Open Label Two-Arm Study to Evaluate Rilzabrutinib in IgG4-Related Disease Participants

Phase 2CompletedNCT04520451

Principia Biopharma, a Sanofi Company27 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Immunoglobulin G4 Related Disease

Interventions

rilzabrutinibDRUG

oral tablet

GlucocorticoidsDRUG

oral tablet or capsule

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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/Exclusion criteria may apply.

Locations (9)

Investigational Site Number 84019

Stanford, California, United States

Investigational Site Number 84016

Boston, Massachusetts, United States

Investigational Site Number 84018

Detroit, Michigan, United States

Investigational Site Number 84030

Outcomes

Primary Outcomes

Proportion of participants who are without disease flare following the first dose of rilzabrutinib until the end of treatment

Disease flare is defined as an increase in IgG4-RD responder index (RI) \>2 or initiation of rescue treatment.

Time frame: Up to 64 weeks

Incidence of SAE, AE leading to discontinuation and possible glucocorticoid-related AE

Time frame: Up to 68 weeks

Number of participants with Potentially clinically significant abnormalities (PCSAs) for clinical laboratory tests, vital signs and ECG

Time frame: Up to 68 weeks

Secondary Outcomes

Proportion of participants with reduction from baseline IgG4-RD RI activity score by ≥2 points over time

Time frame: At Week 52

Proportion of patients with an IgG4-RD RI activity score = 0 at over time

Time frame: Up to 64 weeks

Level and change from baseline of each subclass of the serological markers over time

serum protein electrophoresis to determine IgG subclasses and complement factors C3 and C4

Time frame: Up to 64 weeks

Proportion of participants achieving reduction in baseline serum IgG4 level of 10% over time

Time frame: Up to 64 weeks

Change from baseline in IgG4-RD RI over time

Time frame: From baseline up to 64 weeks

Change from baseline in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI over time

Data from ClinicalTrials.gov

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