Clinical Trials Register

Summary
EudraCT Number:	2017-004558-41
Sponsor's Protocol Code Number:	PCYC-1146-IM
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:	2018-11-23
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

2017-004558-41

A.3

Full title of the trial

Phase 1/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Estudio en fase I/II de búsqueda de dosis y de la eficacia y seguridad de ibrutinib en pacientes pediátricos con enfermedad del injerto contra el huésped crónica (EICHc)

A.3.1

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

Phase 1/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Estudio en fase I/II de búsqueda de dosis y de la eficacia y seguridad de ibrutinib en pacientes pediátricos con enfermedad del injerto contra el huésped crónica (EICHc)

A.4.1

Sponsor's protocol code number

PCYC-1146-IM

A.5.4

Other Identifiers

Name:

IND

Number:

102,688

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	Pharmacyclics LLC
B.1.3.4	Country	United States
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	Pharmacyclics LLC
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Pharmacyclics LLC
B.5.2	Functional name of contact point	Clinical Operations
B.5.3	Address:
B.5.3.1	Street Address	995 East Arques Avenue
B.5.3.2	Town/ city	Sunnyvale
B.5.3.3	Post code	94085-4521
B.5.3.4	Country	United States
B.5.4	Telephone number	+1408215 3770
B.5.5	Fax number	+1408215 3684
B.5.6	E-mail	info@pcyc.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/3/16/1780
D.3 Description of the IMP
D.3.1	Product name	Ibrutinib, 70mg capsules
D.3.2	Product code	PCI-32765
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	Ibrutinib
D.3.9.1	CAS number	936563-96-1
D.3.9.2	Current sponsor code	PCI-32765
D.3.9.3	Other descriptive name	IBRUTINIB
D.3.9.4	EV Substance Code	SUB120863
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	70
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/3/16/1780
D.3 Description of the IMP
D.3.1	Product name	Ibrutinib, 140mg tablets
D.3.2	Product code	PCI-32765
D.3.4	Pharmaceutical form	Tablet
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	Ibrutinib
D.3.9.1	CAS number	936563-96-1
D.3.9.2	Current sponsor code	PCI-32765
D.3.9.3	Other descriptive name	IBRUTINIB
D.3.9.4	EV Substance Code	SUB120863
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	140
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/16/1780
D.3 Description of the IMP
D.3.1	Product name	Ibrutinib, 70mg/mL suspension
D.3.2	Product code	PCI-32765
D.3.4	Pharmaceutical form	Oral suspension
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	Ibrutinib
D.3.9.1	CAS number	936563-96-1
D.3.9.2	Current sponsor code	PCI-32765
D.3.9.3	Other descriptive name	IBRUTINIB
D.3.9.4	EV Substance Code	SUB120863
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	70
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

Chronic Graft Versus Host Disease (cGVHD)

Enfermedad del injerto contra el huésped crónica (EICHc)

E.1.1.1

Medical condition in easily understood language

After HCT, the donor’s immune system senses and attacks the recipient’s normal tissues, resulting in graft versus host disease (GVHD)

Después de la HCT, el sistema inmune del donante detecta y ataca los tejidos normales del receptor, lo que resulta en una enfermedad de injerto contra huésped (GVHD)

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	20.1
E.1.2	Level	PT
E.1.2	Classification code	10066261
E.1.2	Term	Chronic graft versus host disease
E.1.2	System Organ Class	10021428 - Immune system 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

Part A - Dose Finding Study
Primary Objective: To determine the RPED (based on PK and, if applicable, pharmacodynamic data) for use in pediatric subjects (age ≥1 to <12 years) with cGVHD as
defined by the 2014 NIH Consensus Development Project Criteria.
Part B - Pharmacokinetics, Safety, and Efficacy Study
Primary Objective: To assess the PK and safety and efficacy of ibrutinib in pediatric subjects (age ≥ 1 to < 22 years) with cGVHD.

Parte A: búsqueda de dosis
Objetivo principal:
Determinar la dosis pediátrica recomendada equivalente (RPED; a partir de datos farmacocinéticos [FC] y farmacodinámicos, si corresponde) para su uso en pacientes pediátricos (edad ≥1 a <12 años) con EICHc según lo definido en los criterios del proyecto de desarrollo de consenso de los Institutos Nacionales de Salud de los EE. UU. de 2014
Parte B: farmacocinética, seguridad y eficacia
Objetivo principal:
Evaluar la FC y la seguridad de ibrutinib en pacientes pediátricos (edad ≥1 a <22 años) con EICHc.

E.2.2

Secondary objectives of the trial

Part A - Dose Finding Study
• To determine the safety of ibrutinib in pediatric subjects with cGVHD
• To assess pharmacodynamics (BTK occupancy) of ibrutinib in pediatric subjects with cGVHD
• For those subjects continuing therapy after dose escalation (Part A Continuation Cohort), secondary endpoints will be the same as those outlined under Part B below and will include safety and efficacy
Part B - Pharmacokinetics, Safety, and Efficacy Study
• To evaluate the efficacy of ibrutinib treatment at 24 weeks in pediatric subjects with cGVHD using the 2014 NIH Consensus Development Project Criteria
• To evaluate the duration of response to ibrutinib treatment in pediatric subjects with cGVHD
• To evaluate the overall survival rate in pediatric subjects with cGVHD treated with ibrutinib
• To evaluate safety by assessing the potential impact of ibrutinib on late effects (including effects on growth and development and immune reconstitution) in pediatric subjects with
GVHD

Parte A: búsqueda de dosis:Determinar seguridad de ibrutinib en ptes pediátricos con EICHc. Evaluar farmacodinámic(ocupación de la tirosina cinasa de Bruton [BTK]) de ibrutinib en pacientes pediátricos con EICHc. Cohorte continuación de parte A, criterios d valoración secundarios de pacientes q sigan con tratamiento tras aumento gradual dedosis (cohorte de continuación de la parte A) serán los mismos que los descritos en la parte B que se describe:Parte B:farmacocinética seguridad y eficacia.Evaluar eficacia de tratamiento con ibrutinib a las 24 semanas en ptes pediátricos con EICHc.Evaluar duración de respuesta del tratamiento con ibrutinib en pacientes pediátricos con EICHc.Evaluar tasa supervivencia general en pacientes pediátricos con EICHc tratados con ibrutinib.
Evaluar la seguridad mediante el análisis de la posible influencia de ibrutinib en los efectos tardíos (incluyendo los efectos en el crecimiento y el desarrollo y en la reconstitución inmunitaria) en pacientes con EICHc

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Part A: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic
therapy
2. Part B: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy, or subjects with new onset moderate or severe cGVHD and in need of systemic immunosuppression.
a. Subjects with new onset moderate or severe cGVHD must not have received previous systemic therapy for cGVHD with the exception of corticosteroids received within
72 hours prior to signing the informed consent form.
b. Subjects with newly diagnosed cGVHD may be receiving other immunosuppressants for the prophylaxis or treatment of acute GVHD, but if the subject is receiving prednisone for prophylaxis or treatment of acute GVHD it must be at or below 0.5 mg/kg/d at the time of enrollment.
3. History of allogeneic stem cell transplantation
4. Age
• Part A: ≥1 to <12 years of age at the time of enrollment
• Part B: ≥1 to <22 years of age at the time of enrollment
5. Written informed consent or parental or guardian permission and assent of children capable of understanding the nature of the study, per country specific or site-specific standards.
6. Ability of subject or, if a minor, parent/guardian to understand the purpose and risks of the study and to provide a signed and dated parental permission and authorization to use protected health information (in accordance with national and local subject privacy
regulations); willingness of child to provide an assent, if developmentally able to do so.

Parte A: pacientes con EICHc de moderada a grave tras el fracaso de 1 o más líneas de tratamiento sistémico.
2. Parte B: pacientes con EICHc de moderada a grave tras el fracaso de 1 o más líneas de tratamiento sistémico o pacientes con EICHc de moderada a grave de reciente aparición con necesidad de inmunodepresión sistémica.
a.Los pacientes con EICHc de moderada a grave de reciente aparición no deben haber recibido tratamiento sistémico anteriormente para tratar la EICHc, con la excepción de corticosteroides en el plazo de las 72 horas anteriores a la firma del formulario de consentimiento informado
b.Los pacientes con EICHc recién diagnosticada pueden estar recibiendo otros inmunodepresores para la profilaxis o el tratamiento de la EICH aguda, pero si el paciente está recibiendo prednisona para la profilaxis o el tratamiento de la EICH aguda, la dosis debe ser igual o inferior a 0,5 mg/kg/d en el momento de la inclusión
3.Antecedentes de alotrasplante de células madre
4.Edad
Parte A: edad de ≥1 a <12 años en el momento de la inclusión
Parte B: edad de ≥1 a <22 años en el momento de la inclusión
5.Consentimiento informado por escrito o permiso de los padres o el tutor legal y asentimiento de los niños con capacidad para comprender la naturaleza del estudio según las normas de cada país o centro
6.Capacidad del paciente, o si es un menor, de los padres o el tutor para comprender la finalidad y los riesgos del estudio y facilitar el permiso de los padres firmado y fechado y la autorización para usar información médica protegida (de acuerdo con la normativa nacional y local sobre la privacidad de los pacientes); voluntad del niño para otorgar el asentimiento si puede hacerlo desde el punto de vista madurativo.

E.4

Principal exclusion criteria

1. Presence of single organ genito-urinary involvement as the only manifestation of cGVHD.
Concurrent Conditions
2. Received an investigational agent within 28 days before enrollment.
3. Received donor lymphocyte infusion (DLI) within 56 days before enrollment.
4. Progressive underlying malignant disease or active post-transplant lymphoproliferative disease.
5. Ongoing anticoagulation treatment with warfarin or equivalent vitamin K antagonist.
6. History of other malignancy (not including the underlying malignancy that was the indication for transplant), with the following exceptions:
• Malignancy treated with curative intent and with no evidence of active disease present for more than 3 years prior to enrollment and felt to be at low risk for recurrence by treating physician
• Adequately treated non-melanomatous skin cancer or lentigo maligna melanoma without current evidence of disease
• Adequately treated cervical carcinoma in situ without current evidence of disease
7. History of major surgery within 28 days before enrollment or lack of full recovery from surgery.
8. Any life-threatening illness, medical condition, or organ system dysfunction that, in the investigator’s opinion, could compromise the subject’s safety or put the study outcomes at undue risk.
9. Female subject who is pregnant, breastfeeding, or planning to become pregnant while enrolled in this study or within 3 months of the last dose of study drug. Male subject who plans to father a child while enrolled in this study or within 3 months after the last dose of study drug.
10. Unwilling or unable to participate in all required study evaluations and procedures.

1. Afectación genitourinaria como única manifestación de la EICHc.
Enfermedades concurrentes
2. Haber recibido algún fármaco experimental en el plazo de los 28 días anteriores a la inclusión
3. Haber recibido una infusión de linfocitos del donante (DLI) en el plazo de los 56 días anteriores a la inclusión.
4. Enfermedad maligna subyacente en progresión o enfermedad linfoproliferativa activa posterior al trasplante
5.Tratamiento anticoagulante en curso con warfarina o antagonista de la vitamina K equivalente.
6.Antecedentes de otras neoplasias malignas (excepto la neoplasia maligna subyacente que fue la indicación para el trasplante), con las excepciones siguientes:
Neoplasia maligna tratada con intención curativa sin indicios de enfermedad activa presente durante más de 3 años antes de la inclusión y con consideración de riesgo bajo de recurrencia según el médico responsable.
Cáncer de piel distinto del melanoma o melanoma sobre lentigo maligno tratados adecuadamente sin indicios actuales de enfermedad
Carcinoma in situ de cuello uterino tratado adecuadamente sin indicios actuales de enfermedad
Antecedentes de cirugía mayor en el plazo de los 28 días anteriores a la inclusión o falta de recuperación completa de la cirugía
Enfermedad potencialmente mortal o disfunción orgánica que a juicio del investigador puede poner en peligro la seguridad del paciente o poner los resultados del estudio bajo un riesgo excesivo.
Mujeres embarazadas, en periodo de lactancia o con intención de quedarse embarazadas durante la inclusión en el estudio o en el plazo de los 3 meses posteriores a la última dosis del fármaco del estudio. Participantes del sexo masculino que tienen previsto concebir un hijo durante la inclusión en el estudio o en el plazo de los 3 meses posteriores a la última dosis del fármaco del estudio
Reticencia o incapacidad para participar en la totalidad de evaluaciones y procedimientos del estudio requeridos

E.5 End points

E.5.1

Primary end point(s)

Part A: The primary endpoint is the PK (AUC) to determine the RPED of ibrutinib for use in pediatric subjects (age ≥ 1 to < 12 years) with cGVHD.
Part B: The primary endpoint is the PK (AUC) and safety (treatment-emergent AEs and laboratory abnormalities) of ibrutinib in pediatric subjects (age ≥ 1 to < 22 years) with cGVHD.

Parte A: FC (área bajo la curva de la concentración plasmática respecto al tiempo [ABC]) para determinar la RPED de ibrutinib para su uso en pacientes pediátricos (edad ≥1 a <12 años) con EICHc.
Parte B: FC (ABC) y seguridad (AA surgidos durante el tratamiento y anomalías en el análisis) de ibrutinib en pacientes pediátricos (edad ≥1 a <22 años) con EICHc.

E.5.1.1

Timepoint(s) of evaluation of this end point

The primary analysis for PK, safety and efficacy will occur after all subjects have had the opportunity to complete 24 weeks of treatment.

El análisis principal de la FC, seguridad y eficacia tendrá lugar cuando todos los pacientes hayan tenido la oportunidad de terminar 24 semanas de tratamiento

E.5.2

Secondary end point(s)

Part A: The secondary endpoints are safety, including treatment-emergent AEs, laboratory abnormalities, and other safety endpoints; pharmacodynamics (BTK occupancy) and for those subjects continuing therapy after dose escalation (Part A Continuation Cohort), secondary
endpoints will be the same as outlined under Part B below.
Part B: The secondary endpoints include cGVHD response rate (i.e. the proportion of responders [CR or PR]) at 24 weeks; duration of response; overall survival rate; growth and development in pediatric subjects (i.e., height, weight, Tanner Stage, and head circumference in subjects < 3years), and immune reconstitution data

Criterios de valoración secundarios:
Parte A:
Seguridad, incluyendo AA surgidos durante el tratamiento, anomalías en el análisis y otros criterios de valoración de la seguridad. Farmacodinámica (ocupación de la BTK). Los criterios de valoración secundarios de los pacientes que sigan con el tratamiento tras el aumento gradual de la dosis (cohorte de continuación de la parte A) serán los mismos que los descritos en la parte B que se describe a continuación.
Parte B:
Tasa de respuesta a las 24 semanas.
Duración de la respuesta.
Tasa de supervivencia general.
Crecimiento y desarrollo en pacientes pediátricos (Altura, peso, tanner stage, y circunferencia de la cabeza en sujetos <3 años y datos de reconstitución inmune
Reconstitución inmunitaria.

E.5.2.1

Timepoint(s) of evaluation of this end point

After all subjects have had the opportunity to complete 24 weeks of treatment.

Después de que todos los sujetos hayan tenido la oportunidad de completar 24 semanas de tratamiento

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

Yes

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)

Yes

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

new indication

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.8.3

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

Australia

Austria

Canada

France

Germany

Italy

Netherlands

Russian Federation

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

Último Paciente Última visita

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

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:

F.1.3

Elderly (>=65 years)

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)

In addition to regular assessments of safety and efficacy, subjects in Part A and B will continue to be followed every 12 months until 60 months post-enrollment, to assess for potential late effects of ibrutinib, including effects on growth and development as well as immune reconstitution.

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

2019-03-06

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-02-13

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

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