Clinical Trials Register

Summary
EudraCT Number:	2018-004253-24
Sponsor's Protocol Code Number:	CLNP023B12001B
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2019-06-27
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

France - ANSM

A.2

EudraCT number

2018-004253-24

A.3

Full title of the trial

An open-label, non-randomized extension study to evaluate the long-term efficacy, safety and tolerability of LNP023 in subjects with C3 glomerulopathy

Etude d’extension en ouvert non randomisée, destinée à évaluer l'efficacité, la sécurité d'emploi et la tolérance à long terme du LNP023 chez des patients atteints de glomérulopathie C3

A.3.1

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

Long-term efficacy, safety and tolerability of LNP023 in C3G

Efficacité, sécurité et tolérabilité à long terme de LNP023 dans C3G

A.4.1

Sponsor's protocol code number

CLNP023B12001B

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
B.5.3.2	Town/ city	92563 Rueil Malmaison Cedex
B.5.3.3	Post code	CS 40150
B.5.3.4	Country	France
B.5.4	Telephone number	+33155476600
B.5.5	Fax number	+33155476100
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.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

C3 Glomerulopathy

Glomérulopathie C3

E.1.1.1

Medical condition in easily understood language

Kidney Disorder

Rein Disorder

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

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

•Primary Efficacy Objective: To characterize the effect of treatment with LNP023 on a composite renal response endpoint at the 9 months visit
•Primary Safety Objective: To evaluate the long-term safety and tolerability of LNP023 in subjects with C3G

•Objectif principal d’efficacité : Caractériser l'effet du traitement par LNP023 sur un critère composite de réponse rénale lors de la visite à 9 mois
•Objectif principal de sécurité d’emploi : Evaluer la sécurité d'emploi et la tolérance à long terme du LNP023 chez des patients atteints de glomérulopathie C3G

E.2.2

Secondary objectives of the trial

•To assess the long-term effect of LNP023 on renal function in C3G subjects at 9 months visit.
•To describe the status of C3G disease progression based on glomerular histopathology in a renal biopsy at 6-9 months.
•To evaluate the long-term effect of LNP023 on specific components of the complement pathway at the 9 months visit.
•To assess the long-term effect of LNP023 on urine markers of renal damage at the 9 months visit.
•To assess the longer term (>9 months of treatment in CLNP023B12001B) effects of LNP023 on the composite renal response endpoint, renal function , renal histopathology and specific components of the complement pathway in C3G subjects.
•To evaluate pharmacokinetics of LNP023 in subjects under prolonged treatment.

•Evaluer l’effet à long terme du LNP023 sur la fonction rénale chez des patients C3G lors de la visite à 9 mois
• Décrire le statut de progression de la C3G d’après l’histopathologie glomérulaire d'une biopsie rénale entre les visites à 6 et 9 mois
• Evaluer l’effet à long terme du LNP023 sur des composants spécifiques de la voie du complément lors de la visite de 9 mois
• Evaluer l’effet à long terme du LNP023 sur les marqueurs urinaires d’atteinte rénale lors de la visite à 9 mois
• Evaluer les effets à plus long terme (> 9 mois et jusqu'à 36 mois de traitement dans l’étude CLNP023B12001B) du LNP023 sur le critère composite de réponse rénale, la fonction rénale, l'histopathologie rénale et des composants spécifiques de la voie du complément chez des patients C3G
• Evaluer la pharmacocinétique du LNP023 chez des patients sous traitement prolongé

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Patients must have completed the treatment period of the CLNP023X2202 trial on study drug.

Les patients doivent avoir terminé la période de traitement de l’essai CLNP023X2202 sur le médicament à l’étude.

E.4

Principal exclusion criteria

•Severe concurrent co-morbidities, e.g. advanced cardiac disease (NYHA class IV), severe pulmonary arterial hypertension (WHO class IV), or any illness or medical condition that in the opinion of the investigator and sponsor is likely to prevent the patient from safely tolerating LNP023 or complying with the requirements of the study.
•Patients with an active infection requiring medical treatment.
•Chronic infection with Hepatitis B (HBV) or Hepatitis C (HCV) virus.
•History of HIV or any other immunodeficiency disease.
•History or current diagnosis of ECG abnormalities indicating significant risk of safety for subjects.

•Comorbidités concomitantes sévères, par ex. une maladie cardiaque avancée (classe NYHA IV), une hypertension artérielle pulmonaire sévère (classe OMS IV) ou toute maladie ou tout état pathologique qui, de l'avis de l'investigateur et du promoteur, est susceptible d'empêcher le patient de tolérer sans risque LNP023 ou de se conformer aux exigences du l'étude.
•Patients présentant une infection active nécessitant un traitement médical.
•Infection chronique par le virus de l'hépatite B (VHB) ou de l'hépatite C (VHC).
•Antécédents de VIH ou de toute autre maladie d'immunodéficience.
•Antécédents ou diagnostic actuel d'anomalies de l'ECG indiquant un risque significatif d'innocuité pour les sujets.

E.5 End points

E.5.1

Primary end point(s)

•A subject is defined as a responder for the composite renal response endpoint if they meet the following criteria at the 9 months visit in CLNP023B12001B: (1) a stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR), and (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or a reduction to <300 mg/g in UPCR and (3) an increase in C3 to ≥80% (i.e., ≥72 mg/dL or 0.72 g/L) of the lower limit of normal (LLN, 90 mg/dL or 0.9 g/L). Initiation of treatment with eculizumab or any other complement pathway modifying agent automatically designates the subject as a non-responder.

•Occurrence of clinically significant vital signs (msDBP, msSBP, heart rate), ECGs, and safety laboratory measurements, as well as adverse events (AEs), AEs of special interest, and study drug discontinuation due to an AE (or any safety issue).

•Une visite de 9 mois à CLNP023B12001B: (1) le DFGe stable ou amélioré par rapport à la visite initiale au CLNP023X2202 (réduction ≤ 10% du DFGP) est un sujet défini comme répondant pour la réponse du critère composite de l’élément terminal rénal, et (2) soit une réduction ≥ 50% par rapport à la valeur initiale dans CLNP023X2202, soit une réduction à <300 mg / g dans le RCUP et (3) une augmentation de C3 à ≥80% (soit ≥72 mg / dL ou 0,72 g / L) de la limite inférieure de la normale (LLN, 90 mg / dL ou 0,9 g / L). L'initiation du traitement par l'éculizumab ou tout autre agent modifiant la voie du complément identifie automatiquement le sujet comme non répondeur.
•Occurrence de signes vitaux cliniquement significatifs (MSDBP, MSSBP, fréquence cardiaque), de l'ECG et des mesures de sécurité en laboratoire, ainsi que d'événements indésirables (EI), d'EI présentant un intérêt particulier et de l'arrêt du médicament à l'étude en raison d'un EI (ou de tout problème de sécurité).

E.5.1.1

Timepoint(s) of evaluation of this end point

•9-months visit

•9-months visit, end of study

• visite de 9 mois

• visite de 9 mois, fin des études

E.5.2

Secondary end point(s)

•Change from baseline in log-transformed urine protein/creatinine ratio (UPCR), change from baseline in log-transformed urine albumin/creatinine ratio (UACR), change from baseline in serum creatinine concentration and change from baseline in estimated glomerular filtration rate (eGFR) at the 9 months visit in CLNP023B12001B.
•C3 deposit score, disease activity and chronicity scores from a renal biopsy at 6 to 9 months from entry to the CLNP023B12001B study. Biopsies will be compared to those obtained in the CLNP023X2202 study (if available).
•Change from baseline in circulating levels of complement components C3 (serum), change from baseline in Bb (plasma) and change from baseline in sC5b-9 (plasma) at the 9 months visit in CLNP023B12001B.
•Change from baseline in urinary kidney biomarkers such as neutrophil gelatinase-associated lipocalin (NGAL) at the 9 months visit in CLNP023B12001B compared to the baseline visit in CLNP023X2202.
•A subject is defined as a responder for the composite renal response endpoint if they meet the following criteria at times >9 months and up to 36 months in CLNP023B12001B: (1) a stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR), and (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or a reduction to <300 mg/g in UPCR and (3) an increase in C3 to ≥80% (i.e., ≥72mg/dL or 0.72 g/L) of the lower limit of normal (LLN, 90 mg/dL or 0.9 g/L). Initiation of treatment with eculizumab or any other complement pathway modifying agent automatically designates the subject as a non-responder.
•Change from baseline in log-transformed UPCR, log-transformed UACR, serum creatinine concentration and estimated glomerular filtration rate (eGFR) at times >9 months and up to 36 months in CLNP023B12001B.
•Change in C3 deposit score, disease activity and chronicity scores from a renal biopsy at times >9 months and up to 36 months from entry to the CLNP023B12001B study.
•Change from baseline in circulating levels of complement pathway components C3, Bb and sC5b-9 at times >9 months and up to 36 months in CLNP023B12001B.
•Determine plasma LNP023 concentration up to 12 months at trough

•Variation du ratio albumine / créatinine dans l'urine (RCAA) par rapport à la référence, modification de la concentration sérique de créatinine dans le sérum et variation du taux de filtration glomérulaire estimé (DFGe) par rapport à la référence ) lors de la visite de 9 mois dans CLNP023B12001B.
• Score de dépôt C3, scores d'activité de la maladie et de chronicité issus d'une biopsie rénale 6 à 9 mois après l'entrée dans l'étude CLNP023B12001B. Les biopsies seront comparées à celles obtenues dans l'étude CLNP023X2202 (si disponible).
• Variation par rapport aux valeurs initiales des taux circulants de composants C3 du sérum (sérum) en circulation, modification par rapport aux valeurs initiales dans Bb (plasma) et modification par rapport aux valeurs initiales dans sC5b-9 (plasma) à la visite de 9 mois dans CLNP023B12001B.
• Un sujet est défini comme répondeur pour le critère composite de la fonction rénale s'il répond aux critères suivants plus de 9 mois et jusqu'à 36 mois dans CLNP023B12001B: (1) un eGFR stable ou amélioré par rapport au niveau de base dans CLNP023X2202 (≤ Réduction de 10% du DFG), et (2) soit une réduction de ≥ 50% par rapport au niveau de base de CLNP023X2202, soit une réduction à <300 mg / g de RCPU et (3) une augmentation de C3 à ≥ 80% 72 mg / dL ou 0,72 g / L) de la limite inférieure de la normale (LLN, 90 mg / dL ou 0,9 g / L). L’initiation du traitement par eculizumab ou tout autre agent modifiant la voie du complément identifie automatiquement le sujet comme non répondeur.
• Changement par rapport au niveau initial du taux de PCRA transformé par logarithérapie, du taux de UACR transformé par logarithme, de la concentration sérique de créatinine et du débit de filtration glomérulaire estimé (DFG) à des périodes> 9 mois et jusqu'à 36 mois chez CLNP023B12001B.
•Changement du score de dépôt C3, de l'activité de la maladie et des scores de chronicité d'une biopsie rénale à des périodes> 9 mois et jusqu'à 36 mois à compter de l'entrée dans l'étude CLNP023B12001B.
• Changement par rapport aux valeurs de départ des niveaux circulants des composants de la voie du complément C3, Bb et sC5b-9 à des périodes> 9 mois et jusqu'à 36 mois dans CLNP023B12001B.
• Déterminez la concentration plasmatique de LNP023 jusqu'à 12 mois à la fois.

E.5.2.1

Timepoint(s) of evaluation of this end point

•9-months visit
•6 to 9 months
•9-months visit
•9-months visit
• > 9 months
•3-months, 6-months, 9-months and 12-months visits

•Visite de 9 mois
• 6 à 9 mois
• visite de 9 mois
• visite de 9 mois
•> 9 mois
• Visites de 3, 6, 9 et 12 mois

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

Yes

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

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

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

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

France

Germany

Italy

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)

Participants will switch to commercial drug as study will end when the drug is commercially available

Les participants passeront à des médicaments commerciaux car une étude sera disponible lorsque le médicament sera disponible dans le commerce.

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

2019-07-12

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-08-28

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

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IMP with orphan designation in the indication
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