Study to evaluate the Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy
Prospective, Double-blind, Randomized, Multicenter Phase III Study Evaluating Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy ("ProCID trial")
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
142
In the Dose-evaluation Phase, all patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5, 1.0 or 2.0 g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). If a patient is randomized to receive the low or medium NewGam dose, the same volume with the same infusion rate as would have been applied in case the patient would have been randomized to 2.0 g/kg NewGam will be used, thus supplemented with an authorized 0.9% w/v isotonic sodium chloride solution as appropriate and detailed in the following infusion bag split to maintain the blinding
MHAT Puls EOOD
Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
Efficacy - Proportion of responders in the 1.0 g/kg NewGam arm at Week 24 (Termination Visit) relative to baseline (Week 0). A responder being defined as a patient with a decrease of at least 1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score (a scale from 0 to 10, from healthy to unable to make any purposeful movements with arms and/or legs)
Time frame: at Week 24
Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline compared to the 1.0 g/kg arm, based on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
Time frame: at Week 24
Grip Strength Score
Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the grip strength (Martin Vigorimeter) using the previously published minimum clinically important difference (MCID) cut-off of 8 kilopascal (kPa)
Time frame: at Week 24
Inflammatory Rasch-built Overall Disability Scale (I-RODS Score)
Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the Inflammatory Rasch-built overall disability scale (I-RODS Score) using the MCID concept related to the varying standard errors (MCID-SE) as recently demonstrated
Time frame: at Week 24
Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
Time to first confirmed worsening on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale by at least 1 point from the value at baseline (Week 0)
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Blagoevgrad, Bulgaria
St. Naum Hospital
Sofia, Bulgaria
Toronto General Hospital
Toronto, Ontario, Canada
Octapharma Research Site
Montreal, Canada
Outpatient Clinic of Neurology
Hradec Králové, Czechia
Regional Hospital Pardubice
Pardubice, Czechia
Thomayer Faculty Hospital
Prague, Czechia
University Medical Center Goettigen
Goettigen, Germany
Jahn Ferenc Del Pesti Korhaz
Budapest, Hungary
Szegedi Tudományegyetem ÁOK Neurológiai Klinika
Szeged, Hungary
...and 16 more locations
Time frame: Week 24
Mean Change in Grip Strength
Mean change from baseline (Week 0) to Termination Visit in grip strength of both hands (assessed by Martin vigorimeter). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
Time frame: Up to 24 weeks
Inflammatory Rasch-built Overall Disability Scale (I-RODS)
Mean change from baseline (Week 0) to Termination Visit in Inflammatory Rasch-built overall disability sum score (I-RODS using the concept of MCID-SE as recently reported) and number of improvers. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome.
Time frame: Up to 24 weeks
Motor Nerves
Mean change from baseline (Week 0) to Termination Visit in sum of the distal evoked amplitude of 4 right sided and 4 left sided motor nerves (peroneal, tibial, ulnar and median). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
Time frame: Up to 24 weeks
Mean Change in Pain Intensity Numerical Rating Scale (PI-NRS Scale)
Mean change from baseline (Week 0) to Termination Visit in Pain Intensity Numeric Rating Scale (PI-NRS). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. The Pain Intensity Numeric Rating Scale (PI-NRS, a numeric scale where 0 = no pain and 10 = worst possible pain) is an 11-point scale for patient self-reporting of pain. A higher value represents a worse outcome.
Time frame: Up to 24 weeks
Worsening on the Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)
Time to first confirmed worsening on the I-RODS scale. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome. Worsening is determined using the concept of MCID (minimum clinically important difference) using the individually obtained standard errors (MCID-SE).
Time frame: 24 weeks
1 Point Decrease in the INCAT Disability Score
Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
Time frame: 24 weeks
Decrease in Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)
Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores
Time frame: 24 weeks