E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary Immune Deficiency
Secondary Immune Deficiency |
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E.1.1.1 | Medical condition in easily understood language |
Primary Immune Deficiency
Secondary Immune Deficiency |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
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 determine tolerability and safety of biweekly IgPro20 injection regimen
• To assess the pharmacokinetics of weekly and biweekly IgPro20 injections |
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E.2.2 | Secondary objectives of the trial |
• To determine the efficacy of biweekly IgPro20 injection regimen
• To evaluate the dose of biweekly IgPro20 injections needed for subjects switching from weekly IgPro20 therapy to biweekly therapy
• To assess the Quality of Life of patients on biweekly IgPro20 injections |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
A pharmacokinetic substudy will assess the pharmacokinetics of weekly and biweekly IgPro20 injections |
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E.3 | Principal inclusion criteria |
Full study:
• Female or male patients with primary or secondary immunodeficiency, aged from 5 to 64 years, receiving steady state immunoglobulin G replacement therapy.
Pharmacokinetic substudy:
• Female or male patients with primary immunodeficiency, aged between 12 and 64 years of age, on steady-state IgPro20 replacement therapy and willing to undergo pharmacokinetic sample collection on weekly and biweekly IgPro20 dosing regimens. |
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E.4 | Principal exclusion criteria |
• Patients with protein losing conditions such as: lymphangiectasis, nephrosis, status post cardio-thoracic surgery requiring drainage tubes for more than 48 hours, protein-losing enteropathy.
• Concomitant treatment with plasma or other blood products (including any IgGs other than IgPro20 [Hizentra]) within 21 days before study entry and/or during the study.
• Any other condition or treatment that, in the opinion of the investigator, would interfere with obtaining valid results for that subject.
• Women of childbearing potential with a positive pregnancy test.
• Active infection at the time of screening. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Annualized rate of adverse events (local and systemic)
2. Area under the concentration-time curve, AUC(0-t )
3. Maximal serum IgG concentration (Cmax)
4. Time to maximal serum IgG concentration (Tmax)
5. Trough serum IgG concentration (Ctrough)
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. During biweekly treatment period, up to approximately 52 weeks
2. and 5. Immediately before and after infusion, and at up to 6 time points between 1 and 14 days after infusion, for one infusion each during the weekly and biweekly treatment periods.
3. and 4. Immediately after infusion, and at up to 6 time points between 1 and 14 days after infusion, for one infusion each during the weekly and biweekly treatment periods. |
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E.5.2 | Secondary end point(s) |
1. Annualized rate of infections per patient
2. Serum IgG trough levels (Ctrough)
3. Health-related Quality of Life |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. and 3. During weekly and biweekly treatment periods, up to approximately 64 - 76 weeks
2. Immediately before the next infusion during the weekly treatment period and on week 24 and 48 of biweekly treatment.
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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 | Yes |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| Yes |
E.8.4 | Will this trial be conducted at multiple sites globally? | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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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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E.8.9 Initial estimate of the duration of the trial |
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 | 6 |