Clinical trial NCT05817669: A Study of the Safety and Effectiveness of Efgartigimod in Patients With Primary Sjögren's Syndrome (pSS)
Official title: A Phase 2, Randomized, Placebo-controlled, Parallel-group, Double-blinded, proof-of Concept Study to Evaluate the Safety and Efficacy of Intravenous Efgartigimod in Adult Participants With Primary Sjögren's Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Primary Sjögren's Syndrome
Lead sponsor: argenx
Sponsor class: INDUSTRY
Collaborators: name: Iqvia Pty Ltd
First posted: 2023-04-18
Enrollment: 34
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 purpose of this study is to assess the efficacy and safety of human FcRn blocking therapy with efgartigimod compared to placebo, in participants with pSS.

Detailed description: Primary Sjogren Syndrome (pSS) is an autoimmune disease with still unmet treatment needs. Efgartigimod, a human FcRn antagonist, has the potential to successfully treat pSS and improve disease manifestations by the reduction of IgG autoantibodies and immune complexes in pSS. The study design is randomized, double-blinded, and placebo-controlled to evaluate the effect of efgartigimod administered as an IV infusion compared to placebo. The study consists of a treatment period when all participants will receive infusions of IP/placebo for 24 weeks. At the end of the randomized treatment period, eligible participants may roll over to an OLE study or remain in this study through the end of the 56-day follow-up period.

Eligibility criteria: Inclusion Criteria:

* Is at least the legal age of consent for clinical trials when signing the informed consent form
* Is capable of providing signed informed consent and complying with protocol requirements
* Agrees to use contraceptive measures consistent with local regulations and measures described in the protocol
* Meets the following criteria at screening: ACR/EULAR 2016 pSS who met criteria ≤7 years before screening; ESSDAI ≥5; Anti-Ro/SS-A positive; Residual salivary flow (UWSF rate \>0 and/or SWSF rate \>0.10)

Exclusion Criteria:

* Known autoimmune disease or any medical condition that, in the investigator's judgment,would interfere with an accurate assessment of clinical symptoms of pSS or puts the participant at undue risk
* History of malignancy unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP.
* Adequately treated participants with the following cancers may be included at any time: Basal cell or squamous cell skin cancer; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological finding of prostate cancer (TNM stage T1a or T1b) Clinically significant uncontrolled active acute or chronic bacterial, viral, or fungal infection
* Positive serum test at screening for an active infection with any of the following: HBV that is indicative of an acute or chronic infection, unless associated with a negative HBsAg or negative HBV DNA test; HCV based on HCV antibody assay unless a negative RNA test is available; HIV based on test results of a CD4 count of \<200 cells/mm3 that are associated with an AIDS-defining condition, HIV based on test results of a CD4 count of \>200 cells/mm3 not adequately treated with antiviral therapy
* Clinically significant disease, recent major surgery (within 3 months of screening), or intention to have surgery during the study; or any other medical condition that, in the investigator's opinion, would confound the results of the study or put the participant at undue risk
* Immunoglobulin G (IgG) levels cannot be below a certain threshold ( 4g/L)
* Positive covid test at study start
* Some of the medications such as vaccines with live components or medicines that may be prescribed cannot be taken either shortly before or during this study
* Current participation in another interventional clinical study or previously participation in an efgartigimod clinical study and treatment with ≥1 dose of IMP
* Known hypersensitivity to IMP or 1 of its excipients
* History (within 12 months of screening) of current alcohol, drug, or medication abuse as assessed by the investigator
* Pregnant or lactating state or intention to become pregnant during the study
* Secondary Sjögren's syndrome overlap syndromes where another confirmed autoimmune rheumatic or systemic inflammatory condition is the primary diagnosis
* Chinese traditional medicine with known immunomodulatory action

Primary outcomes:
measure: Proportion of CRESS responders on ≥3 of 5 items at week 24
timeFrame: up to week 24
Secondary outcomes:
measure: Change in the relative counts of lymphocytic infiltrate (stained for CD45) at week24
timeFrame: up to 24 weeks
measure: Change in B/B+T cell ratio at week 24
timeFrame: up to 24 weeks
measure: Incidence and severity of TEAEs, AESIs, and SAEs by SOC and PT
timeFrame: up to 35 weeks
measure: Clinically significant changes in vital sign measurements
timeFrame: Up to 35 weeks
measure: Clinically significant changes in ECG results
timeFrame: Up to 35 weeks
measure: Clinically significant changes in clinical laboratory safety evaluations
timeFrame: Up to 35 weeks
measure: Proportion of participants with minimal clinically important improvement in ESSDAI: improvement of ≥3 points in ESSDAI score at week 24
timeFrame: up to 24 weeks
measure: Proportion of participants with low disease activity: ESSDAI score of <5 at week 24
timeFrame: up to 24 weeks
measure: Proportion of participants with minimal clinically important improvement in clinESSDAI: improvement of ≥3 points in clinESSDAI score at week 24
timeFrame: up to 24 weeks
measure: Proportion of participants with low disease activity: clinESSDAI score of <5 at week 24
timeFrame: up to 24 weeks
measure: Proportion of participants with minimal clinically important improvement in ESSPRI: decrease of 1 point or ≥15% at week 24
timeFrame: up to 24 weeks
measure: Change in ESSDAI score at week 24
timeFrame: up to 24 weeks
measure: Change in clinESSDAI score at week 24
timeFrame: up to 24 weeks
measure: Change in ESSPRI score at week 24
timeFrame: up to 24 weeks
measure: Proportion of participants with STAR score of ≥5 at week 24
timeFrame: up to 24 weeks
Other outcomes:
Locations:
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Debrecen
country: Hungary
facility: Debreceni Egyetem
city: Szekesfehervar
country: Hungary
facility: Vita Verum Medical Egeszsegugyi Szolgaltato Bt.
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen , Dept of Rheumatology and Clinical Immunology
city: Elblag
country: Poland
facility: Ambulatorium Barbara Bazela
city: Grodzisk Mazowiecki
country: Poland
facility: MCBK Iwona Czajkowska Anna Podrażka- Szczepaniak S.C.
city: Krakow
country: Poland
facility: FutureMeds Krakow
city: Kraków
country: Poland
facility: Centrum Medyczne Plejady
city: Lublin
country: Poland
facility: ETG Lublin
city: Lublin
country: Poland
facility: Reumed Spolka z o.o.
city: Poznan
country: Poland
facility: Clinical Research Center Spółka z ograniczoną odpowiedzialnością Medic-R Sp.k.
city: Skórzewo
country: Poland
facility: Centrum Medyczne Pratia Poznan
city: Warsaw
country: Poland
facility: Centrum Medyczne Reuma Park
city: Warsaw
country: Poland
facility: FutureMeds Targowek
city: Warszawa
country: Poland
facility: MICS Centrum Medyczne Warszawa
city: Warszawa
country: Poland
facility: KO-Med - Centrum Badań Medycznych NIGRiR
city: Wroclaw
country: Poland
facility: FutureMeds Wroclaw
Study officials:
References: