Efficacy and Safety Study of Nipocalimab for Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Janssen (J&J Innovative Medicine)

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Treatments: Drug: Nipocalimab; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05327114

2021-003234-37 (EudraCT Number)

CR109195

2023-508425-28-00 (Registry Identifier)

80202135CDP3001 (Other Identifier)

Details and patient eligibility

About

The main purpose of this study is to evaluate the safety and efficacy of nipocalimab compared to placebo in delaying relapse in adults with chronic inflammatory demyelinating polyneuropathy (CIDP) who initially respond to nipocalimab in Stage A.

Full description

CIDP is a rare, chronic autoimmune disease of the peripheral nervous system characterized by progressive weakness and impaired sensation. Nipocalimab (also referred to as JNJ-80202135 or M281) is a fully human aglycosylated immunoglobulin (Ig)G1 monoclonal antibody (mAb) designed to selectively bind, saturate, and block the IgG binding site on the endogenous neonatal Fc receptor. The study will consist of the following periods: (a) identification of participants with active CIDP (including screening [up to 4 weeks] and run-in [up to 12 weeks]); (b) open-label treatment with nipocalimab (Stage A) (12 weeks); (c) double-blind, placebo-controlled, randomized withdrawal (Stage B) (up to 52 weeks); and (d) an open-label extension (OLE) (until to 2 years after marketing authorization in the participant's local country or until nipocalimab becomes available commercially or via other continued access program, whichever comes first). Participants who discontinue treatment and intend to withdraw from the study at any point during the treatment periods (Stage A, Stage B, or the OLE) will be requested to enter an 8-weeks follow-up after the last dose of study intervention. Efficacy, safety, pharmacokinetics (PK), immunogenicity, and biomarker evaluations will be assessed.

Enrollment: 201 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Adults greater than or equal to (>=) 18 years of age at the time of consent and as applicable, must also meet the legal age of consent in the jurisdiction in which the study is taking place
• Diagnosed with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) according to criteria of the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) 2021, progressing or relapsing forms. CIDP diagnosis to be adjudicated by independent committee during screening period
• Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score between 2 and 9 (a score of 2 has to be exclusively from leg disability)
• Fulfilling any of the following treatment conditions: a) Currently treated with oral corticosteroids (CS) less than or equal to (<=) 20 milligrams (mg)/day; or b) Currently treated with pulsed CS, and/or intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) and the participant is willing to discontinue no later than the run-in baseline visit; or c) Currently treated with oral CS greater than (>) 20 mg/day and the participant is willing to taper to <=20 mg/day during the run-in period; or d) Without previous treatment (treatment naive); or treatment with CS and/or IVIg or SCIg discontinued at least 3 months prior to screening (untreated)
• Active disease as determined by CIDP Disease Activity Status (CDAS) score >= 3
• Other protocol-defined inclusion criteria will apply

Exclusion criteria

• Has a history of severe and/or uncontrolled hepatic (example, viral/alcoholic/autoimmune hepatitis/cirrhosis and/or metabolic liver disease), gastrointestinal, renal, pulmonary, cardiovascular, psychiatric, neurological or musculoskeletal disorder, hypertension and/or any other medical or uncontrolled autoimmune disorder(s) (example, diabetes mellitus) or clinically significant abnormalities in screening laboratory that, might interfere with the participants full participation in the study, or might jeopardize the safety of the participant or the validity of the study results
• Pure sensory CIDP or chronic immune sensory polyradiculopathy (CISP) (EAN/PNS definition)
• Any other disease that could better explain the participant's signs and symptoms, such as significant persisting neurological deficits from stroke or central nervous system (CNS) trauma or peripheral neuropathy from another cause such as connective tissue disease or systemic lupus erythematosus
• Polyneuropathy of other causes, including the following: Multifocal motor neuropathy (MMN); Monoclonal gammopathy of uncertain significance with antimyelin associated glycoprotein (anti-MAG) immunoglobulin M (IgM) antibodies; Hereditary motor neuropathy; Hereditary neuropathy with liability to pressure palsies (HNPP); Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes; Lumbosacral radiculoplexus neuropathy; Polyneuropathy most likely due to diabetes mellitus; Polyneuropathy most likely due to systemic illnesses; Drug- or toxin-induced polyneuropathy Note: A concomitant polyneuropathy of other causes (example, a mild, stable diabetic polyneuropathy) is not necessarily exclusionary if chronic inflammatory demyelinating polyneuropathy (CIDP) is confirmed as the main diagnosis, as determined by the investigator and confirmed by the adjudication committee
• Has known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients
• Other protocol-defined exclusion criteria will apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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201 participants in 2 patient groups, including a placebo group

Nipocalimab

Experimental group

Description:

Participants in Stage A (Open-label) will receive a loading dose of nipocalimab (Dose 1) intravenous (IV) infusion on Day 1, followed by nipocalimab (Dose 2) IV infusion once every 2 weeks (q2w) from Week 2 to Week 12. Participants who demonstrate evidence of clinical improvement in Stage A (responders) will enter Stage B (Double-blind) and receive nipocalimab (Dose 2) IV infusion q2w starting on Day 1 up to Week 52. After completion of Stage B or discontinuation from Stage B due to relapse, participants will have the option to enter the open label extension (OLE) phase and receive nipocalimab (Dose 2) IV infusion q2w starting on OLE Day 1 until 2 years after marketing authorization in a participant's local country or until nipocalimab becomes available commercially or via other continued access program, whichever comes first.

Treatment:

Drug: Nipocalimab

Placebo

Placebo Comparator group

Description:

Participants receiving nipocalimab in Stage A and who demonstrate evidence of clinical improvement in Stage A (responders) will enter Stage B (Double-blind) and receive placebo IV infusion q2w starting on Day 1 up to Week 52. After completion of Stage B or discontinuation from Stage B due to relapse, participants will have the option to enter the open label extension (OLE) phase and receive nipocalimab (Dose 2) IV infusion q2w starting on OLE Day 1 until 2 years after marketing authorization in a participant's local country or until nipocalimab becomes available commercially or via other continued access program, whichever comes first.

Treatment:

Drug: Placebo