A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Empasiprubart in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

The primary objective of this study is to evaluate the efficacy of empasiprubart administered via intravenous infusion compared to placebo in improving functional ability in adults diagnosed with chronic inflammatory demyelinating polyneuropathy. 5

Secondary objectives include the assessment of the following outcomes:

• Efficacy regarding muscle strength, muscle function, and long-term impact on activity and social limitations. 5
• Clinical progression parameters, specifically the time to improvement and the time to clinical deterioration. 5
• Evaluation of gait impairment and long-term prevention of clinical decline. 5
• Impact on health-related quality of life and patient-reported outcome measures. 13
• Safety and tolerability profiles, including the assessment of immunogenicity. 4, 13
• Characterization of pharmacokinetics and pharmacodynamics. 6, 7

The study population consists of 100 patients diagnosed with chronic inflammatory demyelinating polyneuropathy. Participants include both males and females within specific age ranges. Inclusion requires meeting the EAN/PNS Task Force CIDP guidelines, second revision (2021), for typical CIDP or specific variants such as motor CIDP, multifocal CIDP, focal CIDP, or distal CIDP. Eligible individuals must present with active disease and residual disability. The cohort may include those who have not previously received treatment, those who have discontinued prior therapy, or those currently undergoing treatment with corticosteroids, immunoglobulins, plasma exchange, or FcRn inhibitors.

This Phase 3, randomized, double-blind, placebo-controlled study is designed to evaluate the efficacy and safety of empasiprubart administered via intravenous infusion in adults diagnosed with chronic inflammatory demyelinating polyneuropathy. The primary objective is to demonstrate improvements in functional ability compared to a placebo, specifically measuring the reduction in the aINCAT score at week 24. The research methodology includes an initial screening visit to confirm eligibility based on established clinical guidelines. Following screening, participants will undergo treatment and subsequent follow-up visits to monitor secondary endpoints, such as changes in the I-RODS centile points score, MRC-SS, and grip strength. The study duration for assessments includes observations up to 96 weeks in specific parts of the protocol to evaluate long-term changes in various clinical parameters. Safety monitoring involves the assessment of adverse events, serious adverse events, and laboratory parameters. Participation may be subject to early termination based on clinical conditions or safety requirements.

Empasiprubart is administered as a concentrate for solution for infusion. This investigational medicinal product is delivered via intravenous infusion at a dose of 000 mg.

The control group receives a placebo prepared as a solution for intravenous infusion.

The efficacy of empasiprubart in adults with chronic inflammatory demyelinating polyneuropathy is assessed through several clinical parameters. The primary endpoint is the reduction of ≥1 point from baseline in the aINCAT score at week 24. Secondary efficacy assessments include the following:

• Change from baseline in I-RODS centile points score up to 24 weeks (Part A).
• Change from baseline in MRC-SS at week 24 and over time up to 96 weeks (Part B).
• Change from baseline in grip strength, measured as a 3-day moving average in the dominant hand at week 24, and as both a 3-day moving average and a daily average for both hands over time.
• Time to reduction of ≥1 point from baseline in the aINCAT score up to 24 weeks.
• Time to increase of ≥1 point compared with baseline in the aINCAT score up to week 24.
• Change from baseline in EQ-5D-5L, RT-FSS, and BPI-SF over time.
• PGI-S and PGI-C values over time.
• Change from baseline in the TUG at week 24.
• Absolute values and percentage change from baseline in free C2 and total C2 over time.
• Serum concentrations of empasiprubart over time.
• Incidence and prevalence of ADA and NAb against empasiprubart in serum.