E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Immune thrombocytopenic purpura (ITP) |
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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 efficacy of SB-497115-GR as a thrombopoietic agent, when administered once daily for 6 weeks to adult patients with refractory chronic ITP. |
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E.2.2 | Secondary objectives of the trial |
- To assess the safety and tolerability of SB-497115-GR when administered once daily for 6 weeks to adult patients with refractory chronic ITP. - To characterize the population pharmacokinetic profile of oral SB-497115-GR using a combined sparse and serial pharmacokinetic sampling strategy when administered once daily for 6 weeks to adult patients with refractory chronic ITP. - To determine the pharmacodynamic effect of SB-497115-GR on markers of thrombopoiesis when administered once daily for 6 weeks to adult patients with refractory chronic ITP. - To assess the impact of SB-497115-GR on the incidence and severity of symptoms of thrombocytopenia when administered once daily for 6 weeks to adult patients with refractory chronic ITP. - To assess the impact of SB-497115-GR on the quality of life when administered once daily for 6 weeks to adult patients with refractory chronic ITP.
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. Diagnosed with chronic ITP at least 6 months prior to screening for this study with a platelet count of <30,000 platelets/L. 2. Refractory patients who have failed at least one previous treatment for chronic ITP, including corticosteroids, immunoglobulins, azathioprin, danazol, immunomodulators and/or splenectomy. 3. Subjects treated with maintenance corticosteroid therapy must be receiving a steroid dose that has been stable for at least 1 month. 4. No past history of myocardial infarction or clinically significant ECG findings at screening. 5. Normal PT and aPTT, no history of clotting disorder, other than ITP. 6. Male, or female subjects that agree to use acceptable methods of contraception [including an intrauterine device (IUD)] and two forms of barrier contraception if engaging in sexual intercourse for at least 7 days prior to the first dose of study medication and continuing until 30 days after the final dose of study medication. 7. Aged over 18 years. 8. A signed and dated written informed consent is obtained from the subject. |
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E.4 | Principal exclusion criteria |
1. Any clinically relevant abnormality, other than ITP, identified on the screening examination or any other medical condition or circumstance, which in the opinion of the Investigator, makes the subject unsuitable for participation in the study. 2. History of thrombosis within the last year. 3. For female subjects, a positive serum or urine human chorionic gonadotrophin pregnancy test at screening or predose on Day 1. 4. For female subjects, no current hormone replacement therapy or current oral contraceptive use. 5. History of alcohol/drug abuse or dependence within 12 months of the study. 6. Treatment with an investigational drug within 30 days or five half-lives (whichever is longer) preceding the first dose of study medication. 7. Consumption of aspirin, aspirin-containing compounds, salicylates, milk of magnesia, or nonsteroidal anti-inflammatories (NSAIDs) within 3 weeks of the study start and until the end of the study. 8. Consumption of substrates of the cytochrome P450 2C9 isozyme, including diclofenac, ibuprofen, meloxicam, naproxen, flurbiprofen, piroxicam, suprofen, tolbutamide, glipizide, losartan, irbesartan, glibenclamide, glimepiride, amitriptyline, celecoxib, fluoxetine, fluvastatin, glyburide, fluoxetine, fluvastatin, glyburide, nateglinide, phenytoin, rosiglitazone, tamoxifen, torsemide and S-warfarin. 9. Consumption of any herbal or dietary supplements, excluding vitamin or mineral supplements, within 1 week of the study start. 10. A complete blood count (CBC) and/or reticulocyte count outside the reference range, except for platelet count. 11. History of platelet aggregation that prevents reliable measurement of platelet counts. 12. History of infection with HIV, hepatitis B virus or hepatitis C virus. |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Proportion of subjects with a platelet count of >50,000/uL after 42 days of dosing. Patients who meet this criterion will be referred to as treatment responders. |
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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 | Yes |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Yes |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
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 | 6 |