Clinical trial NCT06203457: Open-Label Extension Study to Evaluate the Safety of Efgartigimod in Adult Patients With Primary Sjögren's Syndrome
Official title: Open-Label Extension Study to Evaluate the Safety of Efgartigimod in Adult Patients With Primary Sjögren's Syndrome (pSS) Who Complete Qualifying Efgartigimod pSS Studies
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: 2024-01-12
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Efgartigimod contributes to successfully treat pSS and has the potential to improve disease manifestations by the reduction of IgG autoantibodies in pSS. This open-label extension study will evaluate the long-term safety of efgartigimod in participants with pSS who have completed the treatment period of the qualifying efgartigimod studies (including ARGX-113-2106).

Detailed description: ARGX-113-2211 is a long-term, single-arm, open-label, multicenter extension study of the pSS-qualifying efgartigimod studies designed to evaluate the long-term safety of efgartigimod in adult patients with pSS. Participants will be enrolled from both active and placebo arms of qualifying efgartigimod studies and receive efgartigimod 10 mg/kg over 48 weeks in the extension study without knowledge of their treatment assignment in the qualifying study. Eligible participants must have completed the treatment period of the qualifying study and must not have permanently discontinued the IMP in that study.

Eligibility criteria: Inclusion Criteria:

* Is at least the legal age of consent for clinical trials when signing the ICF
* Is capable of providing signed informed consent and complying with protocol requirements
* Agrees to use contraceptive measures consistent with local regulations and the following: WOCBP must have a negative urine pregnancy test at baseline before receiving IMP
* Has completed the qualifying efgartigimod pSS studies and agrees to continue study drug treatment without interruption in the extension study

Exclusion Criteria:

* Clinically significant disease (including newly diagnosed malignancy or cardiovascular disease) 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
* Pregnant or intention to become pregnant during the study
* Any severe systemic pSS manifestation that may put the participant at undue risk based on the investigator's opinion

Primary outcomes:
measure: Number of participants with (serious) adverse events (of special interest)
timeFrame: up to week 48
measure: Number of participants with with abnormal laboratory test results or vital signs
timeFrame: up to 48 weeks
Secondary outcomes:
measure: : Proportion of CRESS (Composite of Relevant Endpoints for Sjögren's Syndrome) responders on ≥ 3 of 5 items at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Proportion of participants with minimal clinically important improvement from baseline in ESSDAI: improvement of ≥ 3 points in ESSDAI score at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Proportion of participants with low disease activity: ESSDAI score of < 5 at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Proportion of participants with minimal clinically important improvement from baseline in clinESSDAI: improvement of ≥ 3 points in clinESSDAI score at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Proportion of participants with low disease activity: clinESSDAI score of < 5 at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Proportion of participants with minimal clinically important improvement from baseline in ESSPRI: decrease of ≥ 1 point or ≥ 15% at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Change from baseline in ESSDAI score at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Change from baseline in clinESSDAI score at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Change from baseline in ESSPRI score at weeks 24 and 48
timeFrame: up to 48 weeks
measure: Proportion of STAR responders (score of ≥ 5) at weeks 24 and 48 when compared to baseline
timeFrame: up to 48 weeks
measure: Percent reduction from baseline in total IgG levels in serum over the 48-week treatment period
timeFrame: up to 48 weeks
measure: Percent reduction from baseline in autoantibodies (Anti-Ro/SS A and Anti-La/SS B) in serum over the 48-week treatment period
timeFrame: up to 48 weeks
measure: Efgartigimod serum concentrations over the 48-week treatment period
timeFrame: up to 48 weeks
measure: Incidence of ADA against efgartigimod over the 48-week treatment period
timeFrame: up to 48 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: Grodzisk Mazowiecki
country: Poland
facility: MCBK SC
city: Krakow
country: Poland
facility: Centrum Medyczne Plejady
city: Poznan
country: Poland
facility: Clinical Research Center Spółka z ograniczoną odpowiedzialnością Medic-R Sp.k.
city: Skorzewo
country: Poland
facility: Centrum Medyczne Pratia Poznan
city: Warsaw
country: Poland
facility: Klinika Reuma Park Sp zoo Sp K
city: Warszawa
country: Poland
facility: MICS Centrum Medyczne Warszawa
city: Warszawa
country: Poland
facility: Narodowy Instytut Geriatrii
city: Wroclaw
country: Poland
facility: FutureMeds sp zoo
Study officials:
References: