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

Официальное название

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

Обзор

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

Вмешательства

• Биопрепарат Empasiprubart IV
  Intravenous infusion of empasiprubart
• Другое Placebo IV
  Intravenous infusion of placebo

Первичные конечные точки

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

Вторичные конечные точки (12)

• Change from baseline in I-RODS centile points score [Срок оценки: Up to 24 weeks (part A) + 96 weeks (Part B)]
• Change from baseline in MRC-SS at week 24 [Срок оценки: Up to 24 weeks]
• Change from baseline in grip strength (3-day moving average) in the dominant hand at week 24 [Срок оценки: Up to 24 weeks]
• Time to reduction of ≥1 point from baseline in aINCAT score [Срок оценки: Up to 24 weeks]
• Change from baseline in TUG [Срок оценки: 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 [Срок оценки: Up to 24 weeks]
• Change from baseline in grip strength (3-day moving average) of both hands over time [Срок оценки: Up to 24 weeks (part A)]
• Change from baseline in grip strength (daily average) for both hands [Срок оценки: Up to 96 weeks (Part B)]
• Change from baseline in MRC-SS over time [Срок оценки: Up to 96 weeks (Part B)]
• Change from baseline in aINCAT score over time [Срок оценки: Up to 24 weeks + 96 weeks (Part B)]
• Change from baseline in EQ-5D-5L over time [Срок оценки: Up to 24 weeks (Part A) + 96 weeks (Part B)]
• Change from baseline in RT-FSS over time [Срок оценки: Up to 24 weeks (Part A) + 96 weeks (Part B)]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 10 центров

• 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
• … и ещё 2 центра

Грузия · 8 центров

• 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

Италия · 6 центров

• 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

Болгария · 5 центров

• 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

Китай · 5 центров

• Fujian Medical University Union Hospital - Yushan Campus — Фучжоу
• Peking University First Hospital - Changqiao Campus — Пекин
• Guangdong Provincial Hospital of Chinese Medicine — Гуанчжоу
• Nanfang Hospital Southern Medical University — Гуанчжоу
• Xi'an Gaoxin Hospital — Сиань

Франция · 5 центров

• 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

Япония · 4 центра

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

Аргентина · 3 центра

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

Польша · 3 центра

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

Румыния · 3 центра

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

Словакия · 3 центра

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

Австрия · 2 центра

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

Греция · 2 центра

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

South Korea · 2 центра

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

Великобритания · 2 центра

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

Бразилия · 1 центр

• PSEG Centro de Pesquisa Clinica — São Paulo

Чехия · 1 центр

• Fakultni nemocnice Brno — Brno

Дания · 1 центр

• Rigshospitalet — Copenhagen

Эстония · 1 центр

• Astra Kliinik — Tallinn

Венгрия · 1 центр

• Semmelweis Egyetem Genomikai Medicina és Ritka Betegségek Intézete — Budapest

Moldova · 1 центр

• Institutul de Neurologie si Neurochirurgie — Chisinau

Нидерланды · 1 центр

• Universitair Medisch Centrum Utrecht — Utrecht

Сингапур · 1 центр

• National Neuroscience Institute — Singapore

Идентификаторы

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

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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