E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute Myeloid Leukaemia (AML), Chronic Lymphocytic Leukaemia (CLL) and B-cell Non-Hodgkins Lymphoma (BNHL) |
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E.1.1.1 | Medical condition in easily understood language |
Two types of Leukaemia and one type of Lymphoma. |
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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 | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10029547 |
E.1.2 | Term | Non-Hodgkin's lymphoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000886 |
E.1.2 | Term | Acute myeloid leukemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10008976 |
E.1.2 | Term | Chronic lymphocytic leukemia |
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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The principal research question of this study is to determine if BaP given to patients with AML and MDS (RAEB2), CLL and BNHL is safe, feasible (i.e. compliance is reasonable) and has anti-cancer activity.
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E.2.2 | Secondary objectives of the trial |
The study will also assess patient quality of life via validated quality of life questionnaire. The study will also register overall survival. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients must: o have one of the following diagnoses: - AML or high risk myelodysplasia (RAEB2 WHO criteria) - CLL - BNHL o be 18 years or older o have given written informed consent
For AML and RAEB-2 • Haemopoiesis must be impaired by the disease as judged by an abnormal FBC (International Working Group response criteria in myelodysplasia [2]) and, where there is doubt as to the cause of impaired haemopoiesis, there must be bone marrow aspirate evidence that impaired haemopoiesis is due to cancer involvement of the bone marrow. • Abnormal values are haemoglobin level less than 11 g/dL or RBC transfusion dependence, platelet count less than 100 x 109/L or platelet-transfusion dependence, absolute neutrophil count less than 1.0x 109/L. Pretreatment baseline measures of cytopenias are averages of at least 2 measurements (not influenced by transfusions, ie, no RBC transfusions for at least 1 week and no platelet transfusions for at least 3 days) over at least 1 week prior to therapy.
For CLL and BNHL • Patients must have either measurable disease (tumour cells in blood at >5 x 109/L, or lymphadenopathy> 1cm) or bone marrow failure due to disease as stated above for MDS/AML.
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E.4 | Principal exclusion criteria |
• Patient considered suitable for other forms of anti-cancer therapy (either accepted standard therapy or therapy in the context of a clinical trial) other than palliative corticosteroids or hydroxyurea • Patient has eGFR<40ml/min • Patient known to be allergic to trial drugs • Patient has received treatment with any investigational medicinal product within the previous 28 days • Patient unable to swallow orally administered medications • Patient has uncontrolled seizures • Patient has active infection requiring systemic antibiotics, antifungal or antiviral drugs • Patient has concurrent severe and/or uncontrolled medical condition (e.g. severe COPD, severe Parkinsons’s disease) or psychiatric condition • Women of child-bearing potential and men who have partners of child-bearing potential who are not willing to practise effective contraception for the duration of the study and for three months after the last study drug administration • Pregnant or lactating women. Pre-menopausal women of child bearing potential must have a negative urine or serum pregnancy test within 7 days prior to registration.
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E.5 End points |
E.5.1 | Primary end point(s) |
• Safety: The number of grade 3 and 4 Adverse Reactions and Serious Adverse Reactions (SARs) attributable to the trial drugs
• Patient compliance: Percentage of allocated treatment taken
• Activity: o Haematological Response in the first 18 weeks of treatment o Clinical Response in the first 18 weeks of treatment
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
When patients have been on treatment for 18 weeks. |
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E.5.2 | Secondary end point(s) |
Quality of Life Overall survival will also be registered. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Quality of life questionnaires completed at baseline will be compared to QoL questionnaires completed between week 7-11 and at week 18.
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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 | 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 | No |
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 | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
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 end of trial will be 6 months from the last data capture. This will allow sufficient time for the completion of protocol procedures, data collection and data input. |
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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 | 4 |
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 | 4 |