A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis

Официальное название

A Randomized, Double-Blinded, Placebo-Controlled, Phase 2, Parallel-Group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Adult Participants With Systemic Sclerosis

Обзор

The main purpose of this study is to evaluate the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis. The study consists of a screening period, a treatment period of up to 48 weeks and a safety follow-up period. After the screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod PH20 SC or placebo. The total study duration can be up to approximately 15 months. More information can be found on: https://clinicaltrials.argenx.com/esscape

Вмешательства

• Комбинированный продукт Efgartigimod PH20 SC
  Subcutaneous efgartigimod PH20 SC given by prefilled syringe
• Другое Placebo PH20 SC
  Subcutaneous placebo PH20 SC given by prefilled syringe

Первичные конечные точки

• Change from baseline in mRSS at week 24 [Срок оценки: Up to 24 weeks]

Вторичные конечные точки (11)

• Change from baseline in mRSS at week 48 [Срок оценки: Up to 48 weeks]
• Incidence of treatment-emergent (serious) adverse events [Срок оценки: Up to 55 weeks]
• Proportion of participants who improve in at least 2 or at least 3 of the 5 core items of CRISS-25 at weeks 24 and 48 and do not have worsening in >1 component and have no significant SSc-related event(s) [Срок оценки: Up to 48 weeks]
• Change from baseline in HAQ-DI at weeks 24 and 48 [Срок оценки: Up to 48 weeks]
• Change from baseline in PGA at weeks 24 and 48 [Срок оценки: Up to 48 weeks]
• Change from baseline in CGA at weeks 24 and 48 [Срок оценки: Up to 48 weeks]
• Annualized rate of decline in FVC (in mL) in participants with interstitial lung disease [Срок оценки: Up to 48 weeks]
• Efgartigimod serum concentrations over time [Срок оценки: Up to 48 weeks]
• Percent change from baseline in total IgG levels in serum over time [Срок оценки: Up to 55 weeks]
• Incidence of anti-drug antibodies against efgartigimod in serum over time [Срок оценки: Up to 55 weeks]
• Incidence of antibodies against rHuPH20 in plasma over time [Срок оценки: Up to 55 weeks]

Критерии участия

Критерии включения

• Is aged ≥18 years and the local legal age of consent for clinical studies
• Has diffuse or limited SSc diagnosis and fulfills the 2013 ACR/EULAR classification criteria
• Has a positive antinuclear antibodies (ANA) test result at the central laboratory with titer of at least 1:160
• Has a Health Assessment Questionnaire-Disability Index (HAQ-DI) score of at least 0.5 OR a Patient Global Assessment (PGA) score of at least 3
• Has a modified Rodnan Skin Score (mRSS) score between 15 and 35
• The participant is anti-RNA polymerase III autoantibody negative at central laboratory and had the first non-Raynaud's phenomenon manifestation less than 5 years before screening or the participant is anti-RNA polymerase III autoantibody positive at central laboratory and had the first non-Raynaud's phenomenon manifestation less than 2 years before screening
• Has uninvolved or mildly thickened skin area in at least 1 injection site

Критерии исключения

• Isolated anticentromere antibodies (ACA) seropositivity at the central laboratory
• Significant Pulmonary Arterial Hypertension
• Severe digital vasculopathy within the past 3 months
• Skin thickening due to scleroderma mimics or localized scleroderma
• Scleroderma renal crisis within the past 6 months of participating to the study
• Another rheumatic autoimmune disease, except for secondary Sjögren's syndrome or fibromyalgia

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 · 9 центров

• Arizona Arthritis and Rheumatology Associates — Phoenix
• UCLA Ronald Reagan University of California Los Angeles Medical Center — Los Angeles
• IRIS Research and Development LLC — Plantation
• University of Illinois Health Outpatient Care Center — Chicago
• DelRicht Research, LLC — New Orleans
• Johns Hopkins Asthma and Allergy Center — Baltimore
• Thomas Jefferson University — Columbia
• University of Michigan Hospital — Ann Arbor
• … и ещё 1 центр

Аргентина · 8 центров

• Aprillus Asistencia e Investigacion — Buenos Aires
• Instituto de Investigación Clínica TyT — Buenos Aires
• Consultorios Médicos Dr. Doreski - Fundacion Respirar — Buenos Aires
• Hospital General de Agudos Dr. José María Ramos Mejia — Buenos Aires
• Sanatorio Allende S.A. — Córdoba
• Centro de Investigaciones Reumatológicas — San Miguel de Tucumán
• Clínica Mayo de U.M.C.B. S.R.L — San Miguel de Tucumán
• Centro de Investigaciones Médicas Tucumán — San Miguel de Tucumán

Италия · 6 центров

• Azienda Ospedaliera Universitaria Careggi — Florence
• Ospedale San Raffaele S.r.l. — Milan
• Azienda Ospedaliera Universitaria Luigi Vanvitelli — Naples
• Fondazione Policlinico Universitario A. Gemelli IRCCS — Roma
• Fondazione Policlinico Universitario Campus Bio-Medico — Roma
• IRCCS Istituto Clinico Humanitas — Rozzano

Франция · 5 центров

• CHU de Bordeaux - Hôpital Pellegrin — Bordeaux
• CHU de Lille - Hopital Claude Huriez — Lille
• CHU de Strasbourg - Hôpital de Hautepierre — Marseille
• CHU de Montpellier- Hôpital Saint Eloi — Montpellier
• AP-HP - Hôpital Cochin - Port-Royal, site Cochin — Paris

Польша · 5 центров

• Malopolskie Badania Kliniczne — Krakow
• Zespol Poradni Specjalistycznych REUMED — Lublin
• Twoja Przychodnia NCM — Nowa Sól
• MICS Centrum Medyczne Warszawa — Warsaw
• Niepubliczny Zaklad Opieki Zdrowotnej Biogenes — Wroclaw

Германия · 4 центра

• Auenlandklinik Bad Bramstedt GmbH — Bad Bramstedt
• Universitätsklinikum Schleswig-Holstein - Campus Lübeck — Lübeck
• Mühlenkreiskliniken - Johannes Wesling Klinikum Minden — Minden
• Universitätsklinikum Tübingen — Tübingen

Мексика · 4 центра

• Mediadvance Clinical S.A.P.I. de C.V. — Chihuahua City
• Centro Integral Reumatologia SA de CV — Guadalajara
• Centro de Investigación y Tratamiento Reumatológico S.C — Mexico City
• Medical Care and Research S.A. de C.V. — Mérida

Чили · 3 центра

• BIOCINETIC Ltda — Santiago
• Centro de Investigaciones Clinicas UC (CICUC) — Santiago
• Centro de especialidades médicas Vanguardia — Temuco

Хорватия · 3 центра

• Clinical Hospital Centre Osijek — Osijek
• University Hospital of Split — Split
• General Hospital Zadar — Zadar

Греция · 3 центра

• Olympion General Clinic — Pátrai
• University General Hospital of Patras — Pátrai
• Euromedica Kianous Stavros — Thessaloniki

Португалия · 3 центра

• ULS de Lisboa Ocidental, EPE - Hospital Egas Moniz — Lisbon
• ULS de Santa Maria,EPE - Hospital de Santa Maria — Lisbon
• ULS de Gaia/Espinho, EPE - Unidade I — Vila Nova de Gaia

Испания · 3 центра

• Hospital Del Mar — Barcelona
• C.H. Regional Reina Sofia — Córdoba
• Corporacio Sanitaria Parc Tauli — Sabadell

Великобритания · 3 центра

• Chapel Allerton Hospital — Leeds
• Royal Free Hospital — London
• Luton and Dunstable University Hospital — Luton

Бельгия · 2 центра

• UZ Gent — Ghent
• UZ Brussel — Jette

Болгария · 2 центра

• Medical Center Artmed OOD — Plovdiv
• Diagnostic Consultative Center Convex EOOD — Sofia

Литва · 2 центра

• Hospital of Lithuanian University of Health Sciences Kauno klinikos — Kaunas
• Klaipeda Hospital, Affiliate of Klaipeda University Hospital — Klaipėda

Румыния · 2 центра

• Sf.Maria Clinical Hospital — Bucharest
• Dr I Cantacuzino Clinical Hospital — Bucharest

Сербия · 2 центра

• Institute of Rheumatology - PPDS — Belgrade
• Military Medical Academy — Belgrade

Швейцария · 2 центра

• Kantonsspital St. Gallen — Sankt Gallen
• Universitätsspital Zürich — Zurich

Чехия · 1 центр

• Revmatologicky Ustav — Prague

Дания · 1 центр

• Rigshospitalet — Copenhagen

Венгрия · 1 центр

• Pecsi Tudomanyegyetem, Klinika Kozpont, Bor, Nemikortani es Onkodermatologiai Klinika — Pécs

Нидерланды · 1 центр

• Leids Universitair Medisch Centrum — Leiden

Пуэрто-Рико · 1 центр

• The Alliance Medical Sciences Campus — San Juan

Идентификаторы

NCT: NCT06655155 · ARGX-113-2317 · 2024-514539-67-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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