A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis
- Conditions
- Systemic Sclerosis (SSc)
- Registration Number
- NCT06655155
- Lead Sponsor
- argenx
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 81
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method Change from baseline in mRSS at week 24 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 Outcome Measures
Name Time Method 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 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.
Change from baseline in HAQ-DI at weeks 24 and 48 Up to 48 weeks 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).
Change from baseline in PGA at weeks 24 and 48 Up to 48 weeks 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).
Change from baseline in CGA at weeks 24 and 48 Up to 48 weeks 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).
Annualized rate of decline in FVC (in mL) in participants with interstitial lung disease Up to 48 weeks Forced Vital Capacity is a tool to assess lung function
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 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.
Trial Locations
- Locations (3)
The Alliance Medical Sciences Campus
🇵🇷San Juan, Puerto Rico
Kantonsspital St. Gallen
🇨🇭Sankt Gallen, Switzerland
Arizona Arthritis and Rheumatology Associates
🇺🇸Phoenix, Arizona, United States