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 |
To assess the clinical and biological safety of IGNG 5% when administered in patients with primary immunodeficiency at a progressively increased infusion flow rate, up to 8 ml/kg/h. |
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E.2.2 | Secondary objectives of the trial |
•To evaluate the clinical and biological efficacy of IGNG •To evaluate the time saved by the increase of infusion flow rate
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Patient already included in study IGNG-0629 and who still meets all the IGNG-0629 inclusion criteria reported here below : a.Informed consent forms signed and dated for study IGNG-0629 and for serum reference sample. b.Patient with primary immunodeficiency (eg X-linked agammaglobulinemia, common variable immunodeficiency, hyper IgM syndrome) c.Need to have an immunoglobulin replacement therapy. d.Age from 12 to 75 years old. e.For women of childbearing potential, a negative pregnancy test before inclusion and a medically-acceptable method of birth control throughout the study are required f.Patient covered by healthcare insurance in accordance with local requirements 2.Having received the specific information and signed the informed consent form for this new protocol. 3.Having undergone the last 4 IGNG infusions at a flow rate of 4 m/kg/h and without any IGNG related adverse event.
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E.4 | Principal exclusion criteria |
1. Patient who repeatedly requires a premedication with antihistamines, corticosteroids, or antipyretics before the IGNG infusion. 2. Patient who meet one of the IGNG-0629 exclusion criteria listed below: a. Known allergy or serious adverse reaction to any IVIG b. Known allergy to mannitol, glycine or polysorbate 80 c. Chronic renal insufficiency or serum creatinine level > 120 µmol/l in adults or creatinine clearance < 80 ml/min/1,73m2 (calculated with Schwartz formula) in adolescent patients d. Protein-loosing enteropathy characterised by serum protein level < 60 g/l and serum albumin level < 30 g/l e.Nephrotic syndrome characterised by proteinuria 3.5 g/24 hours, serum protein level < 60 g/l and serum albumin level < 30 g/l f. Isolated deficiency of a IgG subclass with a normal total serum IgG level g. Having IgA deficiency, and anti-IgA antibodies have been detected h. Allogeneic haematopoeitic stem cells transplantation within the last year before infusion i. Severe or non-controlled cardiac disease (New York Heart Association stage III and IV) j. Long-term immunosuppressive treatments (corticosteroids included) k. Use of loop diuretics (furosemide, bumetanide, piretanide) l. Pregnancy or breastfeeding m. Participation in another clinical study within 3 weeks prior to the start of study treatment, except in a previous IGNG study n. Patients whose use of concomitant medication may interfere with the interpretation of data o. Anticipated poor compliance of patient with study procedures
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E.5 End points |
E.5.1 | Primary end point(s) |
•Number of infusions administered and proportion of those with one or more IMP related adverse events, by highest flow rate reached during the infusion. |
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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 | No |
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 | Information not present in EudraCT |
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 | 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 | 1 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 15 |