E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) |
Kronisk inflammaotrisk demyeliniserende polyneuropati (CIDP) |
|
E.1.1.1 | Medical condition in easily understood language |
Chronic inflammatory neuropathy |
Kronisk inflammatorisk nervebetændelse |
|
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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10064135 |
E.1.2 | Term | Polyneuropathy chronic |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
|
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 |
To evaluate the effect of SCIG versus IVIG in de-novo CIDP patients during a treatment period of 26 weeks (phase I) |
|
E.2.2 | Secondary objectives of the trial |
To evaluate a standardized reduction of dosage regimen to identify the lowest effective dosage of immunoglobulin (phase II). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients diagnosed with typical or pure motor CIDP fulfilling the European Federation of Neurological Societies / Peripheral Nerve Society (EFNS/PNS) clinical and elctrophysiological criteria for definite or propable CIDP Age > 18 and <80 years at inclusion Overall disability sum score (ODSS) > 1 |
|
E.4 | Principal exclusion criteria |
Previous treatment with immmunoglobulin Pregnancy Malignancies Other causes of neuropathy (Diabetes Mellitus) Severe medical diseases Other immunomodulating treatment in the last 6 weeks prior to inclusion Hepatitis B and C or HIV Lactation |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change in Overall Disability Sum score (ODSS) measured by questionnaire from baseline until end of phase I (26 weeks)
Change in Overall Disability Sum score (ODSS) measured by questionnaire from start of phase II and until the lovest effective dosage of immunoglobulin has been reached (up til 60 weeks) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Phase I: All patients will be evaluated at baseline (week 0) and re-evaluated at week 2, 4, 14, 20 and 26
Phase II: All patients will be evaluated every 12th week according to lovering of the dosage of immunoglobulin
|
|
E.5.2 | Secondary end point(s) |
Change in parameters describing muscle strength and sensory: o Grip strength, MRC-score, isokinetic dynamometry, INCAT Sensory Sum Score (ISSS)
Change in parameters describing functional ability: o 10-meter-walk test (10-MWT), 6-spot-step test (6-SST), 9-hole-peg test (9-HPT)
Change in parameters describing disability, quality of life, pain and treatment satisfaction: QoL (EQ-5D-5L), Fatigue Severity Scale (FSS), Neuropathic Pain Symptom Inventory (NPSI), Rasch built overall disability scale (RODS) and Treatment Satisfaction Questionnaire for Medication (TSQM)
Serum samples: Plasma IgG (IgG1, IgG2, IgG3, IgG4)
Hematology: hemoglobin, reticulocyte count, haptoglobin, bilirubin, plasma haemoglobin, leukocyte count, thrombocyte count.
Fluctuations in describing parameter in each arm at pre-defined time points according to IVIG infusions (pre versus post IVIG): o Week 0, 4 and 20 versus week 2, 14 and 26
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Phase I: All patients will be evaluated at baseline (week 0) and re-evaluated at week 2, 4, 14, 20 and 26 At week 10 a telephone interview will be done to evaluate treatment response.
Phase II: All patients will be evaluated every 12th week according to tappering of the dosage of immunoglobulin |
|
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 | No |
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 | Yes |
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 | Yes |
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 | 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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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.8.9.1 | In the Member State concerned years | 6 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |