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 | No |
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) | Information not present in EudraCT |
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) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |