A study to assess the efficacy and safety of Efgartigimod PH20 SC in adults with systemic sclerosis (esScape)
Descrizione riassuntiva dello studio
The main objective of this study is to learn more about how the investigational drug Efgartigimod works and whether it is effective and safe for people with SSc. The study also aims to show how much Efgartigimod affects the symptoms of SSc and quality of life. To understand how well Efgartigimod works, it will be compared to a placebo (a substance that looks like Efgartigimod but contains no active ingredient). Approximately 81 participants worldwide will take part in the study at various testing centers.
(BASEC)
Intervento studiato
Efgartigimod is an investigational drug being developed for the treatment of people with SSc. It is considered experimental as it is currently not approved by health authorities for the treatment of SSc. Efgartigimod helps to eliminate autoantibodies (which are types of proteins produced by the immune system that attack the body’s own tissues). The aim of the study is to understand what happens in the body when Efgartigimod lowers autoantibody levels to rebalance the immune system and potentially reduce the symptoms of SSc. Participants will be randomly assigned to one of two groups and will receive either Efgartigimod PH20 SC or a placebo (this is referred to as “randomization”). However, neither the participants nor the investigators will know which investigational drug is being administered (this is referred to as “double-blind”). The investigational drug (Efgartigimod or placebo) will be administered as a subcutaneous injection using a needle attached to a pre-filled syringe. The patient or a caregiver may administer the injections at the testing center or at home if the investigator approves. The study includes three phases (pre-evaluation period, treatment period, and safety follow-up). Some participants will be interviewed within 4 weeks after the last dose of the investigational drug about their experiences in the study (“closure interview”). At most visits, patients will provide blood and urine samples, undergo physical examinations, electrocardiograms, and lung function tests, and complete questionnaires. A skin biopsy may be performed optionally. The study will last approximately 59 weeks.
(BASEC)
Malattie studiate
Systemic sclerosis (SSc) is a rare, chronic autoimmune disease of connective tissue and has one of the highest mortality rates among rheumatological diseases. It is characterized by inflammation, progressive fibrosis of the skin and internal organs, and severe vascular disease. The disease causes moderate to severe skin problems and persistent difficulties in daily activities.
(BASEC)
Study participants will be included if they: - are at least 18 years old - have systemic sclerosis (SSc) - have test results showing an overactive immune system (based on an antibody test) (BASEC)
Criteri di esclusione
Study participants will not be included if they: - have previously received other medications that may interact with the investigational drug - have other known autoimmune diseases or other medical conditions (e.g., surgery) that may interact with the investigational drug or expose the participant to an unacceptable risk - are pregnant, breastfeeding, or intend to become pregnant during the study (BASEC)
Luogo dello studio
San Gallo, Zurigo
(BASEC)
Sponsor
argenx BV PPD Switzerland GmbH c/o Dufour Treuhand AG
(BASEC)
Contatto per ulteriori informazioni sullo studio
Persona di contatto in Svizzera
Vice President Clinical Development
+32 9 310 34 00
clinicaltrials@clutterargenx.comargenx BV
(BASEC)
Nome del comitato etico approvante (per studi multicentrici solo il comitato principale)
Ethikkommission Ostschweiz EKOS
(BASEC)
Data di approvazione del comitato etico
16.01.2025
(BASEC)
ID di studio ICTRP
NCT06655155 (ICTRP)
Titolo ufficiale (approvato dal comitato etico)
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 (BASEC)
Titolo accademico
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 (ICTRP)
Titolo pubblico
A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis (ICTRP)
Malattie studiate
Systemic Sclerosis (SSc) (ICTRP)
Intervento studiato
Combination Product: Efgartigimod PH20 SCOther: Placebo PH20 SC (ICTRP)
Tipo di studio
Interventional (ICTRP)
Disegno dello studio
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Criteri di inclusione/esclusione
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 Sjgren's syndrome or
fibromyalgia (ICTRP)
non disponibile
Endpoint primari e secondari
Change from baseline in mRSS at week 24 (ICTRP)
Change from baseline in mRSS at week 48;Incidence of treatment-emergent (serious) adverse events;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);Change from baseline in HAQ-DI at weeks 24 and 48;Change from baseline in PGA at weeks 24 and 48;Change from baseline in CGA at weeks 24 and 48;Annualized rate of decline in FVC (in mL) in participants with interstitial lung disease;Efgartigimod serum concentrations over time;Percent change from baseline in total IgG levels in serum over time;Incidence of anti-drug antibodies against efgartigimod in serum over time;Incidence of antibodies against rHuPH20 in plasma over time (ICTRP)
Data di registrazione
22.10.2024 (ICTRP)
Inclusione del primo partecipante
non disponibile
Sponsor secondari
non disponibile
Contatti aggiuntivi
Sabine Coppieters, MD, clinicaltrials@argenx.com, 857-350-4834 (ICTRP)
ID secondari
2024-514539-67-00, ARGX-113-2317 (ICTRP)
Risultati-Dati individuali dei partecipanti
non disponibile
Ulteriori informazioni sullo studio
https://clinicaltrials.gov/ct2/show/NCT06655155 (ICTRP)
Risultati dello studio
Riepilogo dei risultati
non disponibile
Link ai risultati nel registro primario
non disponibile