E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hypogammaglobulinaemia or agammaglobulinaemia |
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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 | 10060384 |
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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 | 10001471 |
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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 |
Evaluation of pharmacokinetics, efficacy, and safety of Kedrion 16% subcutaneous immunoglobulin preparation (Kedrion SCIg) |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients with hypogammaglobulinaemia or agammaglobulinaemia; 2. Male or female with age from 2 to less than 61 years; 3. Patients already on treatment with immunoglobulin preparation (intravenous or subcutaneous) for at least 6 months, with documented IgG trough levels > 500 mg/dl; 4. Written informed consent and authorization for treatment of personal data |
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E.4 | Principal exclusion criteria |
1. Patient not previously treated with immunoglobulin; 2. Patient is positive for HIV, HCV, or HBV; 3. Pregnant or breastfeeding women or women of childbearing potential not using adequate contraception; 4. Patient presents severe systemic diseases or associated conditions contraindicating the use of immunoglobulin (thrombocytopenia gravis, coagulation disorders, allergic reactions to Ig) or record of severe adverse reactions to blood or blood derivatives; 5. Acute bacterial infections that required treatment with intravenous antibiotics in the previous 1 week; 6. Treatment with immunosuppressive drugs in the previous 3 weeks; 7. Allogeneic bone marrow transplantation during the last year before inclusion; 8. Nephrotic syndrome characterized by proteinuria ≥ 3.5 g/24h, serum protein levels < 60 g/l or serum albumin levels < 30 g/l; 9. Chronic renal failure or creatinine clearance < 80 ml/min according to Cockroft formula; 10. History of heart failure (NYHA III/IV), cardiomyopathy, congestive heart failure, severe hypertension, cancer of lymphoid cells, hypoalbuminemia, protein-dispersive enteropathy; 11. Associated condition of chronic broncho-pneumopathy; 12. Presence of any conditions which, in the investigator’s judgment, may interfere with study objective evaluation; 13. Subject participated in another clinical study during the month prior to study start date (the subject received other study drugs during the last 30 days)or refused to sign the written informed consent required for participation |
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E.5 End points |
E.5.1 | Primary end point(s) |
Number of serious acute bacterial infections (bacteraemia or sepsis, bacterial meningitis, osteomyelitis, septic arthritis, bacterial pneumonia, visceral abscess). Limits of acceptability: 1 serious infection/patient/year |
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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 | Yes |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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.8.4.1 | Number of sites anticipated in Member State concerned | 8 |
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
| |
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |