E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary Immune Deficiency
(Common Variable Immunodeficiency and X-linked agammaglobulinemia) |
|
E.1.1.1 | Medical condition in easily understood language |
Primary Immunodeficiency (PID) |
|
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 | 17.1 |
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 assess the safety and efficacy of IgPro10 in patients with PID, and to assess the tolerability of a high infusion rate. |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients with Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia (XLA) who:
Participated in the Phase III clinical study with intravenous IgPro10 (study number ZLB03_002CR) at 3- or 4- weekly intervals for 12 months (referred to as 'old' subjects)
OR
Were ≥ 6 years of age, were on other stable intravenous immunoglobulin therapy (200-800 mg IgG per kg body weight) at 3- or 4-week intervals for at least 6 months, AND were interested in participating in the Phase III clinical study with subcutaneous IgPro20 (study number ZLB04_009CR) (referred to as 'new' subjects)
- Written informed consent |
|
E.4 | Principal exclusion criteria |
- Diagnosis of epilepsia
- Insulin dependent diabetes
- Administration of steroids (daily ≥ 0.15 mg prednisone equivalent/kg/day) or other immunosuppressive drugs
- History of cardiac insufficiency (NYHA III/IV), cardiomyopathy, congestive heart failure, severe hypertension |
|
E.5 End points |
E.5.1 | Primary end point(s) |
1. The Proportion of Infusions With One or More Temporally-associated Adverse Events (AEs)
2. Influence of Infusion Rate on Temporally-Associated AEs
3. Rate of AEs by Severity and Relationship
4. Number of Subjects With Clinically Significant Changes in Vital Signs |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. During each infusion, and within 48 or 72 hours after the end of each infusion
2. Within 72 hours after each infusion
3. For the duration of the study, up to approximately 29 months
4. Before, during, and after each infusion |
|
E.5.2 | Secondary end point(s) |
1. Annualized Rate of Acute Serious Bacterial Infections
2. Number of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Illness
3. Number of Days of Hospitalization
4. Annualized Rate of Any Infection
5. Trough Levels of Total Immunoglobulin (IgG) Serum Concentrations |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1 - 4. For the duration of the study, up to approximately 29 months
5. Prior to each infusion; every 3 or 4 weeks depending upon the dosing schedule |
|
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 | 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 | 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.8.3 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial months | 5 |