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

Phase 2RecruitingNCT06655155

argenx81 enrolled

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Systemic Sclerosis (SSc)

Interventions

Efgartigimod PH20 SC

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (69)

Arizona Arthritis and Rheumatology Associates

Phoenix, Arizona, United States

RECRUITING

UCLA Ronald Reagan University of California Los Angeles Medical Center

Los Angeles, California, United States

RECRUITING

IRIS Research and Development LLC

Plantation, Florida, United States

RECRUITING

University of Illinois Health Outpatient Care Center

Chicago, Illinois, United States

Outcomes

Primary Outcomes

Change from baseline in mRSS at week 24

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.

Time frame: Up to 24 weeks

Secondary Outcomes

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)

Revised Composite Response Index in Systemic Sclerosis (CRISS-25) is a global composite outcome assessment that provides a holistic perspective of the disease. It includes 5 core items: the Modified Rodnan Skin Score, the Clinician's global assessment, the Forced Vital Capacity, the Patient global assessment, and the Health Assessment Questionnaire-Disability Index.

Time frame: Up to 48 weeks

Change from baseline in HAQ-DI at weeks 24 and 48

Health Assessment Questionnaire-Disability Index is a tool to assess physical function and disability. It consists of 8 categories with each question scored from 0 (without any difficulty) to 3 (unable to do).

Time frame: Up to 48 weeks

Change from baseline in PGA at weeks 24 and 48

Patient's Global Assessment is a tool that measures a participant's global evaluation of their overall health during the previous week using a 10-point numerical scale. The score varies between 0 (excellent overall health) and 10 (extremely poor overall health).

Time frame: Up to 48 weeks

Change from baseline in CGA at weeks 24 and 48

Clinician's Global Assessment is a tool that measures a clinician's assessment of the overall health of a participant during the previous week using a 10-point numerical scale. The score varies between 0 (excellent overall health) and 10 (extremely poor overall health).

Time frame: Up to 48 weeks

Annualized rate of decline in FVC (in mL) in participants with interstitial lung disease

Forced Vital Capacity is a tool to assess lung function

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

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

Overview

Conditions

Interventions

Eligibility

Locations (69)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts