E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pediatric Chronic inflammatory demyelinating polyneuropathy (CIDP)
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E.1.1.1 | Medical condition in easily understood language |
Chronic inflammatory demyelinating polyneuropathy (CIDP) is an acquired neurological, demyelinating neuropathy with an assumed autoimmune-mediated pathogenesis.
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061811 |
E.1.2 | Term | Demyelinating polyneuropathy |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 |
The primary objective of this study is to investigate the efficacy of single and multiple dose Privigen administration in IVIG-pretreated and IVIG-untreated pediatric subjects with CIDP.
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E.2.2 | Secondary objectives of the trial |
The secondary objective of the study is to investigate the safety and efficacy of Privigen in pediatric subjects with CIDP.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female subjects 2 to < 17 years of age with confirmed or possible CIDP
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E.4 | Principal exclusion criteria |
1. Absence of CIDP symptoms
2. History or family history of inherited neuropathy
3. Diagnosed developmental delay or regression
4. History of thrombotic episode
5. Known or suspected hypersensitivity to Privigen
6. Known allergic or other severe reactions to blood products
7. Female subject of childbearing potential either not using or not willing to use a medically reliable method of contraception or not sexually abstinent during the study
8. Pregnant or breastfeeding mother
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Percentage (%) of subjects with CIDP relapse in the Randomized Phase
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1. Percentage of subjects with TEAEs
2. Rate of TEAEs per infusion
3. Rate of mild, moderate, and severe TEAEs per infusion
4. Percentage of subjects with serious TEAEs
5. Rate of serious TEAEs per infusion
6. Percentage of subjects with related TEAEs
7. Rate of related TEAEs per infusion
8. Change in mRS from baseline in the Randomized Phase
9. Percentage of subjects with CIDP relapse in the Dose Exploration Phase
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1-7. Up to 52 weeks
8-9. Up to 24 weeks
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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 | 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 | Yes |
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?
| 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 | 5 |
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 |