Safety and Efficacy Study of Three Different Dosages of NewGam in Patients With CIDP

Study Overview
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Brief Summary
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NewGam (current working title for a new IGIV formulation) is a newly developed human normal immunoglobulin solution ready for intravenous administration (IGIV). This study will evaluate the safety and efficacy of three different dosages of NewGam 10% in patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy.

Detailed Description
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This is a Phase 2/3 study that will take place in 2 stages. The primary objective of Stage 1 (Phase 2 dose-finding part)is to determine and select one dosage from three NewGam maintenance dosage arms in comparison with a placebo arm, based on the percentage of responders (response defined as a decrease, meaning improvement, in the adjusted INCAT disability score by at least 1 point). The selected NewGam dosage and placebo will be employed and compared in Stage 2. The primary objective of Stage 2 (Phase 3 confirmatory part) is to demonstrate superiority of the maintenance dosage regimen selected at study Stage 1 over placebo in patients with CIDP as assessed by the percentage of responders. The secondary objective is to evaluate the safety (measured by number of adverse events)and efficacy of NewGam administration in patients with CIDP compared to baseline.

Official Title
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Double-blind, Placebo-controlled, Randomised, Multicentre, Adaptive, Two-stage Phase 2/3 Study Evaluating Safety and Efficacy of Three Dosages of NewGam in CIDP Patients

Conditions
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Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Intervention / Treatment
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* Drug: NewGam 10%
* Drug: Placebo
* Drug: NewGam 10%
* Drug: NewGam 10%


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients diagnosed as having CIDP based on fulfilment of clinical criteria of the INCAT Group and the definite electrophysiological criteria for CIDP ; patients with MADSAM or pure motor CIDP will be included provided they fulfil these criteria Worsening of disability and objective increase in weakness or sensory deficit during the 6 months prior to screening >=18 years of age Exclusion Criteria: Unifocal forms of CIDP Pure sensory CIDP MMN with conduction block Treatment of CIDP with immunoglobulins (intravenous or subcutaneous) at any time prior to study entry Steroids of any type equivalent to prednisolone or prednisone > 10 mg/day or equivalent plasma exchange (PE) during the last 3 months prior to baseline visit Treatment with cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil, interferon or other immunosuppressive or immunomodulatory drugs during the three months prior to baseline visit Clinical evidence of peripheral neuropathy from another Known diabetes mellitus Other serious medical condition complicating assessment or treatment Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever Known IgA deficiency with antibodies to IgA History of hypersensitivity, anaphylaxis or severe systemic response to immunoglobulin, blood or plasma derived products, or any component of NewGam Known blood hyperviscosity

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Dosage Arm 1<br>NewGam 10% 0.4 g/kg | Drug: NewGam 10%<br>* Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam . The maintenance doses to be infused 7 times will be 2.0 g/kg every 21 (+/-4) days.<br>|
| Experimental: Dosage Arm 2<br>NewGam 10% 1.0 g/kg | Drug: NewGam 10%<br>* Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 1.0 g/kg every 21 (+/-4) days.<br>|
| Experimental: Dosage Arm 3<br>NewGam 10% 2.0 g/kg | Drug: NewGam 10%<br>* Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 0.4 g/kg every 21 (+/-4) days.<br>|
| Placebo Comparator: Dosage Arm 4<br>Placebo 0.9% Saline | Drug: Placebo<br>* Loading dose at baseline (Week 0) in Placebo arm will be corresponding volume of an authorised 0.9% saline solution . The maintenance doses of the 0.9% saline solution to be infused 7 times will be given every 21 (+/-4) days.<br>* Other names: 0.9% saline solution;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adjusted INCAT disability score |  | Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Vital Signs |  | During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2) | 
| Grip Strength |  | Visit 9 & 13 | 
| Nerve Conduction Studies |  | Visti 9 & 13 | 
| Motor Impairment Assessment utlizing the Expanded MRC Sum Score | Expanded 'Medical Research Council sum score' will be measured as improvement in MRC units . | Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CIDP, IVIG