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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
2
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.
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.
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.
Adjusted INCAT disability score
Time frame: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Vital Signs
Time frame: During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Grip Strength
Time frame: Visit 9 & 13
Nerve Conduction Studies
Time frame: 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 .
Time frame: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
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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.