Clinical trial NCT06655155: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Systemic Sclerosis (SSc)
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-10-23
Enrollment: 81
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Change from baseline in mRSS at week 24
timeFrame: Up to 24 weeks
Secondary outcomes:
measure: Change from baseline in mRSS at week 48
timeFrame: Up to 48 weeks
measure: Incidence of treatment-emergent (serious) adverse events
timeFrame: Up to 55 weeks
measure: 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)
timeFrame: Up to 48 weeks
measure: Change from baseline in HAQ-DI at weeks 24 and 48
timeFrame: Up to 48 weeks
measure: Change from baseline in PGA at weeks 24 and 48
timeFrame: Up to 48 weeks
measure: Change from baseline in CGA at weeks 24 and 48
timeFrame: Up to 48 weeks
measure: Annualized rate of decline in FVC (in mL) in participants with interstitial lung disease
timeFrame: Up to 48 weeks
measure: Efgartigimod serum concentrations over time
timeFrame: Up to 48 weeks
measure: Percent change from baseline in total IgG levels in serum over time
timeFrame: Up to 55 weeks
measure: Incidence of anti-drug antibodies against efgartigimod in serum over time
timeFrame: Up to 55 weeks
measure: Incidence of antibodies against rHuPH20 in plasma over time
timeFrame: Up to 55 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Arizona Arthritis and Rheumatology Associates
state: Arizona
city: Ann Arbor
country: United States
facility: University of Michigan Hospital
state: Michigan
city: San Juan
country: Puerto Rico
facility: The Alliance Medical Sciences Campus
city: Sankt Gallen
country: Switzerland
facility: Kantonsspital St. Gallen
Study officials:
References: