This is a multi-center, randomized, quadruple-blind, placebo-controlled study to evaluate the efficacy and safety of batoclimab in adult participants with active CIDP. The study includes an up to 4-week Screening Period, an up to 12-week Washout Period, a 12-week Randomized Treatment Period (Period 1), an up to 24-week Randomized Withdrawal Period (Period 2), an up to 52-week Long-term Extension (LTE) Period (optional), and Safety Follow-up 4 weeks after the last dose of study treatment. The total study duration will be up to approximately 109 weeks. Eligible participants will be assigned to one of four cohorts based upon their baseline CIDP treatment (Cohorts A and D - immunoglobulin \[Ig\] or plasma exchange \[PLEX\]; Cohort B - corticosteroids; Cohort C - naive or untreated in previous 3-24 months) and whether they meet diagnosis according to the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) criteria (Cohorts A, B, and C) or clinical criteria only (Cohort D) at the time of screening.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
94
Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody
Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody
Matching placebo SC
Site Number - 1603
Scottsdale, Arizona, United States
Site Number - 1634
Los Angeles, California, United States
Site Number - 1625
Aurora, Colorado, United States
Site Number - 1621
New Haven, Connecticut, United States
Site Number - 1629
Orlando, Florida, United States
Site Number - 1617
Period 2, Cohort A: Proportion of participants who remain relapse-free at Week 36
Relapse is defined as a worsening (increase) of \>=1 point on adjusted inflammatory neuropathy cause and treatment (AdjINCAT) score at any time point during Period 2 relative to Period 2 Baseline which is sustained at a Follow-Up visit 1 week later. The INCAT disability scale is a widely used and validated efficacy assessment of neurologic dysfunction in CIDP. Upper and lower limb dysfunction are each separately assessed on a scale of 0-5 and results are summed together for a total composite score of 0-10. Higher scores represent greater disability. The Adj INCAT disability score is identical to INCAT disability score with exception that changes in upper limb function from 0 (normal) to 1 (minor symptoms) and vice versa are excluded since minor symptoms in the fingers, which are implied by an upper limb score of 1, are not considered clinically significant in all participants. The Adj INCAT disability score will be used for participant selection and measurement of clinical response.
Time frame: Week 36
Period 2, Cohort A: Time to first relapse relative to Period 2 Baseline
Time frame: Baseline (Week 12) to Week 36
Period 2, Cohort A: Change from Period 2 Baseline in Adj INCAT score
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohort A: Change from Period 2 Baseline in Inflammatory Rasch-built Overall Disability Scale (I-RODS)
The I-RODS for immune-mediated peripheral neuropathies is a patient-based linearly weighted scale that captures activity and social participation limitations in patients with CIDP. The assessment consists of a 24-question instrument that addresses upper and lower limb tasks that range in difficulty from reading a book and eating to standing and running. Answers are scored on a scale of 0-2 (complete disability to no disability) and the raw scores are then transformed into a final score ranging from 0-100.
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohort A: Change from Period 2 Baseline in Mean grip strength
Mean Grip strength provides an objective, quantitative and immediate assessment of strength impairment. The Jamar dynamometer and the Martin vigorimeter are both commonly used to assess mean grip strength.
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohort A: Change from Period 2 Baseline in Medical Research Council (MRC) Sum Score
The MRC sum score is a standardized methodology for objectively assessing and reporting muscle function. Six muscle groups are assessed bilaterally, and each scored on a scale of 0 (no visible contraction) to 5 (normal) yielding a sum ranging from 0 (paralysis) to 60 (normal strength). Higher scores indicate normal muscle strength.
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohort A: Change from Period 2 Baseline in Overall Neuropathy Limitations Scale (ONLS)
The ONLS is a scale that focuses on upper and lower limb functions and was designed to assess the limitations of participants with immune-mediated peripheral neuropathies. This scale is completed by adding the total of the arm grade from zero point (less limitation) to 5 points (most limitation) and leg grade from zero point (less limitation) to 7 points (more limitation) yielding a total score of 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condition.
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohorts A and B combined: Change from Period 2 Baseline in Adj INCAT score
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohorts A and B combined: Change from Period 2 Baseline in I-RODS
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohorts A and B combined: Change from Period 2 Baseline in Mean Grip Strength
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohorts A and B combined: Change from Period 2 Baseline in MRC sum score
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohorts A and B combined: Change from Period 2 Baseline in ONLS
Time frame: Baseline (Week 12) and up to Week 36
Period 2, Cohorts A, B, and C: Proportion of participants who remain relapse-free at Week 36
Time frame: Week 36
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Ormond Beach, Florida, United States
Site Number - 1620
Port Charlotte, Florida, United States
Site Number - 1633
Rockledge, Florida, United States
Site Number - 1604
St. Petersburg, Florida, United States
Site Number - 1602
Kansas City, Kansas, United States
...and 88 more locations