E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
PID (Primary Immune Deficiency) |
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E.1.1.1 | Medical condition in easily understood language |
Disability to make normal amounts of antibodies (Primary Immune Deficiency). |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10064859 |
E.1.2 | Term | Primary immunodeficiency syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 objective of this study is to assess the long-term efficacy, tolerability, and safety of IgPro20 in subjects with PID. |
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E.2.2 | Secondary objectives of the trial |
- maintained health related quality of life - subject's satisfaction with subcutaneous treatment - subject's utility score |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Subjects with primary humoral immunodeficiency, namely with a diagnosis of Common variable immunodeficiency (CVID) as defined by the Pan-American Group for Immunodeficiency (PAGID) and the European Society for Immunodeficiencies (ESID) or X-linked agammaglobulinemia (XLA) as defined by PAGID and ESID or autosomal recessive agammaglobulinemia who have participated in the study ZLB06_001CR and who have tolerated IgPro20 well • Written informed consent
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E.4 | Principal exclusion criteria |
• Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined as total urine protein concentration > 0.2g/L) • Other significant medical conditions that could increase the risk to the subject • Females who are pregnant, breast feeding or planning a pregnancy during the course of the study • Participation in a study with an investigational medicinal product within three months prior to enrollment, except for ZLB06_001CR • Evidence of uncooperative attitude • Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study • Subjects who are employees at the investigational site, relatives or spouse of the investigator |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Total serum IgG trough levels. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
- At visit 1, 3, 5, 7, 9, 11 and 13. |
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E.5.2 | Secondary end point(s) |
- Rate of clinically documented serious bacterial infections (SBIs). - Number of infection episodes. - Number of days out of work/school/kindergarten/day care or unable to perform normal daily activities due to infections. - Number of days of hospitalization due to infections. - Use of antibiotics for infection prophylaxis and treatment. - Rate, serverity and relatedness of any AE per infusion and subject. - Changes in vital signs. - Changes in routine laboratory parameters. - Health related Quality of Life Questionnaires. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- At visit 1, 3, 5, 7, 9, 11 and 13. |
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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 | No |
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) | 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 | Information not present in EudraCT |
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 | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
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 | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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 |
France |
Germany |
Poland |
Romania |
Spain |
Sweden |
Switzerland |
United Kingdom |
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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 the study is defined as the last visit of the last subject participating in the study. |
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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 | 36 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 39 |
E.8.9.2 | In all countries concerned by the trial days | 0 |