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

Official title

A Phase 3, Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Empasiprubart IV in Adults With Chronic Inflammatory Demyelinating Polyneuropathy

Overview

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). More information can be found here: https://clinicaltrials.argenx.com/emnergize

Interventions

• Biological Empasiprubart IV
  Intravenous infusion of empasiprubart
• Other Placebo IV
  Intravenous infusion of placebo

Primary outcome measures

• Reduction of ≥1 point compared with baseline in aINCAT score at week 24 [Time frame: Up to 24 weeks]

Secondary outcome measures (12)

• Change from baseline in I-RODS centile points score [Time frame: Up to 24 weeks (part A) + 96 weeks (Part B)]
• Change from baseline in MRC-SS at week 24 [Time frame: Up to 24 weeks]
• 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]
• Change from baseline in TUG [Time frame: Up to 24 weeks (part A) + 96 weeks (Part B)]
• Time to increase of ≥1 point compared with baseline in aINCAT score up to week 24 [Time frame: Up to 24 weeks]
• 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)]
• Change from baseline in aINCAT score over time [Time frame: Up to 24 weeks + 96 weeks (Part B)]
• Change from baseline in EQ-5D-5L over time [Time frame: Up to 24 weeks (Part A) + 96 weeks (Part B)]
• Change from baseline in RT-FSS over time [Time frame: Up to 24 weeks (Part A) + 96 weeks (Part B)]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

United States · 10 centers

• University of Alabama at Birmingham - 1720 University Blvd — Birmingham
• Samir Macwan, M.D., Inc. (S corporation) — Rancho Mirage
• Colorado Springs Neurological Associates — Colorado Springs
• Medstar Health Research Institute — Washington D.C.
• Gables Neurology — Miami
• Aqualane Clinical Research — Naples
• Paradigm Health System — Slidell
• Penn Medicine University City — Philadelphia
• … and 2 more centers

Georgia · 8 centers

• High Technology Hospital MedCenter Ltd — Batumi
• First Medical Clinic LLC — Batumi
• Petre Sarajishvili Institute of Neurology — Tbilisi
• Aleksandre Aladashvili Clinic — Tbilisi
• Curatio JSC — Tbilisi
• LTD New Hospitals — Tbilisi
• Geo Hospitals — Tbilisi
• Jo Ann Medical Center — Tbilisi

Italy · 6 centers

• Fondazione Serena Onlus - Centro Clinico NeMO Brescia — Gussago
• Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI) - Ospedale di Cattinara — Trieste
• IRCCS Istituto delle Scienze Neurologiche di Bologna — Bologna
• IRCCS Azienda Ospedaliera Metropolitana - Ospedale Policlinico San Martino — Genova
• Ospedale San Raffaele S.r.l. — Milan
• Fondazione Istituto Neurologico Nazionale Casimiro Mondino IRCCS — Pavia

Bulgaria · 5 centers

• Multiprofile Hospital for Active Treatment of Neurology and Psychiatry Sv. Naum EAD — Sofia
• University Multiprofile Hospital for Active Treatment — Pleven
• Medical Center Medika 2005 - Branch Sofia — Sofia
• University Multiprofile Hospital for Active Treatment Aleksandrovska EAD — Sofia
• University Multiprofile Hospital for Active Treatment and Emergency Medicine N. I. Pirogov — Sofia

China · 5 centers

• Fujian Medical University Union Hospital - Yushan Campus — Fuzhou
• Peking University First Hospital - Changqiao Campus — Beijing
• Guangdong Provincial Hospital of Chinese Medicine — Guangzhou
• Nanfang Hospital Southern Medical University — Guangzhou
• Xi'an Gaoxin Hospital — Xi'an

France · 5 centers

• CHU de Lille - Hôpital Roger Salengro — Lille
• AP-HP - Hôpital de la Pitié Salpétrière — Paris
• AP-HP - Hôpital Henri Mondor — Créteil
• CHU de Bordeaux - Hôpital — Bourdeaux
• CHU de Nice-Hôpital Pasteur — Nice

Japan · 4 centers

• Institute of Science Tokyo Hospital — Bunkyō-Ku
• Seirei Hamamatsu Hospital — Hiroshima
• Southern TOHOKU Medical Clinic — Kōriyama
• Saga University Hospital — Saga

Argentina · 3 centers

• Instituto Privado Kremer — Córdoba
• Instituto Médico de la Fundación Estudios Clínicos — Rosario
• INECO Neurociencias Oroño — Rosario

Poland · 3 centers

• Centrum Medyczne Neurologia Slaska — Katowice
• SP ZOZ Szpital Uniwersytecki w Krakowie — Krakow
• Galen Clinic - Warszawa — Warsaw

Romania · 3 centers

• S.C. Neurocity S.R.L — Bucharest
• SC Clubul Sanatatii SRL — Campulung Muscel
• Spitalul Judetean de Urgenta Deva — Deva

Slovakia · 3 centers

• Univerzitna nemocnica MARTIN — Martin
• Nemocnica s poliklinikou S. Kukuru a.s. - Pentahospitals — Michalovce
• Penta Hospitals SK, a.s. - Rimavská Sobota — Rimavská Sobota

Austria · 2 centers

• Kepler Universitätsklinikum Linz - Med Campus III — Linz
• Universitätsklinikum AKH Wien — Vienna

Greece · 2 centers

• University General Hospital of Alexandroupolis — Alexandroupoli
• Eginitio Hospital — Athens

South Korea · 2 centers

• Seoul National University Hospital — Seoul
• Korea University Guro Hospital — Seoul

United Kingdom · 2 centers

• The National Hospital for Neurology and Neurosurgery - PPDS — London
• Salford Royal Hospital — Salford

Brazil · 1 center

• PSEG Centro de Pesquisa Clinica — São Paulo

Czechia · 1 center

• Fakultni nemocnice Brno — Brno

Denmark · 1 center

• Rigshospitalet — Copenhagen

Estonia · 1 center

• Astra Kliinik — Tallinn

Hungary · 1 center

• Semmelweis Egyetem Genomikai Medicina és Ritka Betegségek Intézete — Budapest

Moldova · 1 center

• Institutul de Neurologie si Neurochirurgie — Chisinau

Netherlands · 1 center

• Universitair Medisch Centrum Utrecht — Utrecht

Singapore · 1 center

• National Neuroscience Institute — Singapore

Identifiers

NCT: NCT07091630 · ARGX-117-2402 · 2025-520805-10-00

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗