Clinical Trials Register

Summary
EudraCT Number:	2022-002691-36
Sponsor's Protocol Code Number:	CVAY736F12301
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:	2022-11-01
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

2022-002691-36

A.3

Full title of the trial

A randomized, double-blind, parallel group, placebo-controlled multicenter phase 3 study to evaluate efficacy, safety and tolerability of two regimens of ianalumab on top of standard-of-care therapy in patients with systemic lupus erythematosus (SIRIUS-SLE 1)

Estudio de fase III, multicéntrico, aleatorizado, doble ciego, de grupos paralelos y controlado con placebo en el que se evalúan la eficacia, la seguridad y la tolerabilidad de dos pautas posológicas de ianalumab en combinación con el tratamiento de referencia en pacientes con lupus eritematoso sistémico (SIRIUS-SLE 1)

A.3.1

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

Phase 3 study to evaluate two regimens of ianalumab on top of standard-of-care therapy in patients with systemic lupus erythematosus (SIRIUS-SLE 1)

Estudio de fase III en el que se evalúan dos pautas posológicas de ianalumab en combinación con el tratamiento de referencia en pacientes con lupus eritematoso sistémico (SIRIUS-SLE 1)

A.4.1

Sponsor's protocol code number

CVAY736F12301

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 Farmacéutica, S.A.
B.1.3.4	Country	Spain
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 Farmacéutica, S.A.
B.5.2	Functional name of contact point	Trial Monitoring Organization (TMo)
B.5.3	Address:
B.5.3.1	Street Address	Gran Via de les Corts Catalanes, 764
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08013
B.5.3.4	Country	Spain
B.5.4	Telephone number	34 90 0353036
B.5.5	Fax number	34 93 2479903
B.5.6	E-mail	eecc.novartis@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.3.1	Product name	ianalumab
D.3.2	Product code	VAY736
D.3.4	Pharmaceutical form	Solution for injection in pre-filled syringe
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	IANALUMAB
D.3.9.1	CAS number	1929549-92-7
D.3.9.2	Current sponsor code	VAY736
D.3.9.3	Other descriptive name	VAY736
D.3.9.4	EV Substance Code	SUB193896
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	150
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	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	Yes
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	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Systemic Lupus Erythematosus

Lupus eritematoso sistémico

E.1.1.1

Medical condition in easily understood language

An inflammatory disease caused when the immune system attacks its own tissues.

Una enfermedad inflamatoria causada cuando el sistema inmune ataca sus propios tejidos.

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	10042945
E.1.2	Term	Systemic lupus erythematosus
E.1.2	System Organ Class	10028395 - Musculoskeletal and connective tissue disorders

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 demonstrate superiority of subcutaneous (s.c.) ianalumab 300 mg monthly, compared to placebo, in achieving Systemic Lupus Erythematosus Responder Index (SRI-4) at Week 60

Demostrar la superioridad de ianalumab 300 mg subcutáneo una vez al mes, comparado con placebo, para alcanzar una respuesta SRI-4 en el Systemic Lupus Erythematosus Responder Index en la semana 60.

E.2.2

Secondary objectives of the trial

To demonstrate superiority of s.c. ianalumab 300 mg monthly compared to placebo in: Reducing moderate or severe British Isles Lupus Assessment Group flares up to Week(Wk) 60; Maintaining corticosteroid dose ≤5 mg/day or ≤baseline dose, whichever is lower, b/w Wk 36 & Wk 60; Achieving British Isles Lupus Assessment Group-based Composite Lupus Assessment at Wk 60; Achieving Lupus Low Disease Activity State at Wk 60; Time to first occurrence of SRI-4 from baseline up to Wk 60; Achieving SRI-4 at Wk 60 while maintaining reduced corticosteroid dose ≤5 mg/day or ≤baseline dose, whichever is lower, b/w Wk 36 & Wk 60; Achieving SRI-6 at Wk 60; Maintaining corticosteroid dose ≤5mg/day or ≤baseline dose, whichever is lower, b/w Wk 24 & Wk 60

To demonstrate superiority of ianalumab 300 mg s.c. quarterly compared to placebo for SRI-4 & secondary objectives listed above
To evaluate: safety & tolerability, immunogenicity& characterize the PK of ianalumab 300 mg s.c (monthly or quarterly)

Demostrar la superioridad de ianalumab 300 mg subcutáneo una vez al mes comparado con placebo para: Reducir los brotes moderados o graves según el British Isles Lupus Assessment Group hasta la semana 60; Mantener la dosis de corticosteroides <=5 mg/día o <= dosis basal, aquella que sea inferior, entre la semana 36 y la semana 60; Alcanzar una respuesta BICLA (BILAG-based Composite Lupus Assessment) en la semana 60; Alcanzar un estado bajo de actividad del lupus (LLDAS) en la semana 60;Tiempo hasta la primera respuesta SRI-4 desde la basal hasta la semana 60; Alcanzar una respuesta SRI-4 en la semana 60 y mantener a su vez una dosis reducida de CS <=5 mg/día o <= dosis basal, aquella que sea inferior, entre la semana 36 y la semana 60; Alcanzar una respuesta SRI-6 en la semana 60;Mantener la dosis de corticosteroides <=5 mg/día o <= dosis basal, aquella que sea inferior, entre la semana 24 y la semana 60.

Ver protocolo para otros objetivos

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Male and Female participants aged 12 years or older at the time of screening, or limited to 18 years or older in European Economic Area countries and other countries where inclusion of participants below 18 years is not allowed.

Diagnosis of systemic lupus erythematosus (SLE) according to EULAR/ACR SLE classification criteria at least 6 months prior to screening

Elevated serum titers at screening of Antinuclear Antibodies (≥1:80) as determined by a central laboratory with a SLE typical fluorescence pattern.

Currently receiving corticosteroids and/or anti-malarial treatment and/or another Disease-modifying antirheumatic drug (DMARD) as specified in the protocol.

SLEDAI-2K Criteria at screening: SLEDAI-2K score ≥6 points, excluding points attributed to “fever”, “lupus headache”, "alopecia", and “organic brain syndrome”
British Isles Lupus Assessment Group-2004 disease activity level at screening of at least 1 of the following:
British Isles Lupus Assessment Group-2004 level A disease in ≥1 organ system,
Or
British Isles Lupus Assessment Group-2004 level B disease in ≥2 organ systems

Weigh at least 35 kg at screening

Participantes de ambos sexos a partir de 12 años de edad en el momento de la selección o a partir de 18 años de edad en países del Espacio Económico Europeo (EEE) y otros países donde no esté permitida la inclusión de participantes menores de 18 años.

Diagnóstico de lupus eritematoso sistémico conforme a los criterios de clasificación LES de la EULAR/ACR al menos 6 meses antes de la selección.

Títulos séricos elevados en la selección de anticuerpos antinucleares (>=1:80) determinados por un laboratorio central con un patrón de fluorescencia característico del LES.

Pacientes que actualmente reciban corticosterioides y/o tratamiento antimalárico y/u otro fármaco antirreumático modificador de la enfermedad (FAME) como se especifica en el protocolo.

Criterios SLEDAI-2K en la selección: Puntuación del SLEDAI-2K >=6 puntos, excepto los puntos atribuidos a "fiebre", "cefalea por lupus", "alopecia" y "síndrome cerebral orgánico".
Nivel de actividad de la enfermedad según BILAG-2004 en la selección de al menos uno de los siguientes:
Enfermedad de nivel "A" según BILAG-2004 en >=1 sistema orgánico.
O
Enfermedad de nivel "B" según BILAG-2004 en >=2 sistemas orgánicos.
Peso de al menos 35 kg en la selección.

E.4

Principal exclusion criteria

Prior treatment with Ianalumab

History of receiving following treatment I) high dose corticosteroids, calcineurin inhibitors, JAK or other kinase inhibitors or other DMARD (except as listed in inclusion criteria) 12 weeks prior to screening II) Cyclophosphamide or biologics such as immunoglobulins (i.v. or s.c.), plasmapheresis, anti-type I interferon receptor biologic agents, anti-CD40 agents, CTLA4-Fc Ig or B-cell activating factor-targeting agents administered within 24 weeks prior to screening; belimumab administered within 12 weeks prior to screening. III) Any B-cell depleting therapies, other than ianalumab administered within 36 weeks prior to randomization or as long as B cell count is less than the lower limit of normal or baseline value prior to receipt of B cell-depleting therapy (whichever is lower)

Active viral, bacterial or other infections requiring systemic treatment at the time of screening or randomization or history of recurrent clinically significant infection

Chronic infection with hepatitis B (HBV) or hepatitis C (HCV)

Evidence of active tuberculosis infection

History of primary or secondary immunodeficiency, including a positive human immunodeficiency virus (HIV) test result at screening

Any one of the following laboratory values prior to randomization
Platelets <25000/mm^3 (<25 x 10^3/μL)
Hemoglobin (Hgb) <8.0 g/dL (<5 mmol/Lg), or <7.0 g/dL (<4.3 mmol/L) if related to participant's SLE such as in active hemolytic anemia
Absolute neutrophil count (ANC) (<0.8 x 10^3/ μL)

Severe organ dysfunction or life-threatening disease at screening

History of major organ, hematopoietic stem cell or bone marrow transplant

Presence of severe lupus kidney disease as defined by proteinuria above 2g/day or equivalent using spot urine protein creatinine ratio

Receipt of live/attenuated vaccine within a 4-week period before first dosing

Any uncontrolled, co-existing serious disease, which in the opinion of the investigator will place the participant at risk for participation or interfere with evaluation for SLE-related symptoms

History of hypersensitivity to drugs of similar class or oral CS or to any of the constituents of the study drugs (sucrose, L-histidine hydrochloride, L-histidine, polysorbate 20)

History of non-compliance with medical regiments or considered potentially unreliable

Use of other investigational drugs within 5 half lives of enrollment or within 30 days or until the expected pharmacodynamic effect has returned to baseline, whichever is longer, or longer if required by local regulations

Non-lupus conditions such as asthma, gout or urticaria, requiring intermittent or chronic treatment with systemic corticosteroids

History of malignancy of any organ system other than localized basal cell carcinoma of the skin or in situ cervical cancer

Pregnant or nursing (lactating) women.

Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while on study treatment and for 6 months after stopping of investigational drug.

US (and other countries, if locally required): sexually active males unless using barrier protection during intercourse while taking study treatment

Any surgical, medical, psychiatric or additional physical condition that may jeopardize participation in this study

Tratamiento previo con ianalumab.
Antecedentes de haber recibido alguno de los siguientes tratamientos: 1) dosis alta de corticosteroides, inhibidor de la calcineurina, inhibidores de JAK u otras quinasas u otros FAME (excepto los indicados en los criterios de inclusión) administrados durante las 12 semanas anteriores a la selección; 2) ciclofosfamida o productos biológicos como inmunoglobulinas (i.v. o s.c.), plasmaféresis, fármacos biológicos contra los receptores de interferón tipo 1, fármacos anti-CD40, fármacos dirigidos a Ig CTLA4-Fc o al factor activador de células B (BAFF) administrados durante las 24 semanas anteriores a la selección; belimumab administrado durante las 12 semanas anteriores a la selección. 3) cualquier tratamiento de depleción de células B, salvo ianalumab, administrado durante las 36 semanas anteriores a la aleatorización o siempre y cuando el recuento de células B sea inferior al límite inferior de normalidad o al valor basal antes de recibir el tratamiento de depleción de células B (aquel que sea inferior).
Infecciones víricas, bacterianas o de otro tipo que estén activas y requieran tratamiento sistémico en el momento de la selección o la aleatorización o antecedentes de infección recurrente de interés clínico.
Infección crónica por virus de la hepatitis B (VHB) o la hepatitis C (VHC).
Signos de infección tuberculosa activa.
Antecedentes de inmunodeficiencia primaria o secundaria, incluido un resultado positivo en la prueba de virus de la inmunodeficiencia humana (VIH) en la selección.
Cualquiera de los siguientes valores de laboratorio anómalos antes de la aleatorización.
Plaquetas <25 000/ mm3 (<25 x 103/ μl).
Hemoglobina (Hgb) <8,0 g/dl (<5 mmol/l) o <7,0 g/dl (<4,3 mmol/l) si está relacionada con el lupus eritematoso sistémico del participante, como en el caso de la anemia hemolítica activa.
Recuento absoluto de neutrófilos (RAN) (<0,8 x 103/ μl).
Disfunción grave de un órgano o enfermedad potencialmente mortal en la selección.
Antecedentes de trasplante de un órgano sistémico, células madre hematopoyéticas o médula ósea.
Presencia de una enfermedad renal grave por lupus como se define por una proteinuria superior a 2 g/día o equivalente mediante un cociente proteína-creatinina en orina (CPCO) puntual.
Recepción de una vacuna viva/atenuada durante las 4 semanas anteriores a la primera administración del fármaco.
Cualquier enfermedad grave no controlada y coexistente que, en opinión del investigador, coloque al participante en una situación de riesgo debido a su participación en el estudio o que interfiera en la evaluación de los síntomas relacionados con el LES.
Antecedentes de hipersensibilidad a fármacos de clase similar, corticosteroides orales o a cualquiera de los componentes de los fármacos del ensayo (sacarosa, clorhidrato de L-histidina, L-histidina, polisorbato 20).
Antecendentes de no cumplimiento con los regímenes médicos o considerado poco confiable.
Uso de otros fármacos en investigación en un plazo de 5 semividas o 30 días antes del reclutamiento o hasta que el efecto farmacodinámico previsto vuelva a los valores basales, lo que sea más largo, o más largo si lo requiere la regulación local.
Enfermedades no relacionadas con el lupus como asma, gota o urticaria, que requieran tratamiento intermitente o crónico con corticosteroides sistémicos.
Antecedentes de malignidad de cualquier sistema orgánico salvo carcinoma basocelular cutáneo localizado o cáncer de cuello uterino in situ.
Mujeres embarazadas o en periodo de lactancia.
Mujeres con posibilidad de quedarse embarazadas, definidas como toda mujer fisiológicamente capaz de quedarse embarazada, salvo que esté utilizando métodos anticonceptivos altamente eficaces durante el tratamiento del estudio y durante los 6 meses después de dejar de tomar el fármaco en investigación.
En EE. UU. (y otros países si es localmente necesario): varones sexualmente activos, salvo que utilicen métodos anticonceptivos de barrera durante el coito durante el tratamiento del estudio.
Cualquier otra condición quirúrgica, médica, psiquiátrica o física que pudiera impedir la participación del paciente en este estudio.

E.5 End points

E.5.1

Primary end point(s)

Proportion of participants achieving SRI-4 at Week 60

Proporción de participantes que alcanzan una respuesta SRI-4 en la semana 60.

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 60

Semana 60

E.5.2

Secondary end point(s)

1. Proportion of participants with no moderate or severe British Isles Lupus Assessment Group flare up to Week 60

2. Proportion of participants maintaining between Week 36 and Week 60 a reduced corticosteroid dose of predniso(lo)ne ≤5 mg/day or ≤baseline dose, whichever is lower.

3. Proportion of participants achieving British Isles Lupus Assessment Group-based Composite Lupus Assessment at Week 60

4. Proportion of participants achieving Lupus Low Disease Activity State at Week 60

5. Time to first occurrence of SRI-4 from baseline to Week 60
6. Proportion of participants achieving SRI-4 at Week 60 while maintaining between Week 36 and Week 60 a reduced corticosteroiddose of predniso(lo)ne ≤5 mg/day or ≤baseline dose, whichever is lower
7. Proportion of participants achieving SRI-6 at Week 60
8. Proportion of participants maintaining between Week 24 and Week 60 a reduced corticosteroid dose of predniso(lo)ne ≤5 mg/day, or ≤baseline dose, whichever is lower

Proportion of participants with Adverse Events and
Serious Adverse Events
Clinical laboratory measurements
Vital signs
Incidence and titer of anti-ianalumab antibodies in serum (ADA assay) over time

Ianalumab concentration in serum during the treatment and follow-up (up to the end of study)

1. Proporción de participantes sin brotes moderados o graves según el British Isles Lupus Assessment Group hasta la semana 60.
2. Proporción de participantes que, entre la semana 36 y la semana 60, mantienen una dosis reducida de corticosteroides (predniso(lo)na) <=5 mg/día o <= dosis basal, aquella que sea inferior.
3. Proporción de participantes que alcanzan una respuesta BICLA en la semana 60.
4. Proporción de participantes que alcanzan un estado bajo de actividad del lupus en la semana 60.
5. Tiempo hasta la primera respuesta SRI-4 desde la basal hasta la semana 60.
6. Proporción de participantes que alcanzan una respuesta SRI-4 en la semana 60 y mantienen a su vez, entre la semana 36 y la semana 60, una dosis reducida de corticosteroides (predniso(lo)na) <=5 mg/día o <= dosis basal, aquella que sea inferior.
7. Proporción de participantes que alcanzan una respuesta SRI-6 en la semana 60.
8.0 Proporción de participantes que, entre la semana 24 y la semana 60, mantienen una dosis reducida de corticosteroides (predniso(lo)na) <=5 mg/día o <=dosis basal, aquella que sea inferior.
Proporción de participantes con acontecimientos adversos y acontecimientos adversos graves.
Mediciones de las pruebas analíticas.
Constantes vitales.
Incidencia y títulos de anticuerpos antiianalumab en suero (análisis de AAF) a lo largo del tiempo.
Concentración de ianalumab en suero durante el tratamiento y el seguimiento (hasta el fin de estudio).

E.5.2.1

Timepoint(s) of evaluation of this end point

1,3,4,6,7 - Week 60

2 - Between Week 36 and Week 60

5 - from baseline to Week 60

8 - Between Week 24 and Week 60

1,3,4,6,7 - Semana 60

2 - Entre la Semana 36 y la Semana 60

5 desde basal a Semana 60

8 -Entre la Semana 24 y la Semana 60

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

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Tolerability

Tolerabilidad

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

Brazil

Canada

China

Guatemala

Israel

Japan

South Africa

Thailand

United States

Poland

Bulgaria

Spain

Czechia

Hungary

Portugal

Slovakia

Turkey

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

última visita último paciente

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

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:

326

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

100

F.4.2.2

In the whole clinical trial

406

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)

Up to 2 year safety follow-up: Patients will be followed for at least 20 weeks (mandatory follow-up) or longer (conditional follow-up), until B cell recovery or up to 2 years after the end of treatment (EOT visit). In addition, the Investigator must provide follow-up medical care for all subjects, including subjects who are prematurely withdrawn from the study, or must refer them for appropriate ongoing care.

Seguimiento de seguridad de hasta 2 años: Se hará un seguimiento de los pacientes durante al menos 20 semanas (seguimiento obligatorio) o más largo (seguimiento condicional), hasta la recuperación de las células B o hasta 2 años después del fin del tratamiento (EoT). Adicionalmente, el investigador debe proporcionar seguimiento médico continuado a todos los sujetos, incluyendo aquellos que se retiraron prematuramente del ensayo o deben referirlos para que reciban atención médica continuada.

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

2022-12-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-12-07

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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