E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Immune thrombocytopenia patients with low platelets and in need of surgery. |
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E.1.1.1 | Medical condition in easily understood language |
immune thrombocytopenia (ITP) can result in bleedings. When in need of surgery platelets might need increasing for adequate clotting after the procedure. |
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E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutic techniques [E02] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10023095 |
E.1.2 | Term | ITP |
E.1.2 | System Organ Class | 100000004851 |
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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 compare the effect of eltrombopag and IVIG on the achievement of the platelet count threshold before and after surgery in ITP patients. |
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E.2.2 | Secondary objectives of the trial |
- Time to treatment failure; - Bleeding; - Proportion of patients who undergo surgery as planned; - Treatment satisfaction assessed on Day -1 +/-1 day and once during follow up using the Treatment Satisfaction Questionnaire for Medications Score (which incorporates effectiveness, convenience, side effects, and overall satisfaction); - Proportion of patients who have a platelet count greater than 400x109/L during the pre- and post-operative period; - Use of blood transfusions (platelets, red blood cells, plasma); - Pre-surgery platelet count levels; - Change in pre-surgery platelet count levels from baseline; - Proportion of post-surgery days spent below the platelet count threshold [50x 109/L (for minor surgery); 100x 109/L (for major surgery)] during the study period; - Total clinic and hospital days; - Venous thromboembolism and arterial thromboembolism; - Adverse events. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Primary or secondary ITP (according to the ASH 2011 guidelines); 2. Platelet count below the surgical platelet count threshold (50 x109/L for minor surgery; 100 x 109/L for major surgery); 3. 18 years of age or older; 4. On stable doses of concomitant ITP medications (or no medication) for at least 2 weeks (i.e. the dose administered has not changed); 5. At least 3-weeks lead time available between randomization and scheduled surgery; 6. IVIG and Eltrombopag are acceptable ITP treatment options for this patient; 7. Able to provide informed consent. |
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E.4 | Principal exclusion criteria |
1. Pregnancy or breastfeeding; 2. Treatment with IVIG within the last 2 weeks; 3. Treatment with a thrombopoietin receptor agonist (eltrombopag or romiplostim) within the last 4 weeks; 4. AST, ALT above 2X upper limit of normal; 5. Bilirubin above 1.5X upper limit of normal in the absence of clinically benign liver disorder (e.g. Gilberts syndrome); 6. Deep vein thrombosis, myocardial infarction, thrombotic stroke or arterial thrombosis in the last 12 months; 7. History of bone marrow reticulin or fibrosis; 8. Known liver cirrhosis; 9. Active malignancy (defined as requiring treatment or palliation within the last 6 months). 10. Any additional laboratory test result, health related illness or other diagnosis which, in the opinion of the treating physician, may put the subject's health or safety at risk. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary: Proportion of patients achieving the platelet count threshold before surgery and maintaining platelet counts within the target range until 7 days after surgical hemostasis is achieved without the use of ITP rescue treatment. |
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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) |
- Time to treatment failure; - Bleeding; - Proportion of patients who undergo surgery as planned; - Treatment satisfaction assessed on Day -1 +/-1 day and once during follow up using the Treatment Satisfaction Questionnaire for Medications Score (which incorporates effectiveness, convenience, side effects, and overall satisfaction); - Proportion of patients who have a platelet count greater than 400x109/L during the pre- and post-operative period; - Use of blood transfusions (platelets, red blood cells, plasma); - Pre-surgery platelet count levels; - Change in pre-surgery platelet count levels from baseline; - Proportion of post-surgery days spent below the platelet count threshold [50x 109/L (for minor surgery); 100x 109/L (for major surgery)] during the study period; - Total clinic and hospital days; - Venous thromboembolism and arterial thromboembolism; - Adverse events. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Weekly 3 weeks prior to surgery and weekly 4 weeks after surgery. |
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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 | No |
E.6.5 | Efficacy | No |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
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 | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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 | 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.8.9.1 | 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 | 0 |
E.8.9.2 | In all countries concerned by the trial years | 6 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |