Resumen

Este es un estudio multicéntrico, aleatorizado, cuádruple ciego, controlado con placebo, para evaluar la eficacia y seguridad de batoclimab en participantes adultos con PDIC activa. El estudio incluye un período de selección de hasta 4 semanas, un período de lavado de hasta 12 semanas, un período de tratamiento aleatorizado de 12 semanas (período 1), un período de retiro aleatorizado de hasta 24 semanas (período 2), un período de extensión a largo plazo (ELP) de hasta 52 semanas (opcional), y seguimiento de seguridad 4 semanas después de la última dosis del tratamiento del estudio. La duración total del estudio será de aproximadamente 109 semanas. Los participantes elegibles serán asignados a una de cuatro cohortes según su tratamiento de PDIC basal (cohortes A y D: inmunoglobulina [Ig] o plasmaféresis [PLEX]; cohorte B: corticosteroides; cohorte C: sin tratamiento previo o sin tratamiento en los últimos 3-24 meses) y si cumplen el diagnóstico según los criterios de la European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) (cohortes A, B y C) o solo criterios clínicos (cohorte D) al momento de la selección.

Elegibilidad

Edad mínima: 18 YearsSexo: ALL

Criterios de inclusión

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:
Objective improvement to an empiric trial of therapy with immunoglobulin treatment, plasma exchange (PLEX), or corticosteroids.
Diagnostic imaging by ultrasound or magnetic resonance imaging (MRI) supporting the diagnosis of CIDP by demonstrating nerve enlargement.
Cerebrospinal fluid (CSF) demonstrating albuminocytologic dissociation (i.e., elevated CSF protein level \[defined as \> 70 milligrams per deciliter {mg/dL} or \> 10 mg/dL greater than years of age for those aged 60 years and over\] with normal CSF white blood cell \[WBC\] level).
Nerve biopsy demonstrating features supporting the diagnosis of CIDP, such as edema, demyelination, and/or onion bulb formation.
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.
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.

Criterios de exclusión

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:

Intervenciones

drug

Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly

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

drug

Batoclimab 340 mg SC weekly

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

drug

Placebo

Matching placebo SC

Estudio de fase 2b, multicéntrico, aleatorizado, cuádruple ciego, controlado con placebo, de tratamiento con batoclimab en participantes adultos con polineuropatía desmielinizante inflamatoria crónica (PDIC) activa

Resumen

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly

Batoclimab 340 mg SC weekly

Placebo

Ubicaciones

Site Number - 7753

Site Number - 7751

Site Number - 7752

Site Number - 7750

Patrocinadores