E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) |
|
E.1.1.1 | Medical condition in easily understood language |
Inflammatory disease of peripheral nervous system
|
|
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 | 19.0 |
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 |
|
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 the efficacy of 2 different doses of IgPro20 (0.2 g/kg bw and/or 0.4 g/kg bw) in the maintenance treatment of CIDP in comparison to placebo. |
|
E.2.2 | Secondary objectives of the trial |
- To investigate the efficacy of IgPro20 with additional clinical outcome measures in comparison to placebo.
- To investigate the safety and tolerability of IgPro20 in comparison to placebo.
- To investigate health-related quality of life (HRQL) following treatment with IgPro20.
- To investigate the safety and efficacy of IgPro10 re-stabilization therapy.
- To investigate the safety and efficacy of IgPro10 rescue therapy. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Definite or probable CIDP according to the EFNS/PNS criteria 2010.
2. Repeated treatment with IVIG (≥ 4 doses) within the last 9 months prior to enrollment.
3. An IVIG treatment during the last 8 weeks prior to enrollment.
4. Age ≥18 years.
5. Male or female.
6. Written informed consent for study participation obtained before undergoing any studyspecific
procedures. |
|
E.4 | Principal exclusion criteria |
1. Any polyneuropathy of other causes
2. Any other disease (mainly neurological or chronic orthopedic) that has caused neurological symptoms or may interfere with treatment or outcome assessments
3. Severe diseases and conditions that are likely to interfere with evaluation of the study product or satisfactory conduct of the study
4. History of thrombotic episodes within the 2 years prior to enrolment
5. Known allergic or other severe reactions to blood products including intolerability to previous IVIG |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy:
Percentage (%) of subjects who have a CIDP relapse during SC treatment or are withdrawn from the study during SC treatment for any reason. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
During SC treatment period - 24 weeks |
|
E.5.2 | Secondary end point(s) |
Efficacy:
• Changes in means at SC Treatment Period completion visit compared to baseline between groups in:
- INCAT score.
- Mean grip strength (dominant/non-dominant hand).
- MRC sum score.
- R-ODS.
• Difference in “time to CIDP relapse or withdrawal due to any other reason” using a Kaplan-Meier estimation comparing both IgPro20 groups with placebo as well as the 2 IgPro20 groups pairwise.
• Time to improvement on IgPro10 re-stabilization therapy (INCAT, R-ODS, Grip strength).
• Changes in means before and at the end of IgPro10 re-stabilization or rescue therapy in
- Mean grip strength (dominant/non-dominant hand).
- MRC sum score.
- R-ODS.
- INCAT disability score.
• Time to improvement after CIDP relapse in the SC Treatment Period and IgPro10 rescue therapy, defined as a decrease in INCAT score back to or below baseline.
Safety:
• For IgPro20: Rate of AEs per infusion, number and % of subjects with AEs during the SC Treatment Period
• For IgPro10: rate of AEs per infusion, number and % of subjects with AEs during IVIG Restabilization Period and during IVIG rescue therapy. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
During study - up to 52 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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Health-related quality of life (HRQL) |
|
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 70 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Austria |
Belgium |
Canada |
Czech Republic |
Finland |
France |
Germany |
Israel |
Italy |
Japan |
Netherlands |
Spain |
United Kingdom |
United States |
|
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |