A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis
ConditionSystemic Sclerosis (SSc)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
Sponsorargenx
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
Efgartigimod PH20 SC
Combination productSubcutaneous efgartigimod PH20 SC given by prefilled syringe
Placebo PH20 SC
Other interventionSubcutaneous placebo PH20 SC given by prefilled syringe
Treatment groups
81 Participants
are divided into 2 treatment groupsGroup A: Efgartigimod PH20 SCExperimental treatment 1 intervention
Group B: Placebo PH20 SCPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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 weeksSecondary outcomes
1
Change from baseline in mRSS at week 48
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 48 weeks2
Incidence of treatment-emergent (serious) adverse events
Time frame
Up to 55 weeks3
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 weeks4
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 Source ClinicalTrials.gov