E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064859 |
E.1.2 | Term | Primary immunodeficiency syndrome |
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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 primary objective of the study is to assess the clinical and biological safety of IGNG by considering adverse events in patients treated as per current practice over a 2-year-period for primary immunodeficiency. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are: - to evaluate clinical and biological efficacy of IGNG - to describe IGNG safety with clinical and biological parameters - to describe IGNG tolerability by the use of premedication before infusion.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Signed and dated informed consent form 2. Have a primary immunodeficiency (e.g. X-linked agammaglobulinemia ; common variable immunodeficiency ; hyper IgM syndrome) 3. Need to have an immunoglobulin replacement therapy 4. Age from 12 to 75 years old 5. For women of childbearing potential, a negative pregnancy test before inclusion and a medically-acceptable method of birth control throughout the study are required 6. Covered by healthcare insurance in accordance with local requirements |
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E.4 | Principal exclusion criteria |
1. Known allergy or serious adverse reaction to any IVIG 2. Known allergy to mannitol, glycine or polysorbate 80 3. Chronic renal insufficiency or serum creatinine level > 120µmol/l 4. Protein-loosing enteropathy characterised by serum protein level < 60 g/l and serum albumin level < 30 g/l 5. Nephrotic syndrome characterised by proteinuria ≥ 3.5 g/24 hours, serum protein level < 60 g/l and serum albumin level < 30 g/l 6. Isolated deficiency of a IgG subclass with a normal total serum IgG level 7. Having IgA deficiency, and anti-IgA antibodies have been detected 8. Allogeneic haematopoeitic stem cells transplantation within the last year before infusion 9. Severe or non-controlled cardiac disease (New York Heart Association stage III and IV) 10. Long-term immunosuppressive treatment (corticosteroids included) 11. Use of loop diuretics 12. Pregnancy or breastfeeding 13. Participation in another clinical study within 3 weeks prior to the start of study treatment, except in a previous IGNG study 14. Patients whose use of concomitant medication may interfere with the interpretation of data 15. Anticipated poor compliance of patient with study procedures |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Adverse event frequency, nature, duration and time of onset during and after IGNG administration, occurring during the study, overall and by subgroups: - Infusional AEs (ie, an AE temporally associated with an infusion) - Others AEs 2. Proportion of infusions with at least one infusional AE |
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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 | 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.8.3 |
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 | 10 |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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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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 | 6 |
E.8.9.1 | In the Member State concerned days | |