A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP

Not Applicable

Not yet recruiting

• Conditions: Chronic Inflammatory Demyelinating Polyneuropathy; CIDP; Chronic Inflammatory Demyelinating Polyradiculoneuropathy
• Interventions: Biological: Empasiprubart IV; Other: Placebo IV

• Registration Number: NCT07091630
• Lead Sponsor: argenx

Brief Summary

The main purpose of this study is to demonstrate the efficacy and safety of empasiprubart in adults with CIDP. The study consists of a part A where participants will either receive empasiprubart or placebo for 24 weeks (6 months). Following part A, participants will enter part B in which all participants will receive empasiprubart for 96 weeks (24 months).

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-07
• Study First Submitted: 2025-07-22
• Study First Submitted QC: 2025-07-22
• Last Update Submitted: 2025-07-22
• Study First Posted: 2025-07-29
• Last Update Posted: 2025-07-29
• Study Start: 2025-09-01
• Primary Completion: 2027-10-07
• Study Completion: 2031-01-23

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 160

Inclusion Criteria

• Meets criteria for CIDP based on EAN/PNS Task Force CIDP guidelines, second revision (2021)
• Has either typical CIDP or 1 of the following CIDP variants: motor CIDP (including motor-predominant CIDP), multifocal CIDP (also known as Lewis-Sumner syndrome), focal CIDP, or distal CIDP
• Has residual disability and active disease
• Has not received previous treatment for CIDP; or has stopped receiving CIDP treatment; or is receiving CIDP treatment (pulsed or oral corticosteroids, immunoglobulins, PLEX, or FcRn inhibitors)

Exclusion Criteria

• Meets the criteria for possible CIDP based on EAN/PNS Task Force CIDP guidelines, second revision (2021)
• Sensory CIDP (including sensory-predominant CIDP)
• Polyneuropathy of other causes
• Clinical diagnosis of systemic lupus erythematosus (SLE)
• Use of other long-acting immunomodulatory treatment or prior treatment (at any time) with total lymphoid irradiation or bone marrow transplantation

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Part A - Empasiprubart	Empasiprubart IV	Participants receive empasiprubart during part A
Part A - Placebo	Placebo IV	Participants receive placebo during part A
Part B - Empasiprubart	Empasiprubart IV	Participants receive empasiprubart during part B. Participants from the empasiprubart arm in part A will receive placebo once to maintain the blind of part A.

Primary Outcome Measures

Name	Time	Method
Reduction of ≥1 point compared with baseline in aINCAT score at week 24	Up to 24 weeks	The Adjusted Inflammatory Neuropathy Cause and Treatment Disability Score (aINCAT) score is a 10-point scale that covers the functionality of legs and arms. The score varies between 0 and 10 (higher score, worse outcome).

Secondary Outcome Measures

Name	Time	Method
Change from baseline in I-RODS centile points score	Up to 24 weeks (part A) + 96 weeks (Part B)	Inflammatory Rasch-built Overall Disability Scale (I-RODS) assesses the limitations of activities and social participation in patients with inflammatory neuropathies like CIDP. The score ranges from 0 to 100 (higher score, worse outcome).
Change from baseline in MRC-SS at week 24	Up to 24 weeks	The Medical Research Council Sum Score evaluates motor strength. Evaluated on 6 muscle groups on each side, the score varies from 0 to 60 (lower score, worse outcome)
Change from baseline in grip strength (3-day moving average) in the dominant hand at week 24	Up to 24 weeks
Change from baseline in grip strength (daily average) for both hands	Up to 96 weeks (Part B)
Time to reduction of ≥1 point from baseline in aINCAT score	Up to 24 weeks	The Adjusted Inflammatory Neuropathy Cause and Treatment Disability Score (aINCAT) score is a 10-point scale that covers the functionality of legs and arms. The score varies between 0 and 10 (higher score, worse outcome).
Change from baseline in TUG	Up to 24 weeks (part A) + 96 weeks (Part B)	The Timed Up and Go Test (TUG) is a simple test top assess a person's mobility in which the time expended to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down is measured.
Time to increase of ≥1 point compared with baseline in aINCAT score up to week 24	Up to 24 weeks	The Adjusted Inflammatory Neuropathy Cause and Treatment Disability Score (aINCAT) score is a 10-point scale that covers the functionality of legs and arms. The score varies between 0 and 10 (higher score, worse outcome).
Change from baseline in grip strength (3-day moving average) of both hands over time	Up to 24 weeks (part A)
Change from baseline in MRC-SS over time	Up to 96 weeks (Part B)	The Medical Research Council Sum Score evaluates motor strength. Evaluated on 6 muscle groups on each side, the score varies from 0 to 60 (lower score, worse outcome).
Change from baseline in aINCAT score over time	Up to 24 weeks + 96 weeks (Part B)	The Adjusted Inflammatory Neuropathy Cause and Treatment Disability Score (aINCAT) score is a 10-point scale that covers the functionality of legs and arms. The score varies between 0 and 10 (higher score, worse outcome).
Change from baseline in EQ-5D-5L over time	Up to 24 weeks (Part A) + 96 weeks (Part B)	EQ-5D-5L questionnaire is a patient-reported outcome measure, ranging 0 to 100 (lower score, worse outcome).
Change from baseline in RT-FSS over time	Up to 24 weeks (Part A) + 96 weeks (Part B)	Rasch-Transformed Fatigue Severity Scale (RT-FSS) is a patient-reported outcome measure to distinguish fatigue from clinical depression.
Change from baseline in BPI-SF over time	Up to 24 weeks (Part A) + 96 weeks (Part B)	The Brief Pain Inventory-Short Form (BPI-SF) is a patientreported outcome measure to assess pain severity and pain interference.
PGI-S values over time	Up to 24 weeks (Part A) + 96 weeks (Part B)	The Patient Global Impression of Severity (PGI-S) is a patient-reported outcome measure that reflects the patient's belief about the severity of the illness.
PGI-C values over time	Up to 24 weeks (Part A) + 96 weeks (Part B)	The Patient Global Impression of Change (PGI-C) is a patient-reported outcome measure that reflects the patient's belief about the efficacy of treatment.
Incidence of ADA against empasiprubart in serum	Up to 24 weeks + 96 weeks (Part B)	Anti-drug antibodies
Incidence of NAb against empasiprubart in serum	Up to 24 weeks + 96 weeks (Part B)	Neutralizing antibodies
Incidence of AEs and SAEs	Up to 24 weeks + 96 weeks (Part B)
Percentage change from baseline in free C2 and total C2 over time	Up to 24 weeks (part A) + 96 weeks (Part B)
Serum concentrations of empasiprubart over time	Up to 24 weeks (Part A) + 96 weeks (Part B)
Reduction of ≥1 point in aINCAT over time	Up to 96 weeks (Part B)	The Adjusted Inflammatory Neuropathy Cause and Treatment Disability Score (aINCAT) score is a 10-point scale that covers the functionality of legs and arms. The score varies between 0 and 10 (higher score, worse outcome)
Increase of ≥1 point from baseline in aINCAT over time	Up to 96 weeks (Part B)	The Adjusted Inflammatory Neuropathy Cause and Treatment Disability Score (aINCAT) score is a 10-point scale that covers the functionality of legs and arms. The score varies between 0 and 10 (higher score, worse outcome).