Clinical Trials Register

Summary
EudraCT Number:	2017-001746-10
Sponsor's Protocol Code Number:	CSEG101A2301
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:	2019-05-06
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

2017-001746-10

A.3

Full title of the trial

A phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab versus placebo, with or without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients with Vaso-Occlusive Crises (STAND)

Etude de phase III, multicentrique, randomisée, en double aveugle, contrôlée par placebo, évaluant l’efficacité et la tolérance de deux doses de crizanlizumab, avec ou sans hydroxyurée/hydroxycarbamide, chez des adolescents et des adultes atteints de drépanocytose accompagnée de crises vaso-occlusives (STAND)

A.3.1

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

Study of two doses of crizanlizumab versus placebo in adolescent and adult sickle cell disease patients

Etude de deux doses de crizanlizumab contrôlées par placebo chez des adolescents et des adultes atteints de drépanocytose

A.3.2

Name or abbreviated title of the trial where available

STAND

A.4.1

Sponsor's protocol code number

CSEG101A2301

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03814746

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	2 et 4 rue Lionel Terray
B.5.3.2	Town/ city	Rueil-Malmaison
B.5.3.3	Post code	92500
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/3/12/1034
D.3 Description of the IMP
D.3.1	Product name	crizanlizumab
D.3.2	Product code	SEG101
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	CRIZANLIZUMAB
D.3.9.2	Current sponsor code	SEG101
D.3.9.4	EV Substance Code	SUB188615
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	Concentrate for solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Sickle Cell Disease with vaso-occlusive crisis

Drépanocytose accompagnée de crises vaso-occlusives

E.1.1.1

Medical condition in easily understood language

Sickle Cell Disease

Drépanocytose

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

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	10040644
E.1.2	Term	Sickle cell disease
E.1.2	System Organ Class	100000004850

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10002077
E.1.2	Term	Anaemia sickle cell
E.1.2	System Organ Class	100000004850

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

- To compare the efficacy of 7.5 mg/kg of
crizanlizumab versus placebo on the annualized rate of VOC leading to healthcare visit, in addition to standard of care
- To compare the efficacy of 5.0 mg/kg of
crizanlizumab versus placebo on the annualized rate of VOC leading to healthcare visit, in addition to standard of care

- Comparer l’efficacité du crizanlizumab à 7,5 mg/kg versus placebo par rapport au taux annualisé de CVO conduisant à une consultation médicale, en plus des soins courants
- Comparer l’efficacité du crizanlizumab à 5,0 mg/kg versus placebo par rapport au taux annualisé de CVO conduisant à une consultation médicale, en plus des soins courants

E.2.2

Secondary objectives of the trial

Key secondary:
- To compare the efficacy of 7.5 mg/kg vs placebo on the annualized rate of all VOCs (managed at home + leading to healthcare visit)
- To compare the efficacy of 5.0 mg/kg vs placebo on the annualized rate of all VOC (managed at home + leading to healthcare visit)

- To assess the time to first and second VOC leading to healthcare visit in each group
- To assess the number of days with VOC leading to healthcare visit in each group
- To assess rate of subjects free from VOC leading to healthcare visit in each group
- Healthcare resource utilization (visits to clinic, Emergency room (ER) and hospitalizations) in each group vs placebo
- To assess SCD-related renal damage in each group
- To characterize PK profile of crizanlizumab at 5.0 and 7.5 mg/kg
- To characterize PD (P-selectin inhibition) of
crizanlizumab at 5.0 and 7.5 mg/kg
- To assess efficacy, safety and immunogenicity of crizanlizumab over the study period (treatment of 5 y + 105 d follow-up)

Objectif secondaire clé :
- Comparer l’efficacité du crizanlizumab à 7,5 mg/kg versus placebo par rapport au taux annualisé de l’ensemble des CVO (traitées à la maison + conduisant à une consultation médicale)
- Comparer l’efficacité du crizanlizumab à 5,0 mg/kg versus placebo par rapport au taux annualisé de l’ensemble des CVO (traitées à la maison + conduisant à une consultation médicale)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent must be obtained prior to any screening procedures
2. Male or female patients aged 12 years and older on the day of signing informed consent. Adolescent include patients aged 12 to 17 years old and adults ≥ 18 years and older
3. Confirmed diagnosis of SCD by hemoglobin
electrophoresis or high performance liquid
chromatography (HPLC) [performed locally]. All SCD genotypes are eligible, genotyping is not required for study entry
4. Experienced at least 2 VOCs leading to healthcare visit within the 12 months prior to screening visit as determined by medical history. Prior VOC leading to healthcare visit must include:
a. Pain crisis defined as an acute onset of pain for
which there is no other medically determined
explanation other than vaso- occlusion -
b. a visit to a medical facility and/or healthcare
professional,
c. and receipt of oral/parenteral opioids or parenteral nonsteroidal anti-inflammatory drug (NSAID) analgesia
As well as other complicated crises, such as acute
chest syndrome, priapism, and hepatic or splenic
sequestration
5. If receiving HU/HC or erythropoietin stimulating
agent or L-glutamine, must have been receiving the drug for at least 6 months prior to Screening visit and plan to continue taking at the same dose and schedule until the subject has reached one year of study treatment
6. Patients must meet the following central
laboratory values at the screening visit:
• Absolute Neutrophil Count ≥1.0 x 109/L
• Platelet count ≥75 x 109/L
• Hemoglobin: for adults (Hb) ≥4.0 g/dL and for
adolescents (Hb) ≥5.5 g/dL
• Glomerular filtration rate ≥ 45 mL/min/1.73 m2
using CKD-EPI formula in adults, and Shwartz formula in adolescents
• Direct (conjugated) bilirubin < 2.0 x ULN
• Alanine transaminase (ALT) < 3.0 x ULN
7. ECOG performance status ≤2.0 for adults and
Karnofsky ≥ 50% for adolescents

1. Le consentement éclairé écrit doit être obtenu avant toute procédure de sélection.
2. Patients de sexe masculin ou féminin âgés de plus de 12 ans le jour de la signature du formulaire de consentement. Les adolescents sont définis par les patients âgés de 12 à 17 ans et les adultes par les patients âgés de plus de 18 ans.
3. Patients atteints de drépanocytose dont le diagnostic a été confirmé (localement) par électrophorèse de l’hémoglobine ou par chromatographie liquide à haute performance (HPLC). Tous les génotypes de drépanocytose sont éligibles,
4. Patients ayant eu au moins 2 CVO conduisant à une consultation médicale au cours des 12 mois précédant la visite de sélection et documenté dans le dossier médical. Ces CVO conduisant à une consultation médicale doivent répondre aux critères suivants :
a. Se sont manifestées par des crises douloureuses, définies comme la survenue d’une douleur aiguë pour laquelle il n’y a pas d’autre explication médicale qu’une occlusion vasculaire,
b. Ont conduit à une visite dans un établissement de santé et/ou chez un professionnel de la santé,
c. Et ont nécessité un traitement par des opiacés par voie orale ou parentérale ou par des analgésiques de type anti-inflammatoires non stéroïdiens (AINS) par voie parentérale,
Ainsi que d’autres crises compliquées, comme le syndrome thoracique aigu, le priapisme, la séquestration hépatique ou splénique
5. Si les patients sont traités par l’HU/HC ou par la L-glutamine ou par un agent stimulant l’érythropoïèse, ils doivent avoir commencé ce traitement au moins 6 mois avant la visite de sélection et poursuivre ce traitement à la même dose et au même schéma d’administration jusqu’à 1 an après le début du traitement à l’étude.
6. Les patients doivent avoir les valeurs biologiques suivantes lors de la visite de sélection (analysées par le laboratoire central) :
• Nombre absolu de neutrophiles ≥ 1,0 x 109/l
• Plaquettes ≥ 75 x 109/l
• Hémoglobine ≥ 4,0 g/dl pour les adultes et ≥ 5,5 g/dl pour les adolescents
• Débit de filtration glomérulaire ≥ 45 ml/min/1,73 m² selon l’équation CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) pour les adultes et selon la formule de Schwartz pour les adolescents
• Bilirubine directe (conjuguée) < 2,0 x la limite supérieure de la normale (LSN)
• Alanine aminotransférase (ALAT) < 3,0 x LSN
7. Indices de performance : ECOG (pour Eastern Cooperative Oncology Group) ≤ 2,0 pour les adultes et Karnofsky ≥ 50 % pour les adolescents.

E.4

Principal exclusion criteria

1. History of stem cell transplant.
2. Participating in a chronic transfusion program
(pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted.
3. Contraindication or hypersensitivity to any drug or metabolites from similar class as study drug or to any excipients of the study drug formulation. History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction.
4. Received active treatment on another
investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to Screening visit or plans to participate in another investigational drug trial.
5. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant unless they are using highly effective methods of contraception during dosing and for 15 weeks after stopping treatment.
6. Concurrent severe and/or uncontrolled medical
conditions which, in the opinion of the Investigator, could cause unacceptable safety risks or compromise participation in the study.
7. History or current diagnosis of ECG abnormalities indicating significant risk of safety such as:
• Resting QTcF ≥470 msec at pretreatment
(baseline) for both male and female or inestability to determine QTc
• Concomitant clinically significant cardiac
arrhythmias (e.g ventricular tachycardia), and clinically significant second or third degree AV block without a pacemaker
• History of familial long QT syndrome or know
family history of Torsades de Pointes
8. Not able to understand and to comply with study intructions and requirements.

1. Antécédents de greffe de cellules souches.
2. Patients participant à un programme de transfusions sanguines régulières (série de transfusions planifiée à l’avance dans un but prophylactique) et/ou patients pour lesquels une exsanguino-transfusion et/ou une plasmaphérèse est prévue pendant la durée de l’étude ; les transfusions occasionnelles à cause de l’aggravation d’une anémie ou d’une CVO sont permises.
3. Contre-indication ou hypersensibilité à un médicament ou à des métabolites appartenant à une classe de médicament similaire à celle du traitement à l’étude ou à l’un des excipients présents dans sa formulation. Antécédents de réactions d’hypersensibilité sévères à tout autre anticorps monoclonal qui selon l’avis du médecin-investigateur pourrait augmenter le risque de réaction grave à la perfusion.
4. Patients ayant reçu un traitement actif dans le cadre d’une autre étude clinique au cours des 30 jours (ou de la période correspondant à 5 demi-vies de ce traitement, selon ce qui est le plus long) précédant la visite de sélection ou pour lesquels une participation à une autre étude clinique est prévue.
5. Femmes en âge d’avoir des enfants, c’est-à-dire toutes les femmes physiologiquement aptes à être enceintes, sauf si elles utilisent des méthodes de contraception très efficaces pendant la période d’administration du traitement à l’étude et les 15 semaines qui suivent l’arrêt du traitement à l’étude.
6. Pathologie concomitante sévère et/ou non contrôlée susceptible, selon le jugement du médecin-investigateur, d’induire des risques inacceptables pour la santé du patient ou de compromettre sa participation à l’étude.
7. électrocardiogrammes (ECG), qui constituent un risque significatif pour le patient telles que :
• Intervalle QT corrigé par la formule de Fridericia (QTcF) au repos ≥ 470 msec avant le traitement (baseline) pour les patients de sexe masculin ou féminin ou instabilité ne permettant pas de déterminer l’intervalle QTc.
• Arythmies cardiaques concomitantes cliniquement significatives (par ex. tachycardie ventriculaire) et bloc auriculo-ventriculaire de 2e ou 3e degré cliniquement significatif sans pacemaker.
• Antécédents familiaux de syndrome du QT long ou antécédents familiaux connus de Torsades de Pointes.
8. Patients qui ne sont pas en mesure de comprendre et de respecter les instructions et obligations de l’étude.

E.5 End points

E.5.1

Primary end point(s)

Annualized rate of VOC events leading to healthcare visit in each treatment group over the first year post-randomization

Taux annualisé de CVO conduisant à une consultation médicale dans chaque groupe de traitement au cours de la 1ère année suivant la randomisation

E.5.1.1

Timepoint(s) of evaluation of this end point

1 year

1 an

E.5.2

Secondary end point(s)

for key secondary:
- Annualized rate of all VOCs leading to healthcare
visit and treated at home (based on documentation by health care provider following contact with subject) over the first year post randomization

- The time to first and second VOC calculated
respectively as the time from date of randomization until the first and the second VOC leading to healthcare visit over the first year post randomization
- Number of days with VOC leading to healthcare
visit over the first year post randomization
- Number and percentage of subjects free from
VOCs leading to healthcare visit in each group
over the first year post randomization
- Annualized rate of visits to clinic, Emergency room (ER) and hospitalizations, both overall and VOCrelated over the first year post randomization
- Evolution of albuminuria and ACR over the first
year post randomization
- PK parameters after the first and fifth dose (e.g.,
AUC, Cmax, Tmax, half-life)
- PD parameter (P-selectin inhibition) after the first and fifth dose
- Annualized rate of VOCs leading to healthcare
visit
- Annualized rate of VOCs managed at home
- Number, seriousness, severity, and causality
assessments of treatment-emergent adverse
events, including infections (serious, non-serious
and opportunistic infections) and other safety data as considered appropriate
- Absolute change from baseline in hemoglobin
- Growth and sexual maturity assessment in
adolescents (Tanner stage)
- Immunogenicity: measurement of anti-drug
antibodies (ADA) to crizanlizumab

Objectif secondaire clé :
- Taux annualisé de l’ensemble des CVO conduisant à une consultation médicale ou traitées à la maison (documentées par un professionnel de la santé suite à un contact avec le patient) au cours de la 1ère année suivant la randomisation

- Délai jusqu’à la 1ère et la 2ème CVO calculé respectivement comme le délai entre la date de randomisation et la 1ère et la 2ème CVO conduisant à une consultation médicale au cours de la 1ère année suivant la randomisation
- Nombre de jours avec des CVO conduisant à une consultation médicale au cours de la 1ère année suivant la randomisation
- Nombre et pourcentage de patients sans CVO conduisant à une consultation médicale au cours de la 1ère année suivant la randomisation
- Taux annualisé de visites dans une clinique, aux urgences ou hospitalisations, globalement et en lien avec une CVO, au cours de la 1ère année suivant la randomisation
- Evolution de l’albuminurie et du rapport albumine/créatinine au cours de la 1ère année suivant la randomisation
- Paramètres PK après la 1ère et la 5ème dose (par ex. AUC, Cmax, Tmax, demi-vie)
- Paramètres PD (inhibition de la P sélectine ) après la 1ère et la 5ème dose
- Taux annualisé de CVO conduisant à une consultation médicale
-Taux annualisé de CVO traitées à la maison
- Nombre, gravité, intensité et lien de causalité des effets indésirables survenus sous traitement, y compris les infections (graves ou non et opportunistes) et autres données de tolérance jugées appropriées
- Variation absolue par rapport à la baseline de l’hémoglobine
- Evaluation de la croissance et du développement sexuel des adolescents (stade de Tanner)
- IG : mesures des anticorps anti-crizanlizumab

E.5.2.1

Timepoint(s) of evaluation of this end point

1 year

- 1 year
- 5 years
- 5 years
- 5 years
- 5 years
- 5 years
- 5 years
- 5 years

1 an

- 1 an
- 5 ans
- 5 ans
- 5 ans
- 5 ans
- 5 ans
- 5 ans
- 5 ans

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

Yes

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

healthcare resource utilization

Utilisation des resssources médicales

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

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

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)

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

Belgium

Brazil

Canada

Colombia

Egypt

Finland

France

Germany

Ghana

Greece

India

Italy

Kenya

Lebanon

Netherlands

Oman

Panama

Saudi Arabia

South Africa

Spain

Turkey

United Arab Emirates

United Kingdom

United States

Jordan

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

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

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

Yes

F.1.1

Number of subjects for this age range:

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

190

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

240

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)

Novartis will provide crizanlizumab to subjects who benefit from continued treatment as
per the Investigator's opinion after study completion until the earliest occurrence :
- Crizanlizumab is commercially available and reimbursed in the subject's country for this population, and subjects are eligible to be prescribed the commercial drug
- Another clinical study becomes available that can continue to provide crizanlizumab in
this population
- Subject is no longer deriving benefit

Novartis fournira, en fonction de l'opinion de l'investigteur, le crizanlizumab aux patients qui en tireraient bénéfices après la fin de l'étude jusqu'à l'une des situations ci-dessous selon celle qui aura lieu en premier:
- le Crizanlizumab est commercialisé et remboursé dans le pays du patient pour la population concernée
- un autre essai clinique est mis en place permettant de donner du Crizanlizumab à cette population de patients
- le patient ne tire plus de bénéfice

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

2019-06-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-05-15

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

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