E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute Myeloid Leukemia |
Leucemia mieloide aguda |
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E.1.1.1 | Medical condition in easily understood language |
A blood cancer called Acute Myeloid Leukemia |
Un cáncer de sangre llamado Leucemia Mieloide Aguda |
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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 | PT |
E.1.2 | Classification code | 10000880 |
E.1.2 | Term | Acute myeloid leukaemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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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 |
The primary objective of this trial is to compare overall survival (OS) achieved with uproleselan administered with chemotherapy versus chemotherapy alone. |
El objetivo principal de este estudio es comparar la supervivencia global (SG) lograda con uproleselan administrado con quimioterapia frente a la quimioterapia sola. |
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E.2.2 | Secondary objectives of the trial |
The key secondary objectives of this trial are as follows: • To compare the incidence of severe oral mucositis with onset during the induction period with uproleselan administered with chemotherapy versus chemotherapy alone. • To compare the remission rate (complete remission [CR]/complete remission with partial count recovery [CRh]) achieved with uproleselan administered with chemotherapy versus chemotherapy alone. • To compare the CR rate achieved with uproleselan administered with chemotherapy versus chemotherapy alone. |
Los objetivos secundarios principales de este estudio son los siguientes: • Comparar la incidencia de la mucositis oral grave aparecida durante el período de inducción con uproleselan administrado con quimioterapia frente a la quimioterapia sola. • Comparar la tasa de remisión (remisión completa [RC]/remisión completa con recuperación parcial del recuento [RCh]) lograda con uproleselan administrado con quimioterapia frente a la quimioterapia sola. • Comparar la tasa de RC lograda con uproleselan administrado con quimioterapia frente a la quimioterapia sola. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
The most important inclusion criteria are:: • ≥18 years and ≤75 years in age • Patients with relapsed or refractory AML • No more than one prior stem cell transplant • Has not received the chemotherapy regimen to be used for induction on this trial • Is considered medically eligible to receive the chemotherapy regimen to be used for induction on this trial |
Los criterios de inclusión más importantes son:: • ≥18 años y ≤75 años de edad • Pacientes con recaida o refractaria LMA • No más que un trasplante de células madre • No hayan recibido el régimen de quimioterapia que se utilizará para la inducción en este ensayo • Se considere que cumple los requisitos desde el punto de vista médico para recibir el régimen de quimioterapia que se utilizará para la inducción en este ensayo |
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E.4 | Principal exclusion criteria |
The most important exclusion criteria are: • Patients with acute promyelocytic leukemia, acute leukemia of ambiguous lineage (biphenotypic leukemia), chronic myeloid leukemia with myeloid blast crisis, or secondary refractory AML • Active signs or symptoms of CNS involvement by malignancy • Stem cell transplantation ≤4 months prior to dosing • Any immunotherapy or radiotherapy therapy within 28 days of dosing; any other experimental therapy or chemotherapy within 14 days of dosing • Prior use of G-CSF, CM-CSF or plerixafor within 7 days of dosing • Inadequate organ function • Abnormal liver function • Known active infection with hepatitis A, B, or C, or human immunodeficiency virus • Moderate kidney dysfunction (glomerular filtration rate <45 mL/min) • Uncontrolled acute life-threatening bacterial, viral, or fungal infection • Clinically significant cardiovascular disease • Major surgery within 4 weeks of dosing |
Los criterios de exclusión más importantes son: • Pacientes con leucemia promielocítica aguda, leucemia aguda de linaje ambiguo (leucemia bifenotípica), leucemia mieloide crónica con crisis de blastos mieloides o LMA refractaria secundaria • Signos o síntomas activos de afectación del cáncer al SNC • Trasplante de células madre ≤ 4 meses antes de la dosis • Cualquier inmunoterapia o radioterapia dentro de los 28 días de la dosis; cualquier otro tratamiento experimental o antineoplásico dentro de los 14 días de la dosis • Uso previo de G-CSF, GM-CSF o plerixafor dentro de los 7 días anteriores a la dosis • Función inadecuada de órgano • Función hepática anormal • Infección activa conocida por hepatitis A, B, o C, o virus de la inmunodeficiencia humana • Disfunción renal moderada (tasa de filtración glomerular < 45mL/min) • Infección bacteriana, viral o micótica aguda no controlada que ponga en peligro la vida • Enfermedad cardiovascular clinicamente significativa • Cirugía mayor dentro de las 4 semanas anteriores a la dosis |
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall survival compared between the treatment groups. |
Supervivencia global comparada entre los grupos de tratamiento. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Individual subjects will be followed until death. |
Se seguirán sujetos individuales hasta la muerte. |
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E.5.2 | Secondary end point(s) |
• Incidence of severe oral mucositis during the induction period • Remission rate (CR/CRh) • Complete remission (CR) rate |
• Incidencia de mucositis oral grave durante el período de inducción • Tasa de remisión (RC/RCh) • Tasa de remisión completa (RC) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
• Subjects will be actively monitored for oral mucositis at Baseline, then Day 2 prior to chemotherapy, and continue every 3-4 days (i.e., twice weekly) while in the hospital and once weekly (every 7 ± 2 days) while outpatient during the induction period •Disease response assessment will be done within 7 days of counts recovering, as early as 21 days after the first dose of induction chemotherapy, prior to subsequent therapy ,or by 60 days after the first dose of chemotherapy if bone marrow otherwise indicates remission and counts have not recovered. |
• Los sujetos serán monitorizados activamente para la mucositis oral en el momento basal, y en el Día 2 antes de la quimioterapia, y después cada 3-4 días (es decir, dos veces por semana) mientras se encuentre hospitalizado y una vez por semana (cada 7 ± 2 días) mientras sea paciente ambulatorio durante el periodo de inducción • La evaluación de la respuesta a la enfermedad se realizará dentro de los 7 días posteriores a la recuperación de los recuentos, tan pronto como 21 días después de la primera dosis de quimioterapia de inducción, antes de la terapia posterior, o antes de los 60 días posteriores a la primera dosis de quimioterapia si la médula ósea indica la remisión y los recuentos no se han recuperado. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
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 | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
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 | 7 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
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 | No |
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 |
Canada |
Germany |
Ireland |
Italy |
Netherlands |
Spain |
United States |
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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
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LVLS |
Ultima visita último sujeto |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |