E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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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 |
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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 |
The primary objective of this study is to determine the responder rate at higher infusion parameters of IgPro20 under the following conditions:
• Pump-Assisted: Volume per injection site of 25 mL, 40 mL, and 50 mL;
• Pump-Assisted: Flow rate per injection site of 25 mL/h, 50 mL/h, 75 mL/h and 100 mL/h;
• Manual Push (manual infusion using syringe without a pump): Flow rate per injection site of 30 mL/h, 60 mL/h, and 120 mL/h (0.5 mL/min, 1 mL/min, and 2 mL/min, correspondingly). |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are:
• To evaluate the safety of pump-assisted IgPro20 infusions at higher infusion parameters (volume and flow rate).
• To evaluate the safety of manual push IgPro20 infusions.
• To evaluate the tolerability of higher infusion parameters of IgPro20 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female on stable dose of IgPro20 (Hizentra) therapy. 2. Women of childbearing potential must be using and agree to continue using medically approved contraception (which must be discussed with the study doctor) and must have a negative pregnancy test at screening. 3. Subjects with PID, namely with a diagnosis of common variable immunodeficiency or X-linked agammaglobulinemia, as defined by the Pan American Group for Immune Deficiency and the European Society of Immune Deficiencies. 4. With infusion parameters as specified below:
Pump-Assisted Flow Rate Cohort subjects only
4a. Experience with pump-assisted infusions of IgPro20 at the tolerated flow rate of 25 mL/h per injection site for at least 1 month prior to study entry.
Pump-Assisted Volume Cohort subjects only
4b. Total weekly IgPro20 dose of ≥ 50 mL (≥ 10 g).
4c. Experience with pump-assisted infusions of IgPro20 at tolerated volumes of 25 mL/injection site for at least 1 month prior to study entry.
Manual Push Flow Rate Cohort subjects only
4d. Experience with frequent (5-7 times per week) infusions of IgPro20 at the tolerated flow rate of approximately 0.5 mL/min (equivalent of 25-30 mL/h) per injection site for at least 1 month prior to study entry. The dose (volume) per injection site should not exceed 25 mL.
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E.4 | Principal exclusion criteria |
1. Ongoing serious bacterial infections at the time of screening. 2. Other significant medical conditions that could increase the risk to the subject. 3. Females who are pregnant, breast feeding, or planning a pregnancy during the course of the study. 4. Participation in a study with an Investigational Medicinal Product (IMP) other than IgPro20 within three months prior to enrollment. |
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At the end of 4 weeks for each planned infusion volume/flow rate parameter level. |
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E.5.2 | Secondary end point(s) |
1. Rate of total adverse events (AEs)
2. Rate of local reactions
3. Time to onset of local reactions
4. Severity of local reactions
5. Duration of local reactions
6. Percentage of infusions experiencing no severe local reactions |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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) | 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 | 3 |
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 |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |