Clinical Trials Register

Summary
EudraCT Number:	2018-002301-61
Sponsor's Protocol Code Number:	2215-CL-0603
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:	2019-09-04
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

2018-002301-61

A.3

Full title of the trial

A Phase 1/2, Multicenter, Open-Label, Single Arm, Dose Escalation and Expansion Study of Gilteritinib (ASP2215) Combined with Chemotherapy in Children, Adolescents and Young Adults with FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

Estudio de fase 1/2, multicéntrico, abierto, de brazo único, con escalada y expansión de la dosis de gilteritinib (ASP2215) combinado con quimioterapia en niños, adolescentes y adultos jóvenes con leucemia mieloide aguda positiva a duplicación interna en tándem (ITD) del gen FLT3 (FMS-like Tyrosine Kinase 3) en recidiva o refractaria

A.3.1

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

A study of Gilteritinib combined with chemotherapy to treat Children, Adolescents and Young Adults with Relapsed or Refractory Acute Myeloid Leukemia (AML) with a FLT3 gene mutation.

Estudio de Gilteritinib combinado con quimioterapia en niños, adolescentes y adultos jóvenes con leucemia mieloide aguda positiva en recidiva o refractaria con mutación en el gen FLT3

A.4.1

Sponsor's protocol code number

2215-CL-0603

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/194/2019

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Astellas Pharma Global Development, Inc. (APGD)
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	Astellas Pharma Global Development, Inc. (APGD)
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	Astellas Pharma Europe B.V.
B.5.2	Functional name of contact point	Clinical Trial Unit Reg. Affairs
B.5.3	Address:
B.5.3.1	Street Address	Sylviusweg 62
B.5.3.2	Town/ city	Leiden
B.5.3.3	Post code	2333 BE
B.5.3.4	Country	Netherlands
B.5.4	Telephone number	+31 71 545 5050
B.5.5	Fax number	+31 71 545 5840
B.5.6	E-mail	CTU@astellas.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/3/17/1961
D.3 Description of the IMP
D.3.1	Product name	Gilteritinib
D.3.2	Product code	ASP12215
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	Yes
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	GILTERITINIB
D.3.9.1	CAS number	1254053-43-4
D.3.9.2	Current sponsor code	ASP2215
D.3.9.4	EV Substance Code	SUB177203
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.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/3/17/1961
D.3 Description of the IMP
D.3.1	Product name	Gilteritinib
D.3.2	Product code	ASP2215
D.3.4	Pharmaceutical form	Film-coated 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	GILTERITINIB
D.3.9.1	CAS number	1254053-43-4
D.3.9.2	Current sponsor code	ASP2215
D.3.9.4	EV Substance Code	SUB177203
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	40
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

FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or
Refractory Acute Myeloid Leukemia (AML)

Leucemia mieloide aguda positiva a duplicación interna en tándem (ITD) del gen FLT3 (FMS-like Tyrosine Kinase 3) en recidiva o refractaria

E.1.1.1

Medical condition in easily understood language

AML is cancer of myeloid line of blood cells characterized by rapid growth of abnormal white blood cells that accumulate in bone marrow and interfere with production of normal blood cells.

LMA es cancer en la linea mieloide de células sanguíneas caracterizado por rápido crecimiento de glóbulos blancos que se acumulan en la médula ósea interfiriendo la producción de celulas sanguineas

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	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10000886
E.1.2	Term	Acute myeloid leukemia
E.1.2	System Organ Class	100000004864

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

Phase 1 (Dose Escalation Phase):
To determine the maximum tolerated dose (MTD) and/or optimally safe and biologically active recommended phase 2 dose (RP2D) of gilteritinib given in sequential combination with fludarabine, cytarabine and granulocyte colony-stimulating factor (FLAG) in children, adolescents and young adults with relapsed/refractory (R/R) FMS like tyrosine kinase 3 (FLT3)/internal tandem duplication (ITD) acute myeloid leukemia (AML).

Phase 2 (Dose Expansion Phase):
To determine complete remission (CR) rates and composite complete remission (CRc) rates after 2 cycles of gilteritinib in sequential combination with FLAG in children, adolescents and young adults in R/R FLT3/ITD AML.

Fase 1 (Fase de escalada de la dosis):
Determinar la dosis máxima tolerada (maximum tolerated dose, MTD) y/o la dosis óptimamente segura y biológicamente activa recomendada para la fase 2 (recommended phase 2 dose, RP2D) de gilteritinib administrado en combinación secuencial con fludarabina, citarabina y factor estimulante de colonias de granulocitos (FLAG) en niños, adolescentes y adultos jóvenes con leucemia mieloide aguda (acute myeloid leukemia, AML) positiva a duplicación interna en tándem (internal tandem duplication, ITD) del gen FLT3 (FMS-like Tyrosine Kinase 3) en recidiva o refractaria (relapsed/refractory, R/R).

Fase 2 (Fase de expansión de la dosis):
Determinar las tasas de remisión completa (complete remission, CR) y de remisión completa compuesta (composite complete remission, CRc) tras 2 ciclos de gilteritinib en combinación secuencial con FLAG en niños, adolescentes y adultos jóvenes con leucemia mieloide aguda FLT3/ITD en R/R.

E.2.2

Secondary objectives of the trial

The secondary objectives are:
● To assess the safety, tolerability and toxicities of gilteritinib when given in sequential combination with FLAG in children, adolescents, and young adults with R/R FLT3/ITD AML.
● To evaluate FLT3 inhibition due to gilteritinib treatment
● To characterize gilteritinib (and active metabolites if warranted) pharmacokinetics.
● To perform serial measurements of minimal residual disease (MRD) and examine the relationship with study endpoints.
● To obtain preliminary estimates of 1-year event-free survival (EFS) and overall survival (OS) rate.
● To assess the acceptability and palatability of the formulation.

Los objetivos secundarios son:
• Evaluar la seguridad, tolerabilidad y toxicidades de gilteritinib en combinación secuencial con FLAG en niños, adolescentes y adultos jóvenes con leucemia mieloide aguda FLT3/ITD en R/R.
• Evaluar la inhibición de FLT3 como consecuencia del tratamiento con gilteritinib.
• Determinar la farmacocinética de gilteritinib (y de sus metabolitos activos si procede).
• Practicar mediciones seriadas de la enfermedad residual mínima (minimal residual disease, MRD) y examinar su relación con los criterios de valoración del estudio.
• Obtener unas estimaciones preliminares de las tasas de supervivencia sin eventos (event-free survival, EFS) y de supervivencia global (overall survival, OS) transcurrido 1 año.
• Evaluar la aceptabilidad y palatabilidad de la formulación.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved informed consent and privacy language as required per national regulations (e.g., Health Insurance
Portability and Accountability Act Authorization for US sites) must be obtained from the subject or subject’s parent or legal guardian and if required child assent prior to any study-related
procedures (including withdrawal of prohibited medication, if applicable).
2. Subject is positive for the FLT3/ITD mutation in bone marrow or blood as determined by the local institution.
3. Subject is aged ≥ 6 months and < 21 years of age at the time of signing informed consent and/or assent, as applicable.
4. Subject has a diagnosis of AML according to The French–American–British (FAB) classification with ≥ 5% blasts in the bone marrow, with or without extramedullary disease (except subjects
with active central nervous system [CNS] leukemia).
5. Subject has Karnofsky score ≥ 50 (if the subject is of ≥ 16 years of age) or Lansky score of ≥ 50 (if the subject is < 16 years of age).

1. Antes de cualquier procedimiento del estudio (lo que incluye, si procede, la retirada de la medicación prohibida), deberá obtenerse del sujeto o de su progenitor o tutor legal el consentimiento informado aprobado por el Comité de Ética y el texto acerca de la privacidad que puedan exigir las normativas locales (por ejemplo, la Health Insurance Portability and Accountability Act Authorization en el caso de los centros de Estados Unidos) y, si procede, el asentimiento del menor.
2. El sujeto muestra positividad de mutación FLT3/ITD en médula ósea o en sangre en el estudio practicado por el centro local.
3. El sujeto tiene >= 6 meses y < 21 años de edad en el momento de la firma del consentimiento informado y/o del asentimiento, si procede.
4. El sujeto ha sido diagnosticado de AML de acuerdo a la clasificación French–American–British (FAB) con >= 5% de blastos en médula ósea, con o sin enfermedad extramedular (excepto los sujetos con leucemia activa en sistema nervioso central [central nervous system, CNS]).
5. El sujeto presenta una puntuación de Karnofsky >= 50 (si tiene >= 16 años) o una puntuación de Lansky >= 50 (si tiene < 16 años).

E.4

Principal exclusion criteria

1. Subject has active CNS leukemia.
2. Subject has uncontrolled or significant cardiovascular disease
3. Subject has systemic fungal, bacterial, viral or other infection that is exhibiting ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment. The subject needs to be off pressors and have negative blood cultures for 48 hours.
5. Subject is receiving or plans to receive concomitant chemotherapy, radiation therapy, or immunotherapy other than as specified in the protocol.
6. Subject has active clinically significant GVHD or is on treatment with systemic corticosteroids and is receiving > 0.5 mg/kg of prednisone (or equivalent) daily dose for GVHD.
7. Subject has active malignant tumors other than AML.
8. Subject has hypokalemia and/or hypomagnesemia at Screening (defined as values below institutional lower limit of normal [LLN]). Repletion of potassium and magnesium levels during the screening period is allowed.
9. Subject requires treatment with concomitant drugs that are strong inducers of cytochrome P450 (CYP)3A.
10. Subject must wait for at least 5 half-lives after stopping therapy with any investigational agent and before starting gilteritinib.

1. El sujeto presenta leucemia activa en sistema nervioso central.
2. El sujeto presenta enfermedad cardiovascular no controlada o importante,
3. El sujeto presenta una infección sistémica fúngica, bacteriana, vírica o de otro tipo con signos/síntomas actuales de infección que no mejoran a pesar del tratamiento antibiótico o de otro tipo adecuado. El sujeto deberá estar sin vasopresores y presentar hemocultivos negativos desde al menos 48 horas antes.
5. El sujeto está recibiendo o está programado para recibir de forma concomitante quimioterapia, radioterapia o inmunoterapia distintas a lo especificado en el protocolo.
6. El sujeto presenta una GVHD activa clínicamente importante o se encuentra en tratamiento con corticosteroides sistémicos a una dosis diaria > 0,5 mg/kg de prednisona (o equivalente) por GVHD.
7. El sujeto presenta una neoplasia maligna activa distinta de AML.
8. El sujeto presenta hipopotasemia y/o hipomagnesemia en la selección (definidas como valores por debajo del límite inferior de la normalidad [lower limit of normal, LLN] del centro). Se permite la reposición de los niveles de potasio y magnesio durante el periodo de selección.
9. El sujeto precisa tratamiento concomitante con fármacos que son inductores potentes de la isoenzima (CYP)3A del citocromo P450.
10. Antes de comenzar con gilteritinib, el sujeto debe esperar a que transcurran como mínimo 5 semividas después de la suspensión de cualquier fármaco experimental.

E.5 End points

E.5.1

Primary end point(s)

Phase 1:
Determination of MTD and/or RP2D based on the DLT and biologic activity according to PIA.
Phase 2:
o CRc rates (overall best response) after 2 cycles of therapy.
o CR rates after 2 cycles of therapy

Fase 1:
Determinación de la MTD y/o la RP2D en función de la DLT y de la actividad biológica según la PIA.
Fase 2:
o Tasas de CRc (mejor respuesta global) tras 2 ciclos de tratamiento.
o Tasas de CR tras 2 ciclos de tratamiento

E.5.1.1

Timepoint(s) of evaluation of this end point

Phase 1:
DLT observation period will be 28 days from the start of cycle 1 day 1; for the first cycle only.

Phase 2:
CRc and CR rates after 2 cycles of therapy.

Fase 1:
El periodo de observacion de la DLT será de 28 días desde que empieza el día 1 del ciclo 1; unicamente para el primer ciclo.

Fase 2:
Tasas de CRc y CR tras 2 ciclos de tratamiento

E.5.2

Secondary end point(s)

-Inhibition of phosphorylated FLT3 (pFLT3) measured by PIA assay
-Gilteritinib plasma concentration
-Pharmacokinetic parameters (e.g., CL/F, Vd/F) of gilteritinib (and active metabolites if warranted)
-Safety, tolerability and toxicity assessments of gilteritinib when given in combination with FLAG-DNX and FLAG
-EFS rate
-OS rate
-MRD assessment
-Acceptability and palatability assessment of the liquid formulation

- Inhibición de FLT3 fosforilado (pFLT3), en su medición mediante el método de la PIA
- Concentración plasmática de gilteritinib
- Parámetros farmacocinéticos (esto es, aclaramiento oral [CL/F], volumen de distribución aparente [Vd/F] de gilteritinib (y de sus metabolitos activos, si procede)
- Seguridad, tolerabilidad y toxicidad de gilteritinib en su administración en combinación con FLAG
- Tasa de EFS
- Tasa de OS
- MRD
- Aceptabilidad y palatabilidad de la formulación líquida

E.5.2.1

Timepoint(s) of evaluation of this end point

After 2 cycles of therapy

Tras 2 ciclos de tratamiento

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

Yes

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

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Acceptability and palatability assessment of the liquid formulation

Aceptabilidad y palatabilidad de la formulación líquida

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

First use in children

Primera administración en niños

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

Yes

E.8.1.7.1

Other trial design description

combinación secuencial con FLAG

sequential combination with FLAG

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

Germany

Italy

Japan

Spain

United Kingdom

United States

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

Ultima visita del ultimo 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

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Male and female children and adolescents from 6 months to less than 21 years of age.

Niños, adolescentes y adultos jóvenes, de ambos sexos, de edad comprendida entre 6 meses y menos de 21 años

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

114

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)

None

Ninguno

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

2019-12-10

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-12-03

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

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