E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Philadelphia Chromosome Positive Acute Lymphocytic Leukemia (Ph+ALL) |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
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 |
To evaluate efficacy (hematologic response) of STI571 in patients diagnosed Philadelphia-Positive Acute Lymphocytic Leukemia (Ph+ALL) |
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E.2.2 | Secondary objectives of the trial |
To evaluate efficacy (response of duration, cytogenetic response) and safety of STI571 in patients diagnosed Philadelphia-Positive Acute Lymphocytic Leukemia (Ph+ALL) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Histologically confirmed diagnosis of Ph+ or bcr-abl + ALL. Refractory to or ineligible for initial remission induction therapy remission induction therapy
Patients with histologically confirmed diagnosis of ALL.
•Patients confirmed to be Ph chromosome positive or bcr-abl gene positive.
•Patients in relapse
•Patients refractory to initial remission induction therapy
•Patients ineligible for initial remission induction therapy
•Patients with an ECOG Performance Status Score from 0 to 2
•Serum creatinine concentration of not more than 2 × the upper limit of the normal range (ULN)
•AST (SGOT) and ALT (SGPT) of not more than 3 × ULN. In patients with hepatic dysfunction from leukemic involvement, AST (SGOT) and ALT (SGPT) should be not more than 5 × ULN
•Serum bilirubin level not more than 3 × ULN
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E.4 | Principal exclusion criteria |
to receive any hematopoietic stem cell transplantation within 6 weeks of the planned initiation of the study drug
Patients with findings indicative of leukemic involvement of the central nervous system
•Patients with any serious concomitant medical condition (e.g., poorly controllable infection, interstitial pneumonia, pulmonary fibrosis, congestive cardiac failure, poorly controlled diabetes mellitus, mental disorder)
•Patients expected to receive any hematopoietic stem cell transplantation within 6 weeks of the planned initiation of the study drug
•Patients having received any hematopoietic stem cell transplantation who have a Grade 3 or 4 GVHD.
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E.5 End points |
E.5.1 | Primary end point(s) |
To evaluate hematologic response to STI571 in patients diagnosed Philadelphia-Positive Acute Lymphocytic Leukemia (Ph+ALL) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
To valuate cytogenetic response, physical and laboratory evaluations, adverse event monitoring, and ECGs |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
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 | 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 | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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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.8.9.2 | In all countries concerned by the trial years | 3 |