Open Label Two-Arm Study to Evaluate Rilzabrutinib in IgG4-Related Disease Participants
- Conditions
- Immunoglobulin G4 Related Disease
- Interventions
- Drug: Glucocorticoids
- Registration Number
- NCT04520451
- Lead Sponsor
- Principia Biopharma, a Sanofi Company
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 27
- 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
- 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/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Rilzabrutinib + glucocorticoids Glucocorticoids Rilzabrutinib tablets, 400 mg twice daily from Week 0 to Week 12 plus glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 2 weeks on study) Glucocorticoids Glucocorticoids Glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 12 weeks on study) Rilzabrutinib + glucocorticoids rilzabrutinib Rilzabrutinib tablets, 400 mg twice daily from Week 0 to Week 12 plus glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 2 weeks on study)
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Proportion of participants with reduction from baseline IgG4-RD RI activity score by ≥2 points over time Up to 64 weeks Proportion of patients with an IgG4-RD RI activity score = 0 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
Proportion of participants achieving reduction in baseline serum IgG4 level of 10% over time Up to 64 weeks Change from baseline in IgG4-RD RI over time From baseline up to 64 weeks Change from baseline over time in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI over time From baseline up to 64 weeks
Trial Locations
- Locations (9)
Investigational Site Number 84016
🇺🇸Boston, Massachusetts, United States
Investigational Site Number 25013
🇫🇷Marseille, France
Investigational Site Number 84019
🇺🇸Stanford, California, United States
Investigational Site Number 84018
🇺🇸Detroit, Michigan, United States
Investigational Site Number 84036
🇺🇸Portland, Oregon, United States
Investigational Site Number 84030
🇺🇸New York, New York, United States
Investigational Site Number 12403
🇨🇦Vancouver, Canada
Investigational Site Number 38016
🇮🇹Milan, Italy
Investigational Site Number 72415
🇪🇸Barcelona, Spain