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

argenx116 sites in 20 countries81 target enrollmentNovember 11, 2024

ConditionsSystemic Sclerosis (SSc)

InterventionsEfgartigimod PH20 SC Placebo PH20 SC

Overview

Phase: Phase 2
Intervention: Efgartigimod PH20 SC
Conditions: Systemic Sclerosis (SSc)
Sponsor: argenx
Enrollment: 81
Locations: 116
Primary Endpoint: Change from baseline in mRSS at week 24
Status: Recruiting
Last Updated: 12 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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

Registry

clinicaltrials.gov

Start Date

November 11, 2024

End Date

September 1, 2027

Last Updated

12 days ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

argenx

Responsible Party

Sponsor

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

Arms & Interventions

Efgartigimod PH20 SC

Participants receiving efgartigimod PH20 SC

Intervention: Efgartigimod PH20 SC

Placebo PH20 SC

Participants receiving placebo PH20 SC

Intervention: Placebo PH20 SC

Outcomes

Primary Outcomes

Change from baseline in mRSS at week 24

Time Frame: Up to 24 weeks

The Modified Rodnan Skin Score is a scoring tool to assess skin thickness in 17 cutaneous sites across the body. Each site is rated on a semiquantitative score ranging from 0 (normal) to 3 (severe). The total score is the sum of the individual skin scores and can range from 0 to 51.

Secondary Outcomes

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