Immunoglobulin G4 Related Disease
Conditions
Keywords
+IgG4, glucocorticoid, IgG4-RD, BTK, Bruton's
Brief summary
This is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: rilzabrutinib with glucocorticoids and (2) Active Comparator: glucocorticoids only.
Detailed description
4 weeks of screening, 12 weeks of main treatment, 12 weeks of cross-over (for GC-only group), 40 weeks of extension treatment and 4-week follow-up.
Interventions
DRUGrilzabrutinib
oral tablet
DRUGGlucocorticoids
oral tablet or capsule
Sponsors
Massachusetts General Hospital
Principia Biopharma, a Sanofi Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Be male or female with age ≥18 years. * Have a clinical diagnosis of IgG4-RD. * Be willing to taper off an equivalent prednisone dose of between 20-40 mg/day in 2 weeks. Key
Exclusion criteria
* Currently or within 6 months of screening taking rituximab, other B-cell depleting agents, or alkylating agents unless B cell concentrations have been demonstrated by flow cytometry to return to normal values (defined as 5 cells per cubic mm). * History of solid organ transplant * Positive at Screening for HIV, hepatitis B, hepatitis C, or TB * Female patients who are pregnant or nursing. * NOTE: Other Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants who are without disease flare following the first dose of rilzabrutinib until the end of treatment | Up to 64 weeks | Disease flare is defined as an increase in IgG4-RD responder index (RI) \>2 or initiation of rescue treatment. |
| Incidence of SAE, AE leading to discontinuation and possible glucocorticoid-related AE | Up to 68 weeks | — |
| Number of participants with Potentially clinically significant abnormalities (PCSAs) for clinical laboratory tests, vital signs and ECG | Up to 68 weeks | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants achieving reduction in baseline serum IgG4 level of 10% over time | Up to 64 weeks | — |
| Proportion of participants with reduction from baseline IgG4-RD RI activity score by ≥2 points over time | At Week 52 | — |
| Change from baseline in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI over time | From baseline up to 64 weeks | — |
| Change from baseline in IgG4-RD RI over time | From baseline up to 64 weeks | — |
| Proportion of patients with an IgG4-RD RI activity score = 0 at over time | Up to 64 weeks | — |
| Level and change from baseline of each subclass of the serological markers over time | Up to 64 weeks | serum protein electrophoresis to determine IgG subclasses and complement factors C3 and C4 |
Countries
Canada, France, Italy, Spain, United States
Outcome results
None listed