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

Official title

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

Overview

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

Interventions

• Combination product Efgartigimod PH20 SC
  Subcutaneous efgartigimod PH20 SC given by prefilled syringe
• Other Placebo PH20 SC
  Subcutaneous placebo PH20 SC given by prefilled syringe

Primary outcome measures

• Change from baseline in mRSS at week 24 [Time frame: Up to 24 weeks]

Secondary outcome measures (11)

• Change from baseline in mRSS at week 48 [Time frame: Up to 48 weeks]
• Incidence of treatment-emergent (serious) adverse events [Time frame: 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) [Time frame: Up to 48 weeks]
• Change from baseline in HAQ-DI at weeks 24 and 48 [Time frame: Up to 48 weeks]
• Change from baseline in PGA at weeks 24 and 48 [Time frame: Up to 48 weeks]
• Change from baseline in CGA at weeks 24 and 48 [Time frame: Up to 48 weeks]
• Annualized rate of decline in FVC (in mL) in participants with interstitial lung disease [Time frame: Up to 48 weeks]
• Efgartigimod serum concentrations over time [Time frame: Up to 48 weeks]
• Percent change from baseline in total IgG levels in serum over time [Time frame: Up to 55 weeks]
• Incidence of anti-drug antibodies against efgartigimod in serum over time [Time frame: Up to 55 weeks]
• Incidence of antibodies against rHuPH20 in plasma over time [Time frame: Up to 55 weeks]

Eligibility criteria

Inclusion criteria

• 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

Exclusion criteria

• 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

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 · 9 centers

• 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
• … and 1 more center

Argentina · 8 centers

• 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

Italy · 6 centers

• 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

France · 5 centers

• 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

Poland · 5 centers

• 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

Germany · 4 centers

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

Mexico · 4 centers

• 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

Chile · 3 centers

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

Croatia · 3 centers

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

Greece · 3 centers

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

Portugal · 3 centers

• 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

Spain · 3 centers

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

United Kingdom · 3 centers

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

Belgium · 2 centers

• UZ Gent — Ghent
• UZ Brussel — Jette

Bulgaria · 2 centers

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

Lithuania · 2 centers

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

Romania · 2 centers

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

Serbia · 2 centers

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

Switzerland · 2 centers

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

Czechia · 1 center

• Revmatologicky Ustav — Prague

Denmark · 1 center

• Rigshospitalet — Copenhagen

Hungary · 1 center

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

Netherlands · 1 center

• Leids Universitair Medisch Centrum — Leiden

Puerto Rico · 1 center

• The Alliance Medical Sciences Campus — San Juan

Identifiers

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

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗