This is a Phase 3, parallel group, 2-arm, randomized, double blind, placebo-controlled, 52-week treatment study to assess the efficacy and safety of rilzabrutinib as a treatment for adult patients with active IgG4-RD.
The purpose of this study is to measure time to adjudicated IgG4-RD clinical disease flare, and other relevant efficacy endpoints including flare-free rate, control of IgG4-RD disease activity, use of GC rescue and safety parameters such as treatment-emergent adverse events, clinical laboratory values and electrocardiograms (ECG) in participants aged 18 years and above, diagnosed with IgG4-RD and treated with rilzabrutinib tablets over a 52-week placebo-controlled period.
Study details include:
The study duration will be up to 60 weeks, including a Screening period of 4 to 6 weeks, a 52-week double blind treatment period, and 2 weeks of follow up (plus an optional OLE of 108 weeks).
The number of visits will be 16 (plus an optional 9 visits during the OLE).
Participants must have a clinical diagnosis of IgG4-RD confirmed by the Adjudication Committee.
Participants meeting Step 1 Entry criteria of 2019 ACR/EULAR classification criteria for IgG4-RD and Total inclusion points are ≥20
Participants with active disease in at least 1 organ system, excluding lymph nodes, with active disease defined by an IgG4-RD Responder Index organ/site activity score ≥2 based on the manifestations of disease activity in the last 28 days.
Participants with history or current involvement of at least 1 organ/site (excluding lymph nodes) affected with IgG4-RD.
Meet any Step 2 Exclusion criteria from the 2019 ACR/EULAR classification criteria for IgG4-RD.
History of retroperitoneal fibrosis, sclerosing mesenteritis, fibrosing mediastinitis, or other overwhelmingly fibrotic expression of IgG4-RD that is the sole disease manifestation.
Active malignancy or history of malignancy within 5 years before Day 1, except completely treated in situ carcinoma of the cervix, completely treated, and resolved nonmetastatic squamous or basal cell carcinoma of the skin.
Known or suspected immunodeficiency, including history of invasive opportunistic infections (eg, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystosis, and aspergillosis) despite infection resolution, or otherwise recurrent infections of abnormal frequency or prolonged duration suggesting an immune compromised status, as judged by the Investigator.
Parallel
Rilzabrutinib
DrugPharmaceutical form:Tablet-Route of administration:Oral
Placebo
DrugPharmaceutical form:Tablet-Route of administration:Oral
Glucocorticoid
DrugPharmaceutical form:Tablet, solution, suspension formulations according to local standard practices-Route of administration:Oral
Time to first adjudicated clinical disease flare treated by the investigator during the Blinded Treatment period
The Adjudication Committee will include internationally recognized independent experts in diagnosis and management of patients with IgG4-RD, blinded to participant, investigator, and site identifiers
Proportion of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids and immunomodulators
Proportion of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids
Annualized rate of clinical disease flares
Change in IgG4-RD RI total activity scores from baseline to Week 52
