Clinical Trials Register

Summary
EudraCT Number:	2020-004385-19
Sponsor's Protocol Code Number:	CLNP023C12001B
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2021-07-15
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

2020-004385-19

A.3

Full title of the trial

An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan.

Etude d’extension en ouvert, multicentrique, ayant pour but de caractériser la tolérance et la sécurité d’emploi à long terme de l’iptacopan (LNP023) chez des patients atteints d’hémoglobinurie paroxystique nocturne (HPN) ayant terminé des études de phase II et III évaluant l’iptacopan

A.3.1

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

Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria

Sécurité et tolérance à long terme de l'iptacopan chez les patients atteints d’hémoglobinurie paroxystique nocturne (HPN)

A.4.1

Sponsor's protocol code number

CLNP023C12001B

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 Henri 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	+33 1 5547 6600
B.5.5	Fax number	+33 1 5547 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	Yes
D.2.5.1	Orphan drug designation number	EU/3/20/2281
D.3 Description of the IMP
D.3.1	Product name	iptacopan
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	iptacopan
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	200
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/20/2281
D.3 Description of the IMP
D.3.1	Product name	iptacopan
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	iptacopan
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	10
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

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Hémoglobinurie paroxystique nocturne (HPN)

E.1.1.1

Medical condition in easily understood language

Blood disorder

Maladie du sang

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	PT
E.1.2	Classification code	10034042
E.1.2	Term	Paroxysmal nocturnal haemoglobinuria
E.1.2	System Organ Class	10038359 - Renal and urinary disorders

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

Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit

Évaluations de la sécurité, y compris, mais sans s'y limiter, les événements indésirables/événements indésirables graves, les paramètres de laboratoire de sécurité, les signes vitaux, etc. jusqu'à la visite de fin d'étude

E.2.2

Secondary objectives of the trial

1. Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
2. Proportion of participants who remain free from transfusions evaluated over yearly intervals
3. Rate of breakthrough hemolysis (BTH)
4. Proportion of participants with Major Adverse Vascular Events MAVEs (incl. thrombosis) evaluated over yearly intervals

1. Proportion de participants ayant un maintien durable de l’hémoglobine à un taux ≥ 12 g/dl sans recours à des transfusions de globules rouges
2. Proportion de patients ne nécessitant pas de transfusions (évaluation annuelle)
3. Taux de poussées d'hémolyse
4. Proportion de participants présentant des événements vasculaires indésirables majeurs (y compris thrombose) évalués à des intervalles annuels

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed Phase 2 or Phase 3 clinical studies
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
- Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months

- Patients de sexe masculin ou féminin âgés de ≥ 18 ans, diagnostiqués avec une HPN, ayant terminé les études cliniques de phase 2 ou de phase 3
- Les vaccinations antérieures contre Neisseria meningitidis, Streptococcus pneumoniae et Haemophilus influenzae doivent être à jour
- Patients cliniquement stables sous traitement par LNP023 en monothérapie depuis au moins 3 mois et pour lesquels la poursuite de ce traitement pourrait être bénéfique selon l’avis du médecin-investigateur

E.4

Principal exclusion criteria

- Participants who in the parent PNH studies either screen or baseline failed, or prematurely withdrew from the study for any reason
- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
- History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- History of hematopoietic stem cell transplantation
Other protocol-defined inclusion/exclusion criteria may apply.

- Patients n’ayant pas rempli les critères de sélection ou de la baseline dans les études parentes dans l’HPN ou ayant prématurément arrêté l’étude quelle qu’en ait été la raison
- Toute comorbidité ou pathologie (incluant entre autres : infection bactérienne, virale ou fongique systémique active ou cancer) qui pourrait accroître le risque pour le patient ou potentiellement influencer les données de l’étude selon le médecin-investigateur
- Antécédents d’infections invasives récurrentes dues à des organismes encapsulés tels que Neisseria meningitidis, Streptococcus pneumoniae ou Haemophilus influenzae
- Antécédents de greffe de cellules souches hématopoïétiques
D'autres critères d'inclusion/exclusion définis par le protocole peuvent s'appliquer.

E.5 End points

E.5.1

Primary end point(s)

Proportion of participants with adverse events

Proportion de participants présentant des événements indésirables

E.5.1.1

Timepoint(s) of evaluation of this end point

Time Frame: 60 months

Délai : 60 mois

E.5.2

Secondary end point(s)

1. Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
2. Proportion of participants who remain free from transfusions
3. Rate of breakthrough hemolysis (BTH)
4. Proportion of participants with Major Adverse Vascular Events MAVEs

1. Proportion de participants ayant un maintien durable de l’hémoglobine à un taux ≥ 12 g/dl sans recours à des transfusions de globules rouges
2. Proportion de patients ne nécessitant pas de transfusions
3. Taux de poussées d'hémolyse
4. Proportion de participants présentant des événements vasculaires indésirables majeurs

E.5.2.1

Timepoint(s) of evaluation of this end point

Time Frame: 60 months

Délai : 60 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

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

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

Brazil

China

Japan

Korea, Republic of

Malaysia

Singapore

Taiwan

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

Dernière visite du dernier patient

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:

157

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

165

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

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

2021-08-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-08-31

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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