E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
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 | 19.1 |
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 |
|
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 characterize the PK of IgG following intravenous IgPro10 dosing in Japanese PID subjects after a standard wash-in/wash-out period of 12 weeks. |
|
E.2.2 | Secondary objectives of the trial |
To collect safety information about the use of IgPro10 in Japanese PID subjects. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female Japanese subject with a diagnosis of PID. 2. Aged ≥ 6 years with body weight ≥ 19 kg at the time of providing written informed consent/minor assent. 3. Previously receiving stable doses of any intravenous immunoglobulin (IVIG) product currently approved in Japan for at least 6 months prior to study entry at regular 3- or 4-weekly intervals. 4. At least 1 historic IgG trough level of ≥ 5 g/L during the past 6 months prior to study entry (can be obtained at Screening). |
|
E.4 | Principal exclusion criteria |
1. Newly diagnosed PID. 2. Ongoing active serious infection at the time of Screening (e.g., pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess). 3. Ongoing or history of concomitant malignancies of lymphoid cells such as lymphocytic leukemia, non-Hodgkin's lymphoma and immunodeficiency with lymphoma. 4. Known hyperprolinemia, hypoalbuminemia, protein-losing enteropathies, and any proteinuria. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
1. Minimum concentration (Cmin) of IgG following intravenous IgPro10 dosing
2. Maximum concentration (Cmax) of IgG following intravenous IgPro10 dosing
3. Time to reach maximum concentration (Tmax) of IgG following intravenous IgPro10 dosing
4. Area under the concentration-time curve from time zero to the last sample (AUC0-last) following intravenous IgPro10 dosing
5. Total body clearance (CL) of IgG following intravenous IgPro10 dosing |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
1-5. Before infusion on Day 85 and up to approximately 21 days (for 3 week cycle) and up to approximately 28 days (for 4 week cycle) after infusion |
|
E.5.2 | Secondary end point(s) |
Percentage of subjects with adverse events (AEs) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 4 months after first infusion of IgPro10 |
|
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 | No |
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) | 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 | 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?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
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 |
|
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.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 12 |
E.8.9.2 | In all countries concerned by the trial days | 0 |