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

Conditions

Systemic Sclerosis

Brief summary

Change from baseline in modified Rodnan Skin Score (mRSS) at week 24

Detailed description

1. Change from baseline in mRSS at week 48, 2. Incidence and severity of treatment-emergent adverse events (AEs), serious AEs (SAEs), and AEs leading to discontinuation of investigational medicinal product (IMP), 3. Clinically meaningful changes in laboratory parameters, electrocardiograms (ECGs), and vital signs, 4. Proportion of participants who improve in ≥2 or ≥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), 5. Change from baseline in Health Assessment Questionnaire—Disability Index (HAQ-DI) at weeks 24 and 48, 6. Change from baseline in Patient Global Assessment (PGA) at weeks 24 and 48, 7. Change from baseline in Clinician’s Global Assessment (CGA) at weeks 24 and 48, 8. Annualized rate of decline in forced vital capacity (FVC; in mL) in participants with interstitial lung disease (ILD), 9. Efgartigimod serum concentrations over time, 10. Percent change from baseline in total IgG levels in serum over time, 11. Incidence and prevalence of antidrug antibodies (ADA) against efgartigimod in serum over time, 12. Incidence and prevalence of antibodies against recombinant human hyaluronidase PH20 (rHuPH20) in plasma over time

Related papers

No related papers found yet.

Interventions

DRUGplacebo PH20 SC PFS

DRUGVyvgart 1 000 mg solution for injection in pre-filled syringe

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Change from baseline in modified Rodnan Skin Score (mRSS) at week 24	—

Secondary

Measure	Time frame
1. Change from baseline in mRSS at week 48, 2. Incidence and severity of treatment-emergent adverse events (AEs), serious AEs (SAEs), and AEs leading to discontinuation of investigational medicinal product (IMP), 3. Clinically meaningful changes in laboratory parameters, electrocardiograms (ECGs), and vital signs, 4. Proportion of participants who improve in ≥2 or ≥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), 5. Change from baseline in Health Assessment Questionnaire—Disability Index (HAQ-DI) at weeks 24 and 48, 6. Change from baseline in Patient Global Assessment (PGA) at weeks 24 and 48, 7. Change from baseline in Clinician’s Global Assessment (CGA) at weeks 24 and 48, 8. Annualized rate of decline in forced vital capacity (FVC; in mL) in participants with interstitial lung disease (ILD), 9. Efgartigimod serum concentrations over time, 10. Percent change from baseline in total IgG le	—

Measure

Time frame

1. Change from baseline in mRSS at week 48, 2. Incidence and severity of treatment-emergent adverse events (AEs), serious AEs (SAEs), and AEs leading to discontinuation of investigational medicinal product (IMP), 3. Clinically meaningful changes in laboratory parameters, electrocardiograms (ECGs), and vital signs, 4. Proportion of participants who improve in ≥2 or ≥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), 5. Change from baseline in Health Assessment Questionnaire—Disability Index (HAQ-DI) at weeks 24 and 48, 6. Change from baseline in Patient Global Assessment (PGA) at weeks 24 and 48, 7. Change from baseline in Clinician’s Global Assessment (CGA) at weeks 24 and 48, 8. Annualized rate of decline in forced vital capacity (FVC; in mL) in participants with interstitial lung disease (ILD), 9. Efgartigimod serum concentrations over time, 10. Percent change from baseline in total IgG le

—

Countries

Belgium, Bulgaria, Croatia, Czechia, Denmark, France, Germany, Greece, Hungary, Italy, Lithuania, Netherlands, Poland, Portugal, Romania, Spain

Outcome results

None listed