

• Secondary causes of MN, e.g. systemic autoimmune diseases, solid or haematological malignancies, infections or chronic intake of drugs (e.g. gold salts, NSAIDs, penicillamines)
• Diagnostic renal biopsy showing evidence of crescent formation in glomeruli, suggestive of an alternative or additional diagnosis to primary idiopathic MN
• Previous treatment with B-cell depleting or B-cell modifying agents such as, but not limited to rituximab, belimumab, daratumomab or bortezomib
• Previous treatment with immunosuppressive agents such as cyclophosphamide, chlorambucil, mycophenolate mofetil (or equivalent), cyclosporine, tacrolimus or azathioprine within 90 days prior to Day 1. Low dose systemic corticosteroid therapy is permitted, though the subject should have been on stable dose equivalent to ≤10 mg prednisolone for at least 90 days prior to Day 1
• Previous treatment with gemfibrozil or strong CYP2C8 inhibitors such as clopidogrel within 7 days prior to Day 1
• Presence or suspicion (based on judgment of the investigator) of active infection within 30 days prior to Day 1, or history of severe recurrent bacterial infections

• Causes secondaires de MN, par exemple maladies auto-immunes systémiques, tumeurs malignes solides ou hématologiques, infections ou consommation chronique de médicaments (par exemple, sels d'or, AINS, pénicillamines)
• Biopsie rénale diagnostique montrant des signes de formation de croissant dans les glomérules, suggérant un diagnostic alternatif ou complémentaire au MN primitif idiopathique.
• Traitement antérieur avec des agents réduisant les cellules B ou modifiant le groupe B, tels que, sans toutefois s'y limiter, le rituximab, le belimumab, le daratumomab ou le bortézomib
• Traitement antérieur avec des immunosuppresseurs tels que cyclophosphamide, chlorambucil, mycophénolate mofétil (ou équivalent), cyclosporine, tacrolimus ou azathioprine dans les 90 jours précédant le jour 1. Un traitement par corticostéroïde systémique à faible dose est autorisé, bien que le sujet ait dû suivre un traitement à dose équivalente ≤10 mg de prednisolone pendant au moins 90 jours avant le jour 1
• Traitement antérieur par le gemfibrozil ou des inhibiteurs puissants du CYP2C8 tels que le clopidogrel dans les 7 jours précédant le jour 1
• Présence ou suspicion (selon le jugement de l'investigateur) d'infection active dans les 30 jours précédant le jour 1, ou antécédents d'infections bactériennes graves récurrentes

E.5 End points

E.5.1

Primary end point(s)

Ratio between baseline UPCR and UPCR at 24 weeks of treatment (from 24h urine collection)

Rapport entre le RCPU de base et le RCPU à 24 semaines de traitement (à partir de la collecte d'urine sur 24h)

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline, Day 113 and Day 169

Niveau de référence, jour 113 et jour 169

E.5.2

Secondary end point(s)

• ECG parameters, vital signs, safety laboratory data, physical exam and collection of AEs
• Plasma levels of Bb and sC5b-9
• UPCR measured in first morning void
• Ratio between baseline UPCR and UPCR at 24 weeks of treatment (from 24h urine collection)
• Proportion of subjects with a complete remission, defined as proteinuria ≤0.3 g/day at 24 weeks of treatment, derived from 24h urine collection
• Proportion of subjects with a partial remission, defined as reduction of proteinuria from baseline ≥50% plus final UP ≤ 3.5 g/24h but >0.3 g/24h at 24 weeks of treatment, derived from 24h urine collection.
• Change in eGFR applying the Chronic Kidney Disease Epidemiology Collaboration (CKD- EPI) equation from baseline to 24 weeks of treatment
• Plasma: non-compartmental parameters in plasma related to total parent drug, including, but not limited, to Tmax, Cmax, AUClast and AUCtau will be calculated for each dose level.
• Urine: renal plasma clearance derived from 24h urine at week 16.

• Paramètres ECG, signes vitaux, données de laboratoire sur la sécurité, examen physique et collecte des EI
• Niveaux plasmatiques de Bb et de sC5b-9
• RCUP mesuré dans le premier vide du matin
• Rapport entre RCPU de base et RCPP à 24 semaines de traitement (à partir de la collecte d'urine sur 24h)
• Proportion de sujets en rémission complète, définie par une protéinurie ≤ 0,3 g / jour à 24 semaines de traitement, dérivée de la collecte d'urine sur 24 h
• Proportion de sujets en rémission partielle, définie comme une réduction de la protéinurie par rapport aux valeurs initiales ≥ 50% plus un UP final ≤ 3,5 g / 24h mais> 0,3 g / 24h à 24 semaines de traitement, obtenue à partir d'un prélèvement d'urine de 24h.
• Modification du dFGe en appliquant l'équation de la collaboration CKD-EPI selon l'équation de la maladie rénale chronique (EPC) du traitement initial à 24 semaines de traitement.
• Plasma: les paramètres plasmatiques non compartimentés liés au médicament-mère total, y compris, sans s'y limiter, le Tmax, le Cmax, l'AUClast et l'ASCtau, seront calculés pour chaque niveau de dose.
• Urine: clairance plasmatique rénale dérivée de l’urine de 24 heures à la 16e semaine.

E.5.2.1

Timepoint(s) of evaluation of this end point

• Baseline, Day 1, Day 15, Day 29, Day 57, Day 85, Day 113, Day 141, Day 169, Day 266, EOS. Collection of AEs is continuous.
• Baseline, Day 15, Day 29, Day 57, Day 113, Day 169, Day 266, EOS
• Baseline, Day 15, Day 29, Day 57, Day 85, Day 113, Day 169, Day 266, EOS
• Baseline, Day 113 and Day 169
• Baseline, Day 113 and Day 169
• Baseline, Day 113 and Day 169
• Baseline, Day 15, Day 29, Day 57, Day 85, Day 113, Day 141, Day 169, Day 266, EOS
• Day 15, Day 29, Day 57, Day 113, Day 169
• Day 113

• Ligne de base, Jour 1, Jour 15, Jour 29, Jour 57, Jour 85, Jour 113, Jour 141, Jour 169, Jour 266, EOS. La collection d'EI est continue.
• Ligne de base, jour 15, jour 29, jour 57, jour 113, jour 169, jour 266, EOS
• Ligne de base, jour 15, jour 29, jour 57, jour 85, jour 113, jour 169, jour 266, EOS
• Niveau de référence, jours 113 et 169
• Niveau de référence, jours 113 et 169
• Niveau de référence, jours 113 et 169
• Ligne de base, jour 15, jour 29, jour 57, jour 85, jour 113, jour 141, jour 169, jour 266, EOS
• Jour 15, jour 29, jour 57, jour 113, jour 169
• Jour 113

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

Yes

E.6.13.1

Other scope of the trial description

Tolerability

Tolérabilité

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

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

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)

Yes

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

Czech Republic

France

Germany

India

Netherlands

Russian Federation

Singapore

Spain

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

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)

None

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

2019-10-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-10-23

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2023-01-20

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