Clinical Trials Register

Summary
EudraCT Number:	2017-001178-41
Sponsor's Protocol Code Number:	TPI-ALV-201
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2017-10-02
Trial results	View 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

2017-001178-41

A.3

Full title of the trial

A Phase 2, Randomized, Biomarker-driven, Clinical Study in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML) with an Exploratory Arm in Patients with Newly Diagnosed High-Risk AML

Estudio clínico de fase II, aleatorizado, basado en biomarcadores, en pacientes con leucemia mieloide aguda (LMA) recidivante o resistente con un grupo exploratorio para pacientes con diagnóstico reciente de LMA de alto riesgo.

A.3.1

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

A Phase 2 Clinical Trial in Patients with Acute Myeloid Leukemia (AML).

Estudio clínico de fase II en pacientes con leucemia mieloide aguda (LMA).

A.4.1

Sponsor's protocol code number

TPI-ALV-201

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02520011

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	Tolero Pharmaceuticals, Inc.
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	Tolero Pharmaceuticals, Inc.
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	Tolero Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	Mike Bernstein
B.5.3	Address:
B.5.3.1	Street Address	2975 W. Executive Parkway, Suite 320
B.5.3.2	Town/ city	Lehi, Utah
B.5.3.3	Post code	84043
B.5.3.4	Country	United States
B.5.4	Telephone number	+34689772554
B.5.5	Fax number	+1801-746-3314
B.5.6	E-mail	mbernstein@toleropharma.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/3/15/1437
D.3 Description of the IMP
D.3.1	Product name	Alvocidib
D.3.2	Product code	Alvocidib (Flavopiridol)
D.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	alvocidib hydrochloride
D.3.9.1	CAS number	131740-09-5
D.3.9.2	Current sponsor code	Alvocidib
D.3.9.3	Other descriptive name	Alvocidib (formerly flavopiridol)
D.3.9.4	EV Substance Code	SUB22877
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	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	Comparator
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	Cytarabine Injection Solution 20 mg/ml
D.2.1.1.2	Name of the Marketing Authorisation holder	Hospira UK Ltd
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Cytarabine
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Cytarabine
D.3.9.1	CAS number	147-94-4
D.3.9.3	Other descriptive name	CYTARABINE
D.3.9.4	EV Substance Code	SUB06880MIG
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	20
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	Comparator
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	Mitoxantrone 2 mg/ml Concentrate for Solution for Infusion
D.2.1.1.2	Name of the Marketing Authorisation holder	Hospira UK Limited
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Mitoxantrone
D.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	MITOXANTRONE HYDROCHLORIDE
D.3.9.1	CAS number	70476-82-3
D.3.9.4	EV Substance Code	SUB03309MIG
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	2
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

Relapsed/Refractory Acute Myeloid Leukemia (AML)

Leucemia mieloide aguda (LMA) recidivante o resistente.

E.1.1.1

Medical condition in easily understood language

Cancer of the blood and bone marrow cells

Cancer de la sangre y células de médula ósea.

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	10060558
E.1.2	Term	Acute myeloid leukemia recurrent
E.1.2	System Organ Class	100000012987

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	10000886
E.1.2	Term	Acute myeloid leukemia
E.1.2	System Organ Class	100000012984

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	10000887
E.1.2	Term	Acute myeloid leukemia in remission
E.1.2	System Organ Class	100000012977

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	10054294
E.1.2	Term	Acute myeloid leukemia (in remission)
E.1.2	System Organ Class	100000012977

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

Stage 1: To determine the CR rate in patients with relapsed or refractory AML with NOXA BH3 priming ≥40%.
Stage 2: To compare CR rates between patients with relapsed or refractory AML with NOXA BH3 priming ≥40% receiving 1-2 cycles of ACM treatment and those receiving 1-2 cycles of CM.
Exploratory Objective (Stage 2): To determine if treatment with ACM can induce CR in patients with relapsed or refractory AML with NOXA BH3 priming ≥40% who failed to achieve CR following 1-2 cycles of CM.

Etapa 1: Determinar la tasa de RC en los pacientes con LMA recidivante o resistente al tratamiento con cebado mediante NOXA BH3 ≥ 40 %.
Etapa 2: Comparar las tasas de RC entre los pacientes con LMA recidivante o resistente al tratamiento con cebado mediante NOXA BH3 ≥ 40 % que reciben 1-2 ciclos de tratamiento ACM y los pacientes que reciben 1-2 ciclos de tratamiento CM.
Objetivo exploratorio (etapa 2): Determinar si el tratamiento con ACM pueden inducir la RC en pacientes con LMA recidivante o resistente al tratamiento con cebado mediante NOXA BH3 ≥ 40 % que no hayan logrado RC después de 1-2 ciclos de tratamiento CM.

E.2.2

Secondary objectives of the trial

"Not applicable"

No aplica.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

During Prescreening:
1. Be between the ages of ≥18 and ≤65 years
2. Have an established, pathologically confirmed diagnoses of AML by World Health Organization (WHO) criteria excluding acute promyelocytic leukemia (APL-M3) with a bone marrow of >5% blasts based on histology or flow cytometry
3. Be in first relapse (within 24 months of CR) or have primary refractory AML (refractory to initial induction therapy using 1 or 2 cycles of intensive anthracycline/cytarabine ± etoposide or cladribine induction) or have newly diagnosed high-risk AML as defined in this protocol
4. Demonstrate NOXA BH3 priming of ≥40% by mitochondrial profiling in bone marrow or 30-39% for NOXA Exploratory Arm.
During Screening:
5. Have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤2
6. Have a serum creatinine level ≤1.8 mg/dL
7. Have an alanine aminotransferase (ALT)/aspartate aminotransferase (AST) level ≤5 times upper limit of normal (ULN)
8. Have a total bilirubin level ≤2.0 mg/dL (unless secondary to Gilbert syndrome, hemolysis, or leukemia)
9. Have a left ventricular ejection fraction (LVEF) >45% by echocardiogram (ECHO) or multigated acquisition (MUGA) scan
10. Be nonfertile or agree to use an adequate method of contraception. Sexually active patients and their partners must use an effective method of contraception associated with a low failure rate during and for 6 months after completion of study therapy.
11. Be able to comply with the requirements of the entire study.
12. Provide written informed consent prior to any study related procedure. (In the event that the patient is re-screened for study participation or a protocol amendment alters the care of an ongoing patient, a new informed consent form must be signed.)

Durante el proceso previo a la selección:
1.Tener una edad comprendida entre ≥ 18 y ≤ 65 años
2.Tener un diagnóstico de LMA establecido y confirmado por anatomía patológica conforme a los criterios de la Organización Mundial de la Salud (OMS) excluyendo leucemia promielocítica aguda (LPA-M3) con una cifra de blastos en médula ósea > 5 % evaluada mediante histología o citometría de flujo
3.Tener la primera recidiva (en un plazo de 24 meses desde RC) o tener LMA primaria resistente al tratamiento (es decir, resistente al tratamiento de inducción inicial con 1 o 2 ciclos de inducción intensiva con antraciclina/citarabina ± etopósido o cladribina) o pacientes con diagnóstico reciente de LMA de alto riesgo, según lo definido en este protocolo (véase el apartado 4.4.4)
4.Pacientes con cebado mediante NOXA BH3 ≥ 40 % o 30-39 % para el grupo exploratorio NOXA demostrado por determinación del perfil mitocondrial.
Durante la selección:
5.Tener un estado funcional del Eastern Cooperative Oncology Group (ECOG) ≤ 2
6.Tener una concentración sérica de creatinina ≤ 1,8 mg/dl
7.Tener un nivel de alanina-aminotransferasa (ALT)/aspartato-aminotransferasa (AST) ≤ 5 veces el límite superior de la normalidad (LSN)
8.Tener un nivel de bilirrubina total ≤ 2,0 mg/dl (a menos que sea secundario a síndrome de Gilbert, hemólisis o leucemia)
9.Tener una fracción de eyección ventricular izquierda (FEVI) > 45 % evaluada mediante ecocardiografía (Ecocardio) o ventriculografía nuclear (VRN)
10.No ser fértil o usar un método anticonceptivo adecuado. Los pacientes sexualmente activos y sus parejas deben utilizar un método anticonceptivo eficaz asociado a una baja tasa de fallo durante el estudio y hasta 6 meses después de la finalización del tratamiento del estudio (véase el apartado 4.7.3).
11.Ser capaz de cumplir los requisitos de todo el estudio.
12.Otorgar el consentimiento informado por escrito antes de realizar cualquier procedimiento específico del estudio. (En caso de que el paciente repita el proceso de selección para la participación en el estudio o una enmienda al protocolo altere la atención de un paciente que esté participando en el estudio, deberá firmar un nuevo formulario de consentimiento informado.)

E.4

Principal exclusion criteria

1. Received more than 2 cycles of induction therapy for AML. Investigational agents as part of front-line therapy for AML may by acceptable following discussion with the Medical Monitor. Hydroxyurea is permitted (see #5 below).
2. Received any previous treatment with alvocidib or any other CDK inhibitor
3. Received a hematopoietic stem cell transplant within the previous 2 months
4. Have clinically significant graft versus host disease (GVHD), or GVHD requiring initiation or escalation of treatment within the last 21 days
5. Require concomitant chemotherapy, radiation therapy, or immunotherapy. Hydroxyurea is allowed up to the evening before starting (but not within 12 hours) of starting treatment on either arm.
6. Received >360 mg/m2 equivalents of daunorubicin
7. Have a peripheral blast count of >30,000/mm3 (may use hydroxyurea as in #5 above)
8. Received antileukemic therapy within the last 3 weeks (with the exception of hydroxyurea or if the patient has definite refractory disease). Refractory patients who received therapy within the last 3 weeks may be eligible with prior approval of the Medical Monitor.
9. Diagnosed with acute promyelocytic leukemia (APL, M3)
10. Have active central nervous system (CNS) leukemia
11. Have evidence of uncontrolled disseminated intravascular coagulation
12. Have an active, uncontrolled infection
13. Have other life-threatening illness
14. Have other active malignancies or diagnosed with other malignancies within the last 6 months, except nonmelanoma skin cancer or cervical intraepithelial neoplasia
15. Have mental deficits and/or psychiatric history that may compromise the ability to give written informed consent or to comply with the study protocol.
16. Are pregnant and/or nursing.
17. Have received any live vaccine within 14 days prior to first study drug administration.

1.Haber recibido más de 2 ciclos de tratamiento de inducción para la LMA. La presencia de fármacos en fase de investigación como parte de tratamiento de primera línea para la LMA puede ser aceptable después de consultarlo con el monitor médico. La hidroxiurea está permitida (véase el punto n.º 5 que figura a continuación).
2.Haber recibido cualquier tratamiento previo con alvocidib o cualquier otro inhibidor de CDK.
3.Haber recibido un trasplante de células madre hematopoyéticas en los últimos 2 meses.
4.Tener enfermedad de injerto contra huésped (EICH) clínicamente significativa, o EICH que requirió el inicio o el aumento de la dosis de tratamiento en los últimos 21 días.
5.Requerir el uso concomitante de quimioterapia, radioterapia o inmunoterapia. Está permitido el uso de hidroxiurea hasta la noche anterior al inicio del tratamiento (pero no en las 12 horas anteriores) en cualquiera de los grupos.
6.Haber recibido el equivalente a > 360 mg/m2 de daunorubicina (véase el anexo G para consultar la tabla de conversión).
7.Tener un recuento de blastos periféricos > 30 000/mm3 (puede usar hidroxiurea como en el punto n.º 5).
8.Haber recibido tratamiento contra la leucemia en las últimas 3 semanas (con la excepción de hidroxiurea o si el paciente presenta enfermedad resistente al tratamiento definida). Los pacientes resistentes al tratamiento que recibieron tratamiento en las últimas 3 semanas pueden ser aptos para participar con la aprobación previa del monitor médico.
9.Diagnóstico de leucemia promielocítica aguda (LPA, M3).
10.Tener leucemia activa en el sistema nervioso central (SNC).
11.Presentar signos de coagulación intravascular diseminada no controlada.
12.Tener una infección activa no controlada.
13.Tener otra enfermedad potencialmente mortal.
14.Tener otras neoplasias activas o ser diagnosticado de otra neoplasia en los últimos 6 meses, excepto cáncer de piel diferente al melanoma o neoplasia intraepitelial cervical.
15.Tener discapacidades mentales y/o antecedentes psiquiátricos que puedan comprometer la capacidad de otorgar el consentimiento informado por escrito o cumplir el protocolo del estudio.
16.Estar embarazada y/o en periodo de lactancia.
17.Haber recibido cualquier vacuna elaborada con virus vivos en un plazo de 14 días antes de la primera administración del fármaco del estudio.

E.5 End points

E.5.1

Primary end point(s)

Complete Remission (CR) rate = Percentage of patients achieving CR

Tasa de remisión completa (RC) = porcentaje de pacientes que alcanza la RC

E.5.1.1

Timepoint(s) of evaluation of this end point

After every cycle at hematologic recovery or Day 45, whichever occurs fist.

Después de cada ciclo en recuperación hematológica o Día 45, lo que ocurra primero.

E.5.2

Secondary end point(s)

Secondary Efficacy Endpoints
• Overall Survival (OS) = Time from randomization (Day 1) until death from any cause
• Combined CR Rate = Percentage of patients achieving a CR, CRi or CRp
• Combined Response Rate = Percentage of patients achieving a CR, CRi, CRp, or PR
• Rate of Stem Cell Transplantation = Percentage of patients proceeding directly to stem cell transplantation
• Event-free Survival (EFS) = Time from randomization (Day 1) until (a) treatment failure, (b) relapse after CR, or (c) death from any cause, whichever occurs first, censored at 2 years
The CR rate in patients failing CM and crossing over to receive ACM will also be determined.

Criterios secundarios de valoración
•Supervivencia global (SG) = Tiempo desde el momento de la aleatorización (día 1) hasta la muerte por cualquier causa
•Tasa de RC combinada = Porcentaje de pacientes que alcanzan RC, RCi o RCp
•Tasa de respuesta combinada = Porcentaje de pacientes que alcanzan RC, RCi, RCp o RP
•Tasa de trasplante de células madre = Porcentaje de los pacientes que procedan directamente al trasplante de células madre
•Supervivencia sin acontecimientos (SSA) = Tiempo transcurrido desde la aleatorización (día 1) hasta (a) fracaso del tratamiento, (b) recidiva después de RC o (c) muerte por cualquier causa, lo que suceda primero, con censura a los 2 años
También se determinará la tasa de RC en pacientes que fracasan en 1-2 ciclos de CM y pasan a recibir a recibir ACM.

E.5.2.1

Timepoint(s) of evaluation of this end point

Follow-up by telephone every month during 12 months from month 1 to month 13 from randomization and then every 2 months from month 14 to 24 from randomization. This follow-up is made after receiving 4 cycles and off-study visit.

Seguimiento telefónico mensual durante 12 meses desde el mes 1 al mes 13 desde la aleatorización y luego cada 2 meses desde el mes 14 al 24 desde la aleatorización. Este seguimiento se hace después de recibir 4 ciclos y una visita de salida del estudio.

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

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

Yes

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Citarabina (Ara-c) y mitoxantrona (mitoxantrona hidrocloruro).

cytarabine (Ara-c) and mitoxantrone (mitoxantrone hydrochloride)

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the 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

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

After maximum of 4 cycles of CM/ACM, patients will have an-off study visit (in clinic) and then be followed by telephone contact every month for first 12 months after date of randomization (month 1 to month 13) and then every two months from month 14 to month 24 after date of randomization.

Después de un máximo de 4 ciclos de CM/ACM, los pacientes tendrán una última visita (en el hospital) y después serán seguidos por teléfono de manera mensual durante los primeros 12 meses después de la aleatorización (del mes 1 al mes 13) y luego, cada dos meses desde el mes 14 al mes 24 después de la fecha de aleatorización.

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

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)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

119

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

119

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

2018-01-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-11-15

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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