E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10057863 |
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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 study is designed to assess the efficacy and safety of Vivaglobin in patients with PID who have not received prior immunoglobulin treatment |
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E.2.2 | Secondary objectives of the trial |
1.IgG increase (change from baseline) on Day 12 2.Overall rate of infections 3.Total serum IgG trough levels 4.Serum concentrations of specific IgGs 5.Health-related quality of life as measured by an adapted SF-36 questionnaire (age >= 14 years) or CHQ-PF50 (age >= 13 years) 6.Use of antibiotics for infection prophylaxis and treatment |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1.Written informed consent, age-adapted Male or female ages 1 to 70 years 2.Diagnosis of primary immunodeficiency (CVID as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) XLA) 3.No prior immunoglobulin treatment 4.IgG level of <500 mg/dL at screening 5.Women of childbearing potential must use medically approved contraception and must have a negative urine pregnancy test at screening |
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E.4 | Principal exclusion criteria |
1.Evidence of serious infection between screening and Day 1 (day of first Vivaglobin infusion) 2.Bleeding disorders that require 4.Hypoalbuminemia, protein-losing enteropathies, and nephropathy with proteinuria 5.Lymphoprolipherative disease (i.e. lymphoma) 6.Evidence for bronchiectasis 7.Known allergic reaction to blood products 8.Pregnant or breast-feeding females or females planning to become pregnant during the course of the study. Women who have become pregnant during the course of the study have to be withdrawn 9.Participation in a study with an investigational product within 3 months prior to enrollment 10.Known or suspected HIV infection, acute hepatitis or clinically active chronic hepatitis 11.ASAT or ALAT concentration > 2.5 times the UNL Creatinine concentration > 1.5 times the UNL 12.Any condition that is likely to interfere with evaluation of the study drug or satisfactory conduct of the trial |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary objective of this trial is to show an increase in the total serum IgG trough level to >= 500 mg/dL on study Day 12. For that purpose the total serum IgG trough level will be measured on Day 12, i.e. 7 days after administration of the last loading dose of 100 mg/kg Vivaglobin on Day 5. The primary efficacy endpoint consists of the proportion of patients achieving IgG levels >=500 mg/dL on Day 12. |
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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 | Yes |
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 | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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) | Yes |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 0 |
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 |