To Assess Efficacy and Safety of Batoclimab in Adult Participants With Active CIDP
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy
- Interventions
- Registration Number
- NCT05581199
- Lead Sponsor
- Immunovant Sciences GmbH
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 277
Not provided
All Cohorts:
-
Have current or prior history of immunoglobulin M (IgM) paraproteinemia with or without anti-myelin-associated-glycoprotein antibodies.
-
Have Distal CIDP, Sensory CIDP or are suspected of having a diagnosis of auto-immune nodopathy in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP.
-
Have polyneuropathy of causes other than CIDP including but not limited to:
- Multifocal motor neuropathy
- Hereditary demyelinating neuropathy
- Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes (i.e., POEMS)
- Lumbosacral radiculoplexus neuropathy
- Systemic illnesses including vitamin deficiency syndromes and paraneoplastic neuropathies
- Drug- or toxin-induced
-
Have diabetes mellitus (DM) and meets any of the following criteria:
- Does not meet inclusion criteria 2(a) and 3(a).
- In the opinion of the Investigator, there is evidence of poorly controlled DM preceding the diagnosis of CIDP.
- In the opinion of the Investigator, there is evidence of poorly controlled DM at screening.
-
Have a history of myelopathy or evidence of central demyelination.
-
Are receiving chronic oral corticosteroids monotherapy at a dose > 40 mg/day prednisolone/prednisone or its equivalent at the Screening Visit.
-
Are receiving chronic oral corticosteroid at a dose > 10 mg/day prednisolone/prednisone or equivalent in combination with immunoglobulin therapy or PLEX at the Screening Visit.
Additional exclusion criteria are defined in the protocol.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description LTE Period: With Relapse in Period 2: Dose 1 and Dose 2 Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly Participants will receive Dose 1 for the initial 4 weeks only and Dose 2 for the remaining 48 weeks. Withdrawal Period 2: Cohort A, Dose 2 Batoclimab 340 mg SC weekly - Treatment Period 1: Cohort B, Dose 2 Batoclimab 340 mg SC weekly - Treatment Period 1: Cohort B, Dose 1 Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly - Treatment Period 1: Cohort C, Dose 1 Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly - Treatment Period 1: Cohort D, Dose 1 Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly - Treatment Period 1: Cohort A, Dose 2 Batoclimab 340 mg SC weekly - Treatment Period 1: Cohort A, Dose 1 Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly - Treatment Period 1: Cohort C, Dose 2 Batoclimab 340 mg SC weekly - Withdrawal Period 2: Cohort B, Placebo Placebo - Withdrawal Period 2: Cohort D, Placebo Placebo - Withdrawal Period 2: Cohort A, Placebo Placebo - LTE Period: Without Relapse in Period 2: Dose 2 Batoclimab 340 mg SC weekly Participants will receive Dose 2 for all 52 weeks. Treatment Period 1: Cohort D, Dose 2 Batoclimab 340 mg SC weekly - Withdrawal Period 2: Cohort D, Dose 2 Batoclimab 340 mg SC weekly - LTE Period: With Relapse in Period 2: Dose 1 and Dose 2 Batoclimab 340 mg SC weekly Participants will receive Dose 1 for the initial 4 weeks only and Dose 2 for the remaining 48 weeks. Withdrawal Period 2: Cohort C, Placebo Placebo - Withdrawal Period 2: Cohort B, Dose 2 Batoclimab 340 mg SC weekly - Withdrawal Period 2: Cohort C, Dose 2 Batoclimab 340 mg SC weekly -
- Primary Outcome Measures
Name Time Method Period 2, Cohort A: Proportion of participants who remain relapse-free at Week 36 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.
- Secondary Outcome Measures
Name Time Method Period 2, Cohort A: Change from Period 2 Baseline in Adj INCAT score Baseline (Week 12) and up to Week 36 Period 2, Cohort A: Time to first relapse relative to Period 2 Baseline Baseline (Week 12) to Week 36 Period 2, Cohorts A and B combined: Change from Period 2 Baseline in Mean Grip Strength 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) Baseline (Week 12) and up to Week 36 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.
Period 2, Cohorts A and B combined: Change from Period 2 Baseline in Adj INCAT score 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 Week 36 Period 2, Cohort A: Change from Period 2 Baseline in Medical Research Council (MRC) Sum Score Baseline (Week 12) and up to Week 36 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.
Period 2, Cohort A: Change from Period 2 Baseline in Mean grip strength Baseline (Week 12) and up to Week 36 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.
Period 2, Cohort A: Change from Period 2 Baseline in Overall Neuropathy Limitations Scale (ONLS) Baseline (Week 12) and up to Week 36 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.
Period 2, Cohorts A and B combined: Change from Period 2 Baseline in I-RODS Baseline (Week 12) and up to Week 36 Period 2, Cohorts A and B combined: Change from Period 2 Baseline in MRC sum score Baseline (Week 12) and up to Week 36 Period 2, Cohorts A and B combined: Change from Period 2 Baseline in ONLS Baseline (Week 12) and up to Week 36
Trial Locations
- Locations (116)
Site Number - 8403
🇷🇴Timişoara, Timis, Romania
SIte Number - 8400
🇷🇴Constanta, Romania
Site Number - 8501
🇷🇸Belgrade, Serbia
Site Number - 8502
🇷🇸Belgrade, Serbia
Site Number - 8500
🇷🇸Niš, Serbia
Site Number - 8503
🇷🇸Novi Sad, Serbia
Site Number - 8600
🇸🇰Liptovský Mikuláš, Slovakia
Site Number - 8601
🇸🇰Martin, Slovakia
Site Number - 8603
🇸🇰Prešov, Slovakia
Site Number - 8602
🇸🇰Trnava, Slovakia
Site Number - 3701
🇪🇸Hospitalet de Llobregat, Barcelona, Spain
Site Number - 3704
🇪🇸Sant Cugat Del Vallès, Barcelona, Spain
Site Number - 3700
🇪🇸San Sebastián, Gipuzkoa, Spain
Site Number - 3703
🇪🇸Barcelona, Spain
Site Number - 4891
🇸🇪Göteborg, Vastra Gotalands Lan, Sweden
Site Number - 7405
🇬🇧Cambridge, Cambridgeshire, United Kingdom
Site Number - 7402
🇬🇧Southampton, Hampshire, United Kingdom
Site Number - 7404
🇬🇧Glasgow, Lanarkshire, United Kingdom
Site Number - 7403
🇬🇧Preston, Lancashire, United Kingdom
Site Number - 7401
🇬🇧Sheffield, South Yorkshire, United Kingdom
Site Number - 7400
🇬🇧Manchester, United Kingdom
Site Number - 1634
🇺🇸Los Angeles, California, United States
Site Number - 1603
🇺🇸Scottsdale, Arizona, United States
Site Number - 1618
🇺🇸Carlsbad, California, United States
Site Number - 1619
🇺🇸Orange, California, United States
Site Number - 1608
🇺🇸San Francisco, California, United States
Site Number - 1625
🇺🇸Aurora, Colorado, United States
Site Number - 1636
🇺🇸Fort Collins, Colorado, United States
Site Number - 1621
🇺🇸New Haven, Connecticut, United States
Site Number - 1630
🇺🇸Washington, District of Columbia, United States
Site Number - 1600
🇺🇸Boca Raton, Florida, United States
Site Number - 1609
🇺🇸Jacksonville, Florida, United States
Site Number - 1631
🇺🇸Orlando, Florida, United States
Site Number - 1629
🇺🇸Orlando, Florida, United States
Site Number - 1617
🇺🇸Ormond Beach, Florida, United States
Site Number - 1620
🇺🇸Port Charlotte, Florida, United States
Site Number - 1633
🇺🇸Rockledge, Florida, United States
Site Number - 1604
🇺🇸Saint Petersburg, Florida, United States
Site Number - 1607
🇺🇸O'Fallon, Illinois, United States
Site Number - 1602
🇺🇸Kansas City, Kansas, United States
Site Number - 1611
🇺🇸Nicholasville, Kentucky, United States
Site Number - 1622
🇺🇸Brooklyn, New York, United States
Site Number - 1605
🇺🇸New York, New York, United States
Site Number - 1635
🇺🇸Chapel Hill, North Carolina, United States
Site Number - 1610
🇺🇸Charlotte, North Carolina, United States
Site Number - 1614
🇺🇸Portland, Oregon, United States
Site Number - 1623
🇺🇸Philadelphia, Pennsylvania, United States
Site Number - 1624
🇺🇸Philadelphia, Pennsylvania, United States
Site Number -1601
🇺🇸Austin, Texas, United States
Site Number - 1606
🇺🇸Houston, Texas, United States
Site Number - 1628
🇺🇸San Antonio, Texas, United States
Site Number - 1627
🇺🇸Richmond, Virginia, United States
Site Number - 1632
🇺🇸Seattle, Washington, United States
Site Number - 1613
🇺🇸Milwaukee, Wisconsin, United States
Site Number - 7753
🇦🇷Rosario, Santa Fe, Argentina
Site Number - 7751
🇦🇷Rosario, Santa Fe, Argentina
Site Number - 7752
🇦🇷Tucumán, Tucuman, Argentina
Site Number - 7750
🇦🇷Ciudad Autónoma de Buenos Aires, Argentina
Site Number - 4681
🇧🇪Gent, Oost-Vlaanderen, Belgium
Site Number - 4680
🇧🇪Leuven, Vlaams Brabant, Belgium
Site Number - 9103
🇧🇷BrasÃlia, Distrito Federal, Brazil
Site Number - 9101
🇧🇷Curitiba, Paraná, Brazil
Site Number - 9100
🇧🇷Ribeirão Preto, São Paulo, Brazil
Site Number - 9102
🇧🇷Rio De Janeiro, Brazil
Site Number - 9105
🇧🇷São Paulo, Brazil
Site Number - 9111
🇧🇬Sofia, Sofia-Grad, Bulgaria
Site Number - 9110
🇧🇬Sofia, Sofia-Grad, Bulgaria
Site Number - 9112
🇧🇬Pleven, Bulgaria
Site Number - 2600
🇨🇦Edmonton, Alberta, Canada
Site Number - 2603
🇨🇦Vancouver, British Columbia, Canada
Site Number - 4740
🇩🇰Copenhagen, Denmark
Site Number - 3241
🇫🇮Turku, Varsinais-Suomi, Finland
Site Number - 6704
🇩🇪München, Bayern, Germany
Site Number - 6705
🇩🇪Bochum, Nordrhein-Westfalen, Germany
Site Number - 6700
🇩🇪Dresden, Sachsen, Germany
Site Number - 6702
🇩🇪Leipzig, Sachsen, Germany
Site Number - 6706
🇩🇪Berlin, Germany
Site Number - 6341
🇬🇷Patras, Achaïa, Greece
Site Number - 6344
🇬🇷Athens, Attiki, Greece
Site Number - 6345
🇬🇷Athens, Attiki, Greece
Site Number - 6343
🇬🇷Alexandroupolis, Evros, Greece
Site Number - 6346
🇬🇷Ioánnina, Ioannina, Greece
Site Number - 6342
🇬🇷Heraklion, Irakleio, Greece
Site Number - 6347
🇬🇷Larisa, Greece
Site Number - 6302
🇮🇹Gussago, Brescia, Italy
Site Number - 6305
🇮🇹Bologna, Emilia-Romagna, Italy
Site Number - 6304
🇮🇹Udine, Friuli-Venezia Giulia, Italy
Site Number - 6309
🇮🇹Roma, Lazio, Italy
Site Number - 6306
🇮🇹Roma, Lazio, Italy
Site Number - 6301
🇮🇹Bergamo, Lombardia, Italy
Site Number - 6307
🇮🇹Milano, Lombardia, Italy
Site Number - 6303
🇮🇹Pisa, Toscana, Italy
Site Number - 6308
🇮🇹Siena, Toscana, Italy
Site Number - 6300
🇮🇹Pavia, Italy
Site Number - 9901
🇰🇷Seoul, Korea, Republic of
Site Number - 9900
🇰🇷Seoul, Korea, Republic of
Site Number - 6491
🇳🇴Oslo, Norway
Site Number - 3204
🇵🇱Wroclaw, Dolnoslaskie, Poland
Site Number - 3211
🇵🇱Bydgoszcz, Kujawsko-pomorskie, Poland
Site Number - 3210
🇵🇱Lublin, Lubelskie, Poland
Site Number - 3206
🇵🇱Lublin, Lubelskie, Poland
Site Number - 3202
🇵🇱Mazurki, Lubelskie, Poland
Site Number - 3209
🇵🇱Krakow, Malopolskie, Poland
Site Number - 3200
🇵🇱Krakow, Malopolskie, Poland
Site Number - 3208
🇵🇱Kraków, Malopolskie, Poland
Site Number - 3205
🇵🇱Gdańsk, Pomorskie, Poland
Site Number - 3201
🇵🇱Katowice, Slaskie, Poland
Site Number - 3207
🇵🇱Poznan, Wielkopolskie, Poland
Site Number - 3203
🇵🇱Bydgoszcz, Województwo Kujawsko-pomorskie, Poland
Site Number - 3742
🇵🇹Senhora Da Hora, Porto, Portugal
Site Number - 3745
🇵🇹Vila Nova De Gaia, Porto, Portugal
Site Number - 3743
🇵🇹Almada, Setubal, Portugal
Site Number - 3741
🇵🇹Lisboa, Portugal
Site Number - 3744
🇵🇹Porto, Portugal
Site Number - 8406
🇷🇴Bucharest, Bucuresti, Romania
Site Number - 8401
🇷🇴Targu Mures, Mures, Romania