autoimmune disease exocrinopathy
Conditions
Interventions
All participants will receive efgartigimod IV 10 mg/kg or placebo once weekly
for 24 weeks during the treatment period.
Sponsors
IQVIA RDS Netherlands B.V.
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the study only if all of the following criteria apply: 1. Is at least the legal age of consent for clinical trials when signing the informed consent form 2. Is capable of providing signed informed consent, as described in Section 10.1.3 of the protocol, and complying with protocol requirements 3. Agrees to use contraceptive measures consistent with local regulations and the following: a. Male participants: refer to Section 10.4.2.2 of the protocol. b. WOCBP (defined in Section 10.4.1) must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before receiving IMP. Contraceptive requirements are provided in Section 10.4 of the protocol. 4. Meets the following criteria at screening: a. ACR/EULAR 2016 pSS who met criteria =5 c. Anti Ro/SS-A positive d. Residual salivary flow (UWSF rate >0 and/or SWSF rate >0.10)
Exclusion criteria
Exclusion criteria: Participants will be excluded from the study if any of the following criteria apply: 1. 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 2. 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: a. Basal cell or squamous cell skin cancer b. Carcinoma in situ of the cervix c. Carcinoma in situ of the breast d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b) 3. Clinically significant uncontrolled active acute or chronic bacterial, viral, or fungal infection 4. Positive serum test at screening for an active infection with any of the following: a. HBV that is indicative of an acute or chronic infection, unless associated with a negative HBsAg or negative HBV DNA test b. HCV based on HCV antibody assay unless a negative RNA test is available c. HIV based on test results of a CD4 count of =1 dose of IMP 11. Total IgG of <4 g/L at screening 12. Secondary Sjögren*s syndrome overlap syndromes where another confirmed autoimmune rheumatic or systemic inflammatory condition (eg, rheumatoid arthritis, systemic lupus erythematosus, scleroderma, inflammatory bowel disease) is the primary diagnosis 13. Positive SARS-CoV-2 PCR test at screening 14. Any severe systemic pSS manifestation that may put the participant at undue risk based on the investigator*s opinion Note: Medications cited in exclusion criteria 15-22 are also prohibited during the screening period. 15. IVIg, SCIg, or PLEX <12 weeks before screening 16. Live or live-attenuated vaccine <4 weeks before screening 17. Pilocarpine and/or any other pharmacological stimulant for salivary and lacrimal glands that is not at a stable dose in the 4 weeks prior to screening or is started<= 4 weeks from screening 18. Anticholinergic agents which are not at a stable dose 4 weeks prior to screening or during screening 19. Corticosteroids: a. Intramuscular or IV corticosteroids <= 4 weeks from screening b. Oral corticosteroids if above 10 mg, or if not at a stable dose <= 4 weeks from screening c. Intra-articular steroids <= 4 weeks from screening d. Use of topical ophthalmic steroids is prohibited<b
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| • Proportion of CRESS responders on >=3 of 5 items at week 24 (refer to Section 8.2.1 of the protocol). The 5 items are: * Systemic disease activity: clinESSDAI * Patient-reported symptoms: ESSPRI * Tear gland function: Schirmer*s test and OSS * Salivary gland function: UWSF rate and SGUS * Serology (serum IgG and/or RF) | — |
Secondary
| Measure | Time frame |
|---|---|
| • Change in the relative counts of lymphocytic infiltrate (stained for CD45) at week 24 • Change in B/B+T cell ratio at week 24 • Incidence and severity of TEAEs, AESIs, and SAEs by SOC and PT • Changes in vital sign measurements, ECG results, and clinical laboratory safety evaluations • Proportion of participants with minimal clinically important improvement in ESSDAI: improvement of >=3 points in ESSDAI score at week 24 • Proportion of participants with low disease activity: ESSDAI score of =3 points in clinESSDAI score at week 24 • Proportion of participants with low disease activity: clinESSDAI score of =15% at week 24 • Change in ESSDAI score at week 24 • Change in clinESSDAI score at week 24 • Change in ESSPRI score at week 24 • Proportion of participants with STAR score of >=5 at week 24 • Efgartigimod serum concentration-time profile • Values, changes from baseline, and percent reduction from baseline in total IgG levels in serum • Values, changes from baseline, and percent reduction from baseline in autoantibodies in serum: - Anti-Ro/SS-A - Anti-La/SS-B • Incidence and prevalence of ADA against efgartigimod in serum | — |
Countries
Netherlands
Outcome results
None listed