E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic myeloid leukemia (CML) |
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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 determine the rate and duration of the hematologic response (CHR or RCP) as obtained after an induction phase. |
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E.2.2 | Secondary objectives of the trial |
To estimate the time to disease progression. To estimate the overall survival. To evaluate the cytogenetic and molecular responses.
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
a) Male or female patients aged 18 to 80 years. b) Patients who participated to protocol HHT-CML/INTL/04.2 and who have either achieved a Complete Hematological Response or returned to chronic phase of CML after one or two courses of subcutaneous HHT. c) Estimated life expectancy of at least 12 weeks. d) Adequate performance status as defined by a grading of 2 or better on the ECOG Performance Status Criteria. e) Female patients of child-bearing potential willing to use adequate contraception (oral contraceptive pill, IUD, surgical sterilisation, contraceptive patches, depot injection or barrier method in combination with a spermicide) for the duration of and at least one month after the last dose of investigational product. f) Male patients willing to use adequate contraception for the duration of and at least three months after the last dose of investigational product. g) Patients who have given written informed consent after having read and understood the patient information sheet. |
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E.4 | Principal exclusion criteria |
a)Patients who did not achieve CHR or return to Chronic Phase of CML while treated with subcutaneous HHT in protocol HHT-CML/INTL/04.2. b) Primary resistance to HHT. c) Reappearance of hematological features consistent with accelerated phase or progression to blastic phase of CML. d) Ongoing grade 3-4 hematological toxicity related. e) Patients who need to receive anti-leukemic agents other than HHT, hydroxyurea (HU) or anagrelide during the trial. f) Female patients who are pregnant (confirmed by a serum pregnancy test) or lactating. g) NYHA class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia requiring therapy, uncontrolled hypertension or congestive heart failure. h) Creatinine levels more than 3 x ULN (upper limit of normal range) at the laboratory where the analysis was performed. i) Total serum bilirubin more than 3 x ULN in patients without clinically suspected leukemia involvement of the liver. j) AST (SGOT) or ALT (SGPT) more than 5 x ULN in patients without clinically suspected leukemia involvement of the liver. k) Any other reason, which the Investigator feels, would preclude safe inclusion of the patient. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Hematologic response rate and duration.
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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 | No |
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 | 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.8.3 |
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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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 | 3 |
E.8.9.1 | In the Member State concerned days | |
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 | 3 |