A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP (emnergize)
Purpose of this study?
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).
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)
- Participants already receiving CIDP treatment will have to discontinue their CIDP treatment before first IMP administration and must be willing to switch to the study IMP
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
Primary outcome measures:
- Reduction of ≥1 point compared with baseline in aINCAT score at week 24 [Time Frame: 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:
- Change from baseline in I-RODS centile points score [Time Frame: 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 [Time Frame: 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 [Time Frame: Up to 24 weeks]
- Time to reduction of ≥1 point from baseline in aINCAT score [Time Frame: 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 [Time Frame: 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 [Time Frame: 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 [Time Frame: Up to 24 weeks (part A)]
- Change from baseline in grip strength (daily average) for both hands [Time Frame: Up to 96 weeks (Part B)]
- Change from baseline in MRC-SS over time [Time Frame: 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 [Time Frame: 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 [Time Frame: 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 [Time Frame: 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 [Time Frame: 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 [Time Frame: 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 [Time Frame: 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 [Time Frame: Up to 24 weeks + 96 weeks (Part B)]
- Anti-drug antibodies
- Incidence of NAb against empasiprubart in serum [Time Frame: Up to 24 weeks + 96 weeks (Part B)]
- Neutralizing antibodies
- Incidence of AEs and SAEs [Time Frame: Up to 24 weeks + 96 weeks (Part B)]
- Percentage change from baseline in free C2 and total C2 over time [Time Frame: Up to 24 weeks (part A) + 96 weeks (Part B)]
- Serum concentrations of empasiprubart over time [Time Frame: Up to 24 weeks (Part A) + 96 weeks (Part B)]
- Reduction of ≥1 point in aINCAT over time [Time Frame: 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 [Time Frame: 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).
Study Locations in Europe: Netherlands, Slovakia, Denmark, Czechia, France, Hungary, Greece, Italy, Romania, Slovenia, Austria, Bulgaria, Estonia, Finland, Poland
Estimated Study Completion Date: 2031-01-23
Further information on clinicaltrials.gov – trial number NCT07091630