E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of Acute Myeloid Leukemia (AML) |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective is to compare overall survival of subjects treated with tipifarnib with that of subjects treated with best supportive care including hydroxyurea. |
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E.2.2 | Secondary objectives of the trial |
(1) to compare the following end points between subjects treated with tipifarnib and subjects treated with best supportive care including hydroxyurea. - Progression-Free Survival (PFS) - Complete remission (CR) rate - Rate of morphologic leukemia-free state - One-year survival estimate - Health resources utilization, including: Duration of hospitalization Hospitalization for infections Blood product transfusion (2) to characterize the safety profile of tipifarnib. (3) to characterize potential genetic markers that may be predictive of response to tipifarnib
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
- 70 years of age or older - Newly diagnosed, de novo or secondary AML - Subject not medically fit for, or does not wish to be treated with, combination induction chemotherapy - Pathologic confirmation of AML (>20% bone marrow leukemic blasts) - Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2 (recorded on day of randomization) - Subject has signed the informed consent document. Consent may not be given by a legal representative.
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E.4 | Principal exclusion criteria |
- Previous cytotoxic or biologic treatment for AML - Acute promyelocytic leukemia (APL) - Absolute peripheral blast count >30,000/mm3 - Central nervous system leukemia - Serum biochemical values as follows: Creatinine clearance (calculated by Cockcroft-Gault formula) less than 60ml/min Total bilirubin greater than 1.5 times ULN (CTC Grade 1) ALT (alanine transaminase) and AST (aspartate transaminase) greater than 2.5 times ULN (CTC Grade 1) - Uncontrolled systemic infection - Uncompensated disseminated intravascular coagulation or uncontrolled bleeding - Symptomatic neuropathy of grade 2 or worse - Known allergy to imidazole drugs, such as clotrimazole, ketoconazole, miconazole, econazole, fenticonazole, isoconazole, sulconazole, ticonazole or terconazole
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E.5 End points |
E.5.1 | Primary end point(s) |
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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 | No |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
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 | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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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.8.5 | The trial involves multiple Member States | Yes |
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 | Information not present in EudraCT |
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 |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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The study will be considered complete when all subjects have either died, or up to 4 months after clinical cut-off date. The clinical cutoff date for the analysis of efficacy will be based on the number of events (deaths). This is estimated to occur approximately 4 months after the last subject is randomized. |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 9 |