E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
A blood cancer called Acute Myeloid Leukemia |
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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 | 21.1 |
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. |
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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. |
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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 |
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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 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 |
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall survival compared between the treatment groups. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Individual subjects will be followed until death. |
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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 |
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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. |
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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 | Yes |
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 | 2 |
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 | 40 |
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 |
United States |
France |
Ireland |
Italy |
Netherlands |
Spain |
United Kingdom |
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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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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 |