E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic Inflammatory Demyelinating Polyradiculoneuropathy |
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E.1.1.1 | Medical condition in easily understood language |
Chronic Inflammatory Demyelinating Polyradiculoneuropathy |
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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 | 15.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10057645 |
E.1.2 | Term | Chronic inflammatory demyelinating polyradiculoneuropathy |
E.1.2 | System Organ Class | 10029205 - Nervous system 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 |
Primary objective:
The primary objective is to assess the efficacy of ClairYg® in controlling the neurological status of patients with CIDP.
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E.2.2 | Secondary objectives of the trial |
Secondary objective:
The secondary objective is to assess the safety of ClairYg® in patients with CIDP.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Male or female patient aged 18 years or more.
2.Patient diagnosed as having CIDP by an experienced neurologist, after other causes of chronic neuropathy have been ruled out.
3.Patient who meets the diagnosis criteria for definite or probable CIDP proposed by European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) 2010.
4.Patient whose CIDP has already induced a disability, scored at least 2 on the Adjusted INCAT disability scale, even if this disability is controlled by the IVIG treatment at the time of the inclusion.
5.Patient on maintenance treatment with IVIG, i.e., receiving IVIG since at least 6 months and for whom the minimal efficient treatment schedule has been ascertained.
6.Patient for whom the current treatment schedule is within the following range:
Dose per course within 0.4 to 2 g/kg
Course frequency within every 2 to 8 weeks.
7.If female and capable of bearing children, negative pregnancy test at enrollment and agreement to use adequate birth control measures for the duration of the study.
8.Having signed a written informed consent prior to any study-related procedures.
9.Covered by national healthcare insurance in accordance with French regulation. |
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E.4 | Principal exclusion criteria |
1.History of severe allergy or serious adverse reaction to any IVIG.
2.Immunoglobulin A (IgA) deficiency with an IgA ponderal level <0.2g/l or known anti-IgA antibodies.
3.Known hypersensitivity to any of the excipients of ClairYg® (mannitol, glycine and polysorbate 80) or Tégéline® (saccharose and sodium chloride).
4.History of cerebral ischemia, cardiac ischemia, cardiac insufficiency, stroke, deep vein thrombosis or pulmonary embolism.
5.Chronic renal insufficiency or serum creatinine level >120µmol/l.
6.Use of loop diuretics (eg furosemide, bumetanide, piretanide).
7.Progressive hepatic disease that could worsen during the study.
8.Participation in another interventional clinical study within 3 weeks before inclusion (participation in an observational research program may be allowed provided that such program does not require the administration of a specific investigational medicinal product).
9.Pregnant or breastfeeding woman or woman of childbearing potential with no adequate means of contraception.
10.Any serious medical conditions that would prevent the patient from complying with the protocol requirements or interfere with the assessment criteria. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy assessment:
Primary efficacy endpoint:
Proportion of patients with no relapse i.e. whose Adjusted INCAT disability score remains at the same level or improves during the 6-month follow-up.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Just before each course during the 6 months follow-up. |
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E.5.2 | Secondary end point(s) |
Secondary efficacy endpoints:
Mean changes in the following scores:
•INCAT disability score
•ISS
•MRC-sumscore
•Grip strength with the Martin Vigorimeter in the dominant hand.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
For patients who have maintained their dose of IVIG stable during the 6-month follow-up, the change considered will be the one occurring between the baseline and the end of the 6-month follow-up.
For patients who have had their dose of IVIG increased during the study, the change considered will be the one occurring between the baseline and the worst evaluation of the 6-month follow-up period.
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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) | 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) | 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 | 10 |
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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 2 |