Clinical Trials Register

Summary
EudraCT Number:	2018-003296-35
Sponsor's Protocol Code Number:	CINC424G12201
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:	2019-10-21
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

2018-003296-35

A.3

Full title of the trial

A Phase II open-label, single-arm, multi-center study of ruxolitinib added to corticosteroids in pediatric subjects with moderate and severe chronic graft vs. host disease after allogeneic stem cell transplantation

Estudio de fase II, multicéntrico, abierto, de un solo brazo , de ruxolitinib añadido a corticosteroides en sujetos pediátricos con enfermedad de injerto contra huésped crónica moderada y grave tras trasplante alogénico de
células madre

A.3.1

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

Activity, safety and pharmacokinetics in pediatric subjects with moderate and severe chronic graft vs. host disease after allogeneic stem cell transplant

Estudio de la actividad, la seguridad y la farmacocinética en pacientes pediátricos con enfermedad de injerto contra huésped crónica moderada y grave después tras trasplante alogénico de células madre.

A.4.1

Sponsor's protocol code number

CINC424G12201

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/061/2018

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 Vía 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	+34900353036
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	Yes
D.2.1.1.1	Trade name	Jakavi
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm Limited
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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	ruxolitinib
D.3.2	Product code	INC424
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	ruxolitinib
D.3.9.1	CAS number	1092939-17-7
D.3.9.2	Current sponsor code	INC424
D.3.9.3	Other descriptive name	RUXOLITINIB PHOSPHATE
D.3.9.4	EV Substance Code	SUB32897
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.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

enfermedad de injerto contra huésped crónica

E.1.1.1

Medical condition in easily understood language

graft versus host disease

enfermedad de injerto contra huésped

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

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	10064677
E.1.2	Term	Graft versus host disease in intestine
E.1.2	System Organ Class	100000004870

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	10075161
E.1.2	Term	Graft versus host disease in GI tract
E.1.2	System Organ Class	100000004870

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 evaluate the activity of ruxolitinib added to standard dose corticosteroids +/- CNI in pediatric subjects with moderate or severe treatment naivecGvHD or SR-cGvHD measured by overall response rate (ORR) at Cycle 7 Day 1 based on all subjects in the study

Evaluar la actividad del ruxolitinib añadido a dosis habituales de corticosteroides, +/- un inhibidor de la
calcineurina (ICN), en sujetos pediátricos con EICHc sin tratamiento previo o EICHc-RC moderada o grave, según la medición de la tasa de respuesta global (TRG) el día 1 del ciclo 7.

E.2.2

Secondary objectives of the trial

- To assess pharmacokinetics (PK) of
ruxolitinib in treatment-naïve cGvHD
and SR-cGvHD pediatric subjects
- To evaluate the safety of ruxolitinib  
- To assess duration of response (DOR)
- To estimate ORR at end of Cycle 3
- To assess best overall response (BOR)
- To estimate the failure free survival (FFS)
- To assess cumulative incidence of malignancy relapse/recurrence (MR)
- To assess non-relapse mortality (NRM)
- To assess overall survival (OS)
- To assess a reduction of at least ≥ 50% in daily corticosteroid use at Cycle 7 Day 1
- To assess a reduction to a low dose corticosteroid dose at Cycle 7 Day 1
- To assess graft failure

- Evaluar la farmacocinética (FC) del ruxolitinib en pacientes pediátricos con EICHc sin tratamiento previo o EICHc-RC.
- Evaluar la seguridad del ruxolitinib.
- Evaluar la duración de la respuesta (DdR).
- Calcular la TRG al final del ciclo 3.
- Evaluar la mejor respuesta global (MRG).
- Calcular la supervivencia sin fracaso (SSF).
- Evaluar la incidencia acumulada de recidivas/recurrencias de la neoplasia maligna (RM).
- Evaluar la mortalidad sin recidiva (MSR).
- Evaluar la supervivencia global (SG).
- Evaluar la reducción de >/= 50 % del uso diario de corticosteroides en el día 1 del ciclo 7.
- Evaluar la reducción a una dosis baja de corticosteroides en el día 1 del ciclo 7.
- Evaluar el fracaso del injerto.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Male or female subjects age ≥28 days and <18
years at the time of informed consent.
• Subjects who have undergone alloSCT from any
donor source (matched unrelated donor, sibling, haploidentical) using bone marrow, peripheral blood stem cells, or cord blood. Recipients of myeloablative or reduced intensity conditioning are eligible.
• Subjects with diagnosed moderate to severe
cGvHD according to NIH 2014 Consensus Criteria
(Section 16.2) prior to Cycle 1 Day 1. Other possible
diagnoses for clinical symptoms supporting cGvHD
diagnoses must be excluded (e.g., infection, drug side effects, malignancy). Subjects must be either:
o Treatment-naive cGvHD subjects that have not
received any prior systemic treatment for cGvHD
except for a maximum 72h of prior systemic
corticosteroid therapy of methylprednisolone or
equivalent after the onset of chronic GvHD. Subjects
are allowed to have received prior systemic treatment for cGvHD prophylaxis (as long as the prophylaxis was started prior to the diagnosis of cGvHD).
OR
o Steroid-refractory moderate to severe cGvHD as
per institutional criteria, and still receiving systemic
corticosteroids for the treatment of cGvHD for a
duration of <18 months prior to Cycle 1 Day 1. In case the corticosteroids were interrupted due to response,
the duration of < 18 months applies to the last period of corticosteroid use.

Additional inclusion criteria as per fulll protocol may apply.

- Pacientes de ambos sexos de ≥ 28 días y < 18 años de edad en el momento del consentimiento informado
- Sujetos que se hayan sometido a un trasplante alogénico de cualquier tipo de donante (donante no emparentado compatible, hermano/a, donante haploidéntico) con médula ósea, células madre de sangre periférica o bien sangre de cordón umbilical. Tanto los pacientes con acondicionamiento mieloablativo como los sometidos a un acondicionamiento de intensidad reducida son elegibles.
- Pacientes con EICHc moderada o grave diagnosticada según los criterios de consenso del NIH (Apartado 16.2) antes del día 1 del ciclo 1. Los pacientes deben:
* Ser pacientes de EICHc que no hayan recibido ningún tratamiento sistémico previo para la EICHc salvo un tratamiento de una duración máxima de 72 horas con corticosteroides sistémicos (metilprednisolona o equivalente) tras la aparición de la EICH crónica. Se les permite haber recibido tratamiento sistémico previo
profiláctico contra la EICHc (siempre que la profilaxis se haya iniciado antes del diagnóstico de EICHc).
BIEN
* Padecer EICHc moderada o grave resistente a los corticosteroides (RC) según los criterios institucionales y seguir recibiendo corticosteroides sistémicos para el tratamiento de la EICHc durante < 18 meses antes del día 1 del ciclo 1.

Otros criterios de inclusión según protocolo pueden aplicar

E.4

Principal exclusion criteria

• SR-cGvHD subjects with a prior cGvHD
treatment with a JAK1- or a JAK2- or a JAK1/2-inhibitor, except when the subject achieved
complete or partial response and has been off JAK
inhibitor treatment for at least 4 weeks prior to Cycle Day 1 or up to 5 times the half-life of the prior JAK inhibitor, whichever is longer.
* Subjects who initiated systemic calcineurin
inhibitors (CNI; cyclosporine or tacrolimus) within 3
weeks prior to start of ruxolitinib on Cycle 1 Day 1.
Note: systemic CNI are allowed when initiated > 3
weeks from start of ruxolitinib.
• Failed prior alloSCT within the past 6 months
• Significant respiratory disease including subjects
who are on mechanical ventilation or who have a
resting oxygen saturation < 90% by pulse-oximetry on room-air.
• Impairment of gastrointestinal (GI) function
(unrelated to GvHD) or GI disease (unrelated to
GvHD) that may significantly alter the absorption of
oral ruxolitinib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection),
• Cholestatic disorders, or unresolved sinusoidal
obstructive syndrome/veno-occlusive disease of the
liver (defined as persistent bilirubin abnormalities not attributable to cGvHD and ongoing organ dysfunction)
• Presence of clinically active uncontrolled
infection including significant bacterial, fungal, viral or parasitic infection requiring treatment.
• Known human immunodeficiency virus (HIV)
infection.
• Evidence of uncontrolled hepatitis B virus (HBV)
or hepatitis C virus (HCV) based on assessment done
by Investigator or delegate.
• Known allergies, hypersensitivity, or intolerance
to any of the study medications, excipients, or similar compounds.
• History of bone disorders such as osteogenesis
imperfecta, rickets, renal osteodystrophy,
osteomyelitis, osteopenia, fibrous dysplasia,
osteomalacia etc. prior to the underlying diagnosis
which resulted in the alloSCT.
• History of endocrine or kidney related growth
retardation prior to the underlying diagnosis which
resulted in the alloSCT.
• Evidence of clinically active tuberculosis (clinical
diagnosis per local practice)
• Any corticosteroid therapy for indications other
than cGvHD at doses > 1 mg/kg/day methylprednisolone (or equivalent prednisone dose 1.25 mg/kg/day) within 7 days of the screening visit.
• History of progressive multifocal leukoencephalopathy (PML).
• Presence of severely impaired renal function

Other protocol-defined inclusion/exclusion criteria may apply.

- No se permite la inclusión de pacientes con EICHc-RC tratados previamente con un inhibidor de JAK (JAK1, JAK2 o JAK1/2), salvo si el sujeto alcanzara una respuesta completa o parcial y no hubiera recibido tratamiento con un inhibidor de JAK durante al menos 4 semanas antes del día 1 del ciclo 1, o hasta 5 veces la semivida del inhibidor de JAK previo, el período que sea mayor.
- Sujetos que iniciaran tratamiento con inhibidores sistémicos de la calcineurina (ICN; ciclosporina o tacrólimus) en las 3 semanas previas al inicio del tratamiento con ruxolitinib el día 1 del ciclo 1. Nota: Se permiten los ICN sistémicos si han empezado a administrarse > 3 semanas antes del inicio del tratamiento con ruxolitinib.
-Fracaso de un trasplante alogénico previo en los últimos 6 meses
-Enfermedad respiratoria relevante, incluidos pacientes con ventilación mecánica o que tengan una saturación de oxígeno en reposo < 90 % según la pulsioximetría con aire ambiente.
- Pacientes con disfunción gastrointestinal (GI) (no relacionada con la EICH) o enfermedades digestivas (no relacionadas con la EICH) que podrían alterar la absorción del ruxolitinib tomado por vía oral de forma notable (p. ej., enfermedades ulcerosas, náuseas no controlables, vómitos, diarrea, síndrome de malabsorción o resección del intestino delgado).
-Trastornos colestásicos o síndrome obstructivo sinusoidal/enfermedad venooclusiva del hígado no resueltos (definidos como anomalías persistentes de la bilirrubina no atribuibles a la EICHc y disfunción orgánica en curso).
- Presencia de una infección clínicamente activa no controlada, incluidas infecciones bacterianas, fúngicas, víricas o parasitarias significativas que requieran tratamiento.
-Signos de infección por el virus de la hepatitis B (VHB) o el virus de la hepatitis C (VHC) no controlados según la evaluación realizada por el investigador o su representante.
-Alergias, hipersensibilidad o intolerancia conocidas a cualquiera de los medicamentos del estudio, los excipientes o compuestos similares
-Antecedentes de trastornos óseos, como osteogénesis imperfecta, raquitismo, osteodistrofia renal, osteomielitis, osteopenia, displasia fibrosa, osteomalacia, etc. antes del diagnóstico subyacente que dio lugar al trasplante alogénico
-Antecedentes de retraso del crecimiento por causas endocrinas o renales antes del diagnóstico subyacente que dio lugar al ATCM.
-Pruebas de tuberculosis clínicamente activa (diagnóstico clínico según la práctica local).
-Cualquier tratamiento con corticosteroides para indicaciones diferentes de la EICHc en dosis > 1 mg/kg/día de metilprednisolona (o dosis equivalentes a 1,25 mg/kg/día de prednisona) en los 7 días anteriores a la consulta de selección.
-Antecedentes de leucoencefalopatía multifocal progresiva (LMP).
-Presencia de deterioro grave de la función renal (confirmada en las 72 h anteriores al inicio del tratamiento con ruxolitinib)
Otros criterios de exclusión según protocolo pueden aplicar

E.5 End points

E.5.1

Primary end point(s)

Overall Response Rate ORR defined as the
proportion of subjects demonstrating a complete
response (CR) or partial response (PR) without the
requirement of additional systemic therapies for an
earlier progression, mixed response or non-response

La Tasa de respuesta global se define como la proporción de sujetos que demuestran una respuesta completa (RC) o parcial (RP) sin el requisito de recibir un tratamiento sistémico adicional para la progresión temprana, la respuesta mixta o la ausencia de respuesta.

E.5.1.1

Timepoint(s) of evaluation of this end point

Cycle 7 Day 1 (Day 168)

Día 1 del ciclo 7 (Día168)

E.5.2

Secondary end point(s)

1. Ruxolitinib concentrations by timepoint
2. Duration of response (DOR) assessed for
responders only. DOR is defined as the time from first response until cGvHD progression, death, or the date of addition of systemic therapies for cGvHD
3. Proportion of subjects who achieve OR (CR+PR)
at Cycle 4 Day 1
4. Proportion of subjects who achieved OR (CR+PR) at any time point
5. Composite time to event endpoint incorporating the following FFS events: i) relapse or recurrence of
underlying disease or death due to underlying disease, ii) non-relapse mortality, or iii) addition or initiation of another systemic therapy for cGvHD
6. Malignancy relapse/recurrence (MR) is defined
as the time from date of treatment assignment to
hematologic malignancy relapse/recurrence.
Calculated for subjects with underlying hematologic malignant disease
7. Non-relapse mortality (NRM), defined as the time
from date of treatment assignment to date of
death not preceded by underlying disease
relapse/recurrence.
8. Overall survival, defined as the time from the
date of treatment assignment to the date of death due to any cause
9. Proportion of subjects with ≥ 50% reduction from
baseline in daily corticosteroid dose at Cycle 7 Day 1
10. Proportion of subjects with reduction from
baseline in daily corticosteroid dose to ≤ 0.2
mg/kg/day methylprednisolone (or equivalent
dose of ≤ 0.25 mg/kg/day prednisone or
prednisolone) at Cycle 7 Day 1

1. Concentraciones de ruxolitinib en cada medición
2. La duración de la respuesta (DR) se evalúa solo en los sujetos que responden al tratamiento. La DR se define como el tiempo transcurrido desde la primera respuesta hasta la progresión de la EICHc, la muerte o la fecha de la adición de tratamientos sistémicos para la EICHc.
3. Porcentaje de sujetos que alcanzan la RG (RC + RP) el día 1 del ciclo 4.
4. Porcentaje de sujetos que alcanzan la RG (RC + RP) en cualquier momento temporal
5. Criterio de valoración compuesto de tiempo transcurrido hasta el evento, con los siguientes eventos de supervivencia libre de fallo: i) recaída o recurrencia de la enfermedad subyacente o muerte debida a enfermedad subyacente, ii) mortalidad sin recidiva, o iii) adición o inicio de otro tratamiento sistémico para la EICHc.
6. La recaída de la neoplasia maligna se define como el tiempo transcurrido desde la asignación del tratamiento hasta la recaída de la neoplasia maligna hematológica. Se calcula en los sujetos con neoplasias malignas hematológicas subyacentes
7. Mortalidad sin recaída , definida como el tiempo transcurrido desde la fecha de la asignación del tratamiento hasta la fecha de muerte no precedida por recaída de la enfermedad subyacente.
8. Supervivencia global, definida como el tiempo transcurrido desde la fecha de la asignación del tratamiento hasta la fecha de muerte por cualquier causa.
9. Porcentaje de sujetos con una reducción >/= 50% respecto al inicio en la dosis de corticosteroides diaria en el día 1 del ciclo 7
10. Porcentaje de sujetos con una reducción respecto al inicio en la dosis de corticosteroides diaria a </= 0,2 mg/kg/día de metilprednisolona (o una dosis equivalente de </= 0,25 mg/kg/día de prednisona o prednisolona) el día 1 del ciclo 7

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Cycle 7 Day 1 (from baseline to Day 168)
2. From baseline up to endof study treatment, up to
36 months
3. Cycle 4 Day 1 (Day 84)
4. Until Cycle 7 Day 1 (Day 168) or the start of
additional systemic therapy for cGvHD
5. From baseline up to 35 days after end of study
treatment, up to 37 months
6. From baseline up to 35 days after end of study
treatment, up to 37 months
7. From baseline up to 35 days after end of study
treatment, up to 37 months
8. From baseline up to 35 days after end of study
treatment, up to 37 months
9. Cycle 7 Day 1 (Day 168)
10. Cycle 7 Day 1 (Day 168)
11. From baseline up to 35 days after end of study
treatment, up to 37 months

1. Ciclo 7 día 1 (desde punto basal al día 168)
2. Desde el punto basal hasta fin de tratamiento, hasta 36 meses
3. Cciclo 4 día 1 (dia 84)
4. Hasta ciclo7 día 1 (día 168) o inicio de terapias sistémicas adicionales para EICH crónico.
5. Desde el punto basal hasta 35 días después de fin de tratamiento, hasta 37 meses.
6. Desde el punto basal hasta 35 días después de fin de tratamiento, hasta 37 meses.
7. Desde el punto basal hasta 35 días después de fin de tratamiento, hasta 37 meses.
8. Desde el punto basal hasta 35 días después de fin de tratamiento, hasta 37 meses.
9. Ciclo 7 día 1 (día 168)
10. Ciclo 7 día 1 (día 168)
11. Desde el punto basal hasta 35 días después de fin de tratamiento, hasta 37 meses.

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)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.3

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

Argentina

Brazil

Canada

Chile

Czech Republic

Germany

Greece

India

Italy

Japan

Korea, Republic of

Lithuania

Russian Federation

Saudi Arabia

Slovakia

Slovenia

Spain

Switzerland

Taiwan

Thailand

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

LPLV

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

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)

Yes

F.1.1.3.1

Number of subjects for this age range:

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)

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)

subjects who are still receiving ruxolitinib at their end of study, and deriving clinical benefit from ruxolitinib as assessed by the Investigator, will also be given the possibility to continue ruxolitinib outside the study as part of the Novartis post-trial access program, as permitted by and in accordance to local laws and regulations

Los pacientes que continúen teniendo beneficio clínico durante los 3 años de tratamiento, tendrán la posibilidad de contunuar con ruxolitinib fuera del ensayo clínico, según lo permita las leyes o regulaciones locales.

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

2019-10-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-09-05

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

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