To Assess Efficacy and Safety of Batoclimab in Adult Participants With Active CIDP

NCT05581199 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: IMVT-1401-2401
• Study Type: interventional
• Target: 277
• Locations: 20 countries
• Sites: 112

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.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy

Keywords

Chronic Inflammatory Demyelinating Polyneuropathy; IMVT-1401; Monoclonal antibody; Autoimmune disorders

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (12)

• 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

• 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

• Period 2, Cohort A: 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 Medical Research Council (MRC) Sum Score

  Baseline (Week 12) and up to Week 36

Study Arms (18)

Treatment Period 1: Cohort A, Dose 1

EXPERIMENTAL

Drug: Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly

Treatment Period 1: Cohort A, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Treatment Period 1: Cohort B, Dose 1

EXPERIMENTAL

Drug: Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly

Treatment Period 1: Cohort B, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Treatment Period 1: Cohort C, Dose 1

EXPERIMENTAL

Drug: Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly

Treatment Period 1: Cohort C, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Treatment Period 1: Cohort D, Dose 1

EXPERIMENTAL

Drug: Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly

Treatment Period 1: Cohort D, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Withdrawal Period 2: Cohort A, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Withdrawal Period 2: Cohort A, Placebo

EXPERIMENTAL

Drug: Placebo

Withdrawal Period 2: Cohort B, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Withdrawal Period 2: Cohort B, Placebo

EXPERIMENTAL

Drug: Placebo

Withdrawal Period 2: Cohort C, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Withdrawal Period 2: Cohort C, Placebo

EXPERIMENTAL

Drug: Placebo

Withdrawal Period 2: Cohort D, Dose 2

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Withdrawal Period 2: Cohort D, Placebo

EXPERIMENTAL

Drug: Placebo

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.

Drug: Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly; Drug: Batoclimab 340 mg SC weekly

LTE Period: Without Relapse in Period 2: Dose 2

EXPERIMENTAL

Participants will receive Dose 2 for all 52 weeks.

Drug: Batoclimab 340 mg SC weekly

Interventions

Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly DRUG

Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody

Also known as: IMVT-1401

LTE Period: With Relapse in Period 2: Dose 1 and Dose 2; Treatment Period 1: Cohort A, Dose 1; Treatment Period 1: Cohort B, Dose 1; Treatment Period 1: Cohort C, Dose 1; Treatment Period 1: Cohort D, Dose 1

Batoclimab 340 mg SC weekly DRUG

Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody

Also known as: IMVT-1401

LTE Period: With Relapse in Period 2: Dose 1 and Dose 2; LTE Period: Without Relapse in Period 2: Dose 2; Treatment Period 1: Cohort A, Dose 2; Treatment Period 1: Cohort B, Dose 2; Treatment Period 1: Cohort C, Dose 2; Treatment Period 1: Cohort D, Dose 2; Withdrawal Period 2: Cohort A, Dose 2; Withdrawal Period 2: Cohort B, Dose 2; Withdrawal Period 2: Cohort C, Dose 2; Withdrawal Period 2: Cohort D, Dose 2

Placebo DRUG

Matching placebo SC

Withdrawal Period 2: Cohort A, Placebo; Withdrawal Period 2: Cohort B, Placebo; Withdrawal Period 2: Cohort C, Placebo; Withdrawal Period 2: Cohort D, Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Progressive or relapsing, symmetric, proximal, and distal muscle weakness of upper and lower limbs, and sensory involvement of at least two limbs (at any point in the disease course)
• Developing over at least 8 weeks
• Absent or reduced tendon reflexes in all limbs
• CIDP variants: One of the following, but otherwise as in typical CIDP (tendon reflexes may be normal in unaffected limbs):
• Multifocal CIDP: documented sensory loss and muscle weakness in a multifocal pattern, usually asymmetric, upper limb predominant
• Focal CIDP: sensory loss and muscle weakness in only one limb
• Motor CIDP: motor symptoms and signs without sensory involvement
• 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:

You may not qualify if:

• 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:
• 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.

Sponsors & Collaborators

• Immunovant Sciences GmbH: lead

Study Sites (116)

Site Number - 1603

Scottsdale, Arizona, 85028, United States

Location

Site Number - 1618

Carlsbad, California, 92011, United States

Location

Site Number - 1634

Los Angeles, California, 90033-5330, United States

Location

Site Number - 1619

Orange, California, 92868, United States

Location

Site Number - 1608

San Francisco, California, 94109, United States

Location

Site Number - 1625

Aurora, Colorado, 80045, United States

Location

Site Number - 1636

Fort Collins, Colorado, 80528, United States

Location

Site Number - 1621

New Haven, Connecticut, 06519, United States

Location

Site Number - 1630

Washington D.C., District of Columbia, 20010, United States

Location

Site Number - 1600

Boca Raton, Florida, 33487, United States

Location

Site Number - 1609

Jacksonville, Florida, 32224, United States

Location

Site Number - 1631

Orlando, Florida, 32804, United States

Location

Site Number - 1629

Orlando, Florida, 32806-5411, United States

Location

Site Number - 1617

Ormond Beach, Florida, 32174, United States

Location

Site Number - 1620

Port Charlotte, Florida, 33952, United States

Location

Site Number - 1633

Rockledge, Florida, 32955, United States

Location

Site Number - 1604

St. Petersburg, Florida, 33713, United States

Location

Site Number - 1607

O'Fallon, Illinois, 62269, United States

Location

Site Number - 1602

Kansas City, Kansas, 66160, United States

Location

Site Number - 1611

Nicholasville, Kentucky, 40356, United States

Location

Site Number - 1622

Brooklyn, New York, 11220, United States

Location

Site Number - 1605

New York, New York, 10032, United States

Location

Site Number - 1635

Chapel Hill, North Carolina, 27559, United States

Location

Site Number - 1610

Charlotte, North Carolina, 28207, United States

Location

Site Number - 1614

Portland, Oregon, 97239, United States

Location

Site Number - 1623

Philadelphia, Pennsylvania, 19104, United States

Location

Site Number - 1624

Philadelphia, Pennsylvania, 19104, United States

Location

Site Number -1601

Austin, Texas, 78759, United States

Location

Site Number - 1606

Houston, Texas, 77030, United States

Location

Site Number - 1628

San Antonio, Texas, 78229, United States

Location

Site Number - 1627

Richmond, Virginia, 23298, United States

Location

Site Number - 1632

Seattle, Washington, 98195-0001, United States

Location

Site Number - 1613

Milwaukee, Wisconsin, 53226-3548, United States

Location

Site Number - 7753

Rosario, Santa Fe Province, S2000BZL, Argentina

Location

Site Number - 7751

Rosario, Santa Fe Province, S2000DTP, Argentina

Location

Site Number - 7752

San Miguel de Tucumán, Tucumán Province, T4000AXL, Argentina

Location

Site Number - 7750

Buenos Aires, C1199ABB, Argentina

Location

Site Number - 4681

Ghent, Oost-Vlaanderen, 09000, Belgium

Location

Site Number - 4680

Leuven, Vlaams Brabant, 03000, Belgium

Location

Site Number - 9103

Brasília, Federal District, 70200-730, Brazil

Location

Site Number - 9101

Curitiba, Paraná, 81210-310, Brazil

Location

Site Number - 9100

Ribeirão Preto, São Paulo, 14048-900, Brazil

Location

Site Number - 9102

Rio de Janeiro, 22640-100, Brazil

Location

Site Number - 9105

São Paulo, 01409-000, Brazil

Location

Site Number - 9111

Sofia, Sofia-Grad, 01527, Bulgaria

Location

Site Number - 9110

Sofia, Sofia-Grad, 01606, Bulgaria

Location

Site Number - 9112

Pleven, 05800, Bulgaria

Location

Site Number - 2600

Edmonton, Alberta, T6J 1M3, Canada

Location

Site Number - 2603

Vancouver, British Columbia, V6Z 1Y6, Canada

Location

Site Number - 4740

Copenhagen, 02100, Denmark

Location

Site Number - 3241

Turku, Southwest Finland, 20520, Finland

Location

Site Number - 6704

München, Bavaria, 80337, Germany

Location

Site Number - 6705

Bochum, North Rhine-Westphalia, 44791, Germany

Location

Site Number - 6700

Dresden, Saxony, 01307, Germany

Location

Site Number - 6702

Leipzig, Saxony, 04103, Germany

Location

Site Number - 6706

Berlin, 10117, Germany

Location

Site Number - 6341

Pátrai, Achaïa, 265 04, Greece

Location

Site Number - 6344

Athens, Attica, 115 25, Greece

Location

Site Number - 6345

Athens, Attica, 115 28, Greece

Location

Site Number - 6343

Alexandroupoli, Evros, 68100, Greece

Location

Site Number - 6346

Ioannina, Ioannina, 455 00, Greece

Location

Site Number - 6342

Heraklion, Irakleio, 715 00, Greece

Location

Site Number - 6347

Larissa, 41110, Greece

Location

Site Number - 6302

Gussago, Brescia, 25084, Italy

Location

Site Number - 6305

Bologna, Emilia-Romagna, 40139, Italy

Location

Site Number - 6304

Udine, Friuli Venezia Giulia, 33100, Italy

Location

Site Number - 6309

Rome, Lazio, 00133, Italy

Location

Site Number - 6306

Rome, Lazio, 00189, Italy

Location

Site Number - 6301

Bergamo, Lombardy, 24127, Italy

Location

Site Number - 6307

Milan, Lombardy, 20132, Italy

Location

Site Number - 6303

Pisa, Tuscany, 56126, Italy

Location

Site Number - 6308

Siena, Tuscany, 53100, Italy

Location

Site Number - 6300

Pavia, 27100, Italy

Location

Site Number - 6491

Oslo, 00424, Norway

Location

Site Number - 3207

Poznan, Greater Poland Voivodeship, 61-731, Poland

Location

Site Number - 3211

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-065, Poland

Location

Site Number - 3203

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-796, Poland

Location

Site Number - 3208

Krakow, Lesser Poland Voivodeship, 30-688, Poland

Location

Site Number - 3209

Krakow, Lesser Poland Voivodeship, 31-202, Poland

Location

Site Number - 3200

Krakow, Lesser Poland Voivodeship, 31-426, Poland

Location

Site Number - 3204

Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

Location

Site Number - 3210

Lublin, Lublin Voivodeship, 20-064, Poland

Location

Site Number - 3206

Lublin, Lublin Voivodeship, 20-701, Poland

Location

Site Number - 3202

Mazurki, Lublin Voivodeship, 20-093, Poland

Location

Site Number - 3205

Gdansk, Pomeranian Voivodeship, 80-803, Poland

Location

Site Number - 3201

Katowice, Silesian Voivodeship, 40-123, Poland

Location

Site Number - 3742

Senhora da Hora, Porto District, 4464-513, Portugal

Location

Site Number - 3745

Vila Nova de Gaia, Porto District, 4434-502, Portugal

Location

Site Number - 3743

Almada, Setúbal District, 2805-267, Portugal

Location

Site Number - 3741

Lisbon, 1300-344, Portugal

Location

Site Number - 3744

Porto, 4099-001, Portugal

Location

Site Number - 8406

Bucharest, București, 41914, Romania

Location

Site Number - 8401

Târgu Mureş, Mureș County, 540136, Romania

Location

Site Number - 8403

Timișoara, Timiș County, 300736, Romania

Location

SIte Number - 8400

Constanța, 900591, Romania

Location

Site Number - 8501

Belgrade, 11000, Serbia

Location

Site Number - 8502

Belgrade, 11000, Serbia

Location

Site Number - 8500

Niš, 18000, Serbia

Location

Site Number - 8503

Novi Sad, 21000, Serbia

Location

Site Number - 8600

Liptovský Mikuláš, 031 23, Slovakia

Location

Site Number - 8601

Martin, 036 01, Slovakia

Location

Site Number - 8603

Prešov, 081 81, Slovakia

Location

Site Number - 8602

Trnava, 91775, Slovakia

Location

Site Number - 9901

Seoul, 02841, South Korea

Location

Site Number - 9900

Seoul, 06351, South Korea

Location

Site Number - 3701

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

Location

Site Number - 3704

Sant Cugat del Vallès, Barcelona, 08190, Spain

Location

Site Number - 3700

San Sebastián, Gipuzkoa, 20014, Spain

Location

Site Number - 3703

Barcelona, 08025, Spain

Location

Site Number - 4891

Gothenburg, Västra Götaland County, 413 45, Sweden

Location

Site Number - 7405

Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

Location

Site Number - 7402

Southampton, Hampshire, SO16 6YD, United Kingdom

Location

Site Number - 7404

Glasgow, Lanarkshire, G51 4TF, United Kingdom

Location

Site Number - 7403

Preston, Lancashire, PR2 9HT, United Kingdom

Location

Site Number - 7401

Sheffield, South Yorkshire, S10 2JF, United Kingdom

Location

Site Number - 7400

Manchester, M6 8HD, United Kingdom

Location

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Autoimmune Diseases

Interventions

Injections, Subcutaneous

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Injections; Drug Administration Routes; Drug Therapy; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 12, 2022
• First Posted: October 14, 2022
• Study Start: December 15, 2022
• Primary Completion: January 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: November 25, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1); PL (12); DK (1); IT (10); FI (1); GR (7); SL (4); PT (5); BE (2); GB (6); NO (1); BR (5); AR (4); CA (2); KR (2); ES (4); US (33); BU (3); RO (4); DE (5)