E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
paroxysmal nocturnal hemoglobinuria |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10034042 |
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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 |
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic paroxysmal hemoglobinuria (PNH) |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. Individuals at least 18 years of age 2. Patients must have required at least 4 episodes of transfusions in the 12 months prior to Visit 1 for anemia or anemia-related symptoms 3. Presence of a GPI deficient red blood cell clone (type III cells) by flow cytometry equal to or greater than 10% 4. Patients taking erythropoeitin must be on a stable dose for 26 weeks prior to Visit 1 and the dose must be expected to remain stable during the observation period and the treatment phase 5. Patients taking immunosuppressants must be on a stable dose for at least 26 weeks prior to visit 1 and the dose must be expected to rermain stable during the Observation Period and the Treatment Phase 6. Other criteria specified in the Protocol |
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E.4 | Principal exclusion criteria |
1. Patients whose mean hemoglobin levels prior to transfusion over the previous 12 months is greater than 10.5 gm/dL 2. Absolute neutrophil count < 500 per microlitre 3. Presence or suspicion of active bacterial infection in the opinion of the investigator, at Visit 3 or recurrent bacterial infections 4. Known or suspected hereditary complement deficiency 5. Participation in any other drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to Visit 1 6. Pregnant, breast-feeding or intending to conceive during the course of the study, including the post-treatment phase 7. Any of the criteria stated in the Protocol
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E.5 End points |
E.5.1 | Primary end point(s) |
Co-primary endpoints for this study are: hemoglobin stablization and the number of PRBC units transfused during the treatment phase of the study to 26 weeks.
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Yes |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| Information not present in EudraCT |
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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The end of the trial will be when the last patient to receive either eculizumab or placebo completes Visit 18 (26 weeks following first dose). If this patient does not participate in the extension trial, the study concludes after 8 weeks following last dose of investigational product.
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 8 |
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 | 0 |