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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Period 1: Cohort A, Dose 1 | Experimental |
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| Treatment Period 1: Cohort A, Dose 2 | Experimental |
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| Treatment Period 1: Cohort B, Dose 1 | Experimental |
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| Treatment Period 1: Cohort B, Dose 2 | Experimental |
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| Treatment Period 1: Cohort C, Dose 1 | Experimental |
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| Treatment Period 1: Cohort C, Dose 2 | Experimental |
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| Treatment Period 1: Cohort D, Dose 1 | Experimental |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly | Drug | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Week 36 |
| Measure | Description | Time Frame |
|---|---|---|
| Period 2, Cohort A: Time to first relapse relative to Period 2 Baseline | Baseline (Week 12) to Week 36 | |
| Period 2, Cohort A: Change from Period 2 Baseline in Adj INCAT score | Baseline (Week 12) and up to Week 36 |
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Inclusion criteria:
All Cohorts:
Are >= 18 years at the Screening Visit.
Have met clinical diagnostic criteria for typical CIDP, or one of the following CIDP variants: multifocal CIDP, focal CIDP, or motor CIDP in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP. Clinical criteria for typical CIDP and variants are as follows (either criterion must be met):
Typical CIDP: All the following:
CIDP variants: One of the following, but otherwise as in typical CIDP (tendon reflexes may be normal in unaffected limbs):
Cohorts A and B:
Have electrodiagnostic test results supporting the diagnosis of CIDP in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP; for Cohorts A and B, either criterion must be met:
Motor nerve conduction criteria strongly supportive of demyelination.
Motor nerve conduction criteria weakly supportive of demyelination and 2 or more of the following additional diagnostic criteria:
Cohort C only:
Have a diagnosis of CIDP in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP based on clinical criteria and motor nerve conduction criteria strongly supportive of demyelination (i.e., motor nerve conduction criteria weakly supportive of demyelination is insufficient diagnostic evidence for admission to Cohort C).
Cohort D only:
Have met only clinical diagnostic criteria for typical CIDP, or one of the following CIDP variants: multifocal CIDP, focal CIDP, or motor CIDP in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP. Either inclusion criterion 2(a) or 2(b) must be met.
Additional inclusion criteria are defined in the protocol.
Exclusion Criteria:
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:
Have diabetes mellitus (DM) and meets any of the following criteria:
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.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Site Number - 1603 | Scottsdale | Arizona | 85028 | United States | ||
| Site Number - 1618 |
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|
| Treatment Period 1: Cohort D, Dose 2 | Experimental |
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| Withdrawal Period 2: Cohort A, Dose 2 | Experimental |
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| Withdrawal Period 2: Cohort A, Placebo | Experimental |
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| Withdrawal Period 2: Cohort B, Dose 2 | Experimental |
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| Withdrawal Period 2: Cohort B, Placebo | Experimental |
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| Withdrawal Period 2: Cohort C, Dose 2 | Experimental |
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| Withdrawal Period 2: Cohort C, Placebo | Experimental |
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| Withdrawal Period 2: Cohort D, Dose 2 | Experimental |
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| Withdrawal Period 2: Cohort D, Placebo | Experimental |
|
| LTE Period: With Relapse in Period 2: Dose 1 and Dose 2 | Experimental | Participants will receive Dose 1 for the initial 4 weeks only and Dose 2 for the remaining 48 weeks. |
|
| LTE Period: Without Relapse in Period 2: Dose 2 | Experimental | Participants will receive Dose 2 for all 52 weeks. |
|
|
| Batoclimab 340 mg SC weekly | Drug | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
|
|
| Placebo | Drug | Matching placebo SC |
|
| 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. | 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. | 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. | 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. | Baseline (Week 12) and up to Week 36 |
| 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 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 Mean Grip Strength | 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 |
| Period 2, Cohorts A, B, and C: Proportion of participants who remain relapse-free at Week 36 | Week 36 |
| Carlsbad |
| California |
| 92011 |
| United States |
| Site Number - 1634 | Los Angeles | California | 90033-5330 | United States |
| Site Number - 1619 | Orange | California | 92868 | United States |
| Site Number - 1608 | San Francisco | California | 94109 | United States |
| Site Number - 1625 | Aurora | Colorado | 80045 | United States |
| Site Number - 1636 | Fort Collins | Colorado | 80528 | United States |
| Site Number - 1621 | New Haven | Connecticut | 06519 | United States |
| Site Number - 1630 | Washington D.C. | District of Columbia | 20010 | United States |
| Site Number - 1600 | Boca Raton | Florida | 33487 | United States |
| Site Number - 1609 | Jacksonville | Florida | 32224 | United States |
| Site Number - 1631 | Orlando | Florida | 32804 | United States |
| Site Number - 1629 | Orlando | Florida | 32806-5411 | United States |
| Site Number - 1617 | Ormond Beach | Florida | 32174 | United States |
| Site Number - 1620 | Port Charlotte | Florida | 33952 | United States |
| Site Number - 1633 | Rockledge | Florida | 32955 | United States |
| Site Number - 1604 | St. Petersburg | Florida | 33713 | United States |
| Site Number - 1607 | O'Fallon | Illinois | 62269 | United States |
| Site Number - 1602 | Kansas City | Kansas | 66160 | United States |
| Site Number - 1611 | Nicholasville | Kentucky | 40356 | United States |
| Site Number - 1622 | Brooklyn | New York | 11220 | United States |
| Site Number - 1605 | New York | New York | 10032 | United States |
| Site Number - 1635 | Chapel Hill | North Carolina | 27559 | United States |
| Site Number - 1610 | Charlotte | North Carolina | 28207 | United States |
| Site Number - 1614 | Portland | Oregon | 97239 | United States |
| Site Number - 1623 | Philadelphia | Pennsylvania | 19104 | United States |
| Site Number - 1624 | Philadelphia | Pennsylvania | 19104 | United States |
| Site Number -1601 | Austin | Texas | 78759 | United States |
| Site Number - 1606 | Houston | Texas | 77030 | United States |
| Site Number - 1628 | San Antonio | Texas | 78229 | United States |
| Site Number - 1627 | Richmond | Virginia | 23298 | United States |
| Site Number - 1632 | Seattle | Washington | 98195-0001 | United States |
| Site Number - 1613 | Milwaukee | Wisconsin | 53226-3548 | United States |
| Site Number - 7753 | Rosario | Santa Fe Province | S2000BZL | Argentina |
| Site Number - 7751 | Rosario | Santa Fe Province | S2000DTP | Argentina |
| Site Number - 7752 | San Miguel de Tucumán | Tucumán Province | T4000AXL | Argentina |
| Site Number - 7750 | Buenos Aires | C1199ABB | Argentina |
| Site Number - 4681 | Ghent | Oost-Vlaanderen | 09000 | Belgium |
| Site Number - 4680 | Leuven | Vlaams Brabant | 03000 | Belgium |
| Site Number - 9103 | Brasília | Federal District | 70200-730 | Brazil |
| Site Number - 9101 | Curitiba | Paraná | 81210-310 | Brazil |
| Site Number - 9100 | Ribeirão Preto | São Paulo | 14048-900 | Brazil |
| Site Number - 9102 | Rio de Janeiro | 22640-100 | Brazil |
| Site Number - 9105 | São Paulo | 01409-000 | Brazil |
| Site Number - 9111 | Sofia | Sofia-Grad | 01527 | Bulgaria |
| Site Number - 9110 | Sofia | Sofia-Grad | 01606 | Bulgaria |
| Site Number - 9112 | Pleven | 05800 | Bulgaria |
| Site Number - 2600 | Edmonton | Alberta | T6J 1M3 | Canada |
| Site Number - 2603 | Vancouver | British Columbia | V6Z 1Y6 | Canada |
| Site Number - 4740 | Copenhagen | 02100 | Denmark |
| Site Number - 3241 | Turku | Southwest Finland | 20520 | Finland |
| Site Number - 6704 | München | Bavaria | 80337 | Germany |
| Site Number - 6705 | Bochum | North Rhine-Westphalia | 44791 | Germany |
| Site Number - 6700 | Dresden | Saxony | 01307 | Germany |
| Site Number - 6702 | Leipzig | Saxony | 04103 | Germany |
| Site Number - 6706 | Berlin | 10117 | Germany |
| Site Number - 6341 | Pátrai | Achaïa | 265 04 | Greece |
| Site Number - 6344 | Athens | Attica | 115 25 | Greece |
| Site Number - 6345 | Athens | Attica | 115 28 | Greece |
| Site Number - 6343 | Alexandroupoli | Evros | 68100 | Greece |
| Site Number - 6346 | Ioannina | Ioannina | 455 00 | Greece |
| Site Number - 6342 | Heraklion | Irakleio | 715 00 | Greece |
| Site Number - 6347 | Larissa | 41110 | Greece |
| Site Number - 6302 | Gussago | Brescia | 25084 | Italy |
| Site Number - 6305 | Bologna | Emilia-Romagna | 40139 | Italy |
| Site Number - 6304 | Udine | Friuli Venezia Giulia | 33100 | Italy |
| Site Number - 6309 | Rome | Lazio | 00133 | Italy |
| Site Number - 6306 | Rome | Lazio | 00189 | Italy |
| Site Number - 6301 | Bergamo | Lombardy | 24127 | Italy |
| Site Number - 6307 | Milan | Lombardy | 20132 | Italy |
| Site Number - 6303 | Pisa | Tuscany | 56126 | Italy |
| Site Number - 6308 | Siena | Tuscany | 53100 | Italy |
| Site Number - 6300 | Pavia | 27100 | Italy |
| Site Number - 6491 | Oslo | 00424 | Norway |
| Site Number - 3207 | Poznan | Greater Poland Voivodeship | 61-731 | Poland |
| Site Number - 3211 | Bydgoszcz | Kuyavian-Pomeranian Voivodeship | 85-065 | Poland |
| Site Number - 3203 | Bydgoszcz | Kuyavian-Pomeranian Voivodeship | 85-796 | Poland |
| Site Number - 3208 | Krakow | Lesser Poland Voivodeship | 30-688 | Poland |
| Site Number - 3209 | Krakow | Lesser Poland Voivodeship | 31-202 | Poland |
| Site Number - 3200 | Krakow | Lesser Poland Voivodeship | 31-426 | Poland |
| Site Number - 3204 | Wroclaw | Lower Silesian Voivodeship | 50-556 | Poland |
| Site Number - 3210 | Lublin | Lublin Voivodeship | 20-064 | Poland |
| Site Number - 3206 | Lublin | Lublin Voivodeship | 20-701 | Poland |
| Site Number - 3202 | Mazurki | Lublin Voivodeship | 20-093 | Poland |
| Site Number - 3205 | Gdansk | Pomeranian Voivodeship | 80-803 | Poland |
| Site Number - 3201 | Katowice | Silesian Voivodeship | 40-123 | Poland |
| Site Number - 3742 | Senhora da Hora | Porto District | 4464-513 | Portugal |
| Site Number - 3745 | Vila Nova de Gaia | Porto District | 4434-502 | Portugal |
| Site Number - 3743 | Almada | Setúbal District | 2805-267 | Portugal |
| Site Number - 3741 | Lisbon | 1300-344 | Portugal |
| Site Number - 3744 | Porto | 4099-001 | Portugal |
| Site Number - 8406 | Bucharest | Bucharest | 41914 | Romania |
| Site Number - 8401 | Târgu Mureş | Mureș County | 540136 | Romania |
| Site Number - 8403 | Timișoara | Timiș County | 300736 | Romania |
| SIte Number - 8400 | Constanța | 900591 | Romania |
| Site Number - 8501 | Belgrade | 11000 | Serbia |
| Site Number - 8502 | Belgrade | 11000 | Serbia |
| Site Number - 8500 | Niš | 18000 | Serbia |
| Site Number - 8503 | Novi Sad | 21000 | Serbia |
| Site Number - 8600 | Liptovský Mikuláš | 031 23 | Slovakia |
| Site Number - 8601 | Martin | 036 01 | Slovakia |
| Site Number - 8603 | Prešov | 081 81 | Slovakia |
| Site Number - 8602 | Trnava | 91775 | Slovakia |
| Site Number - 9901 | Seoul | 02841 | South Korea |
| Site Number - 9900 | Seoul | 06351 | South Korea |
| Site Number - 3701 | L'Hospitalet de Llobregat | Barcelona | 08907 | Spain |
| Site Number - 3704 | Sant Cugat del Vallès | Barcelona | 08190 | Spain |
| Site Number - 3700 | San Sebastián | Gipuzkoa | 20014 | Spain |
| Site Number - 3703 | Barcelona | 08025 | Spain |
| Site Number - 4891 | Gothenburg | Västra Götaland County | 413 45 | Sweden |
| Site Number - 7405 | Cambridge | Cambridgeshire | CB2 0QQ | United Kingdom |
| Site Number - 7402 | Southampton | Hampshire | SO16 6YD | United Kingdom |
| Site Number - 7404 | Glasgow | Lanarkshire | G51 4TF | United Kingdom |
| Site Number - 7403 | Preston | Lancashire | PR2 9HT | United Kingdom |
| Site Number - 7401 | Sheffield | South Yorkshire | S10 2JF | United Kingdom |
| Site Number - 7400 | Manchester | M6 8HD | United Kingdom |
| ID | Term |
|---|---|
| D020277 | Polyradiculoneuropathy, Chronic Inflammatory Demyelinating |
| D001327 | Autoimmune Diseases |
| ID | Term |
|---|---|
| D011129 | Polyradiculoneuropathy |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D007154 | Immune System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D007279 | Injections, Subcutaneous |
| ID | Term |
|---|---|
| D007267 | Injections |
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |
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