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

NCT ID: NCT04520451

Last Updated: 2025-10-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 27 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-08-22
• Study Completion Date: 2024-10-15

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.

Conditions

Immunoglobulin G4 Related Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Rilzabrutinib + 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)

Group Type: EXPERIMENTAL

rilzabrutinib

Intervention Type: DRUG

oral tablet

Glucocorticoids

Intervention Type: DRUG

oral tablet or capsule

Glucocorticoids

Glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 12 weeks on study)

Group Type: ACTIVE_COMPARATOR

Glucocorticoids

Intervention Type: DRUG

oral tablet or capsule

Interventions

rilzabrutinib

oral tablet

Intervention Type: DRUG

Glucocorticoids

oral tablet or capsule

Intervention Type: DRUG

Other Intervention Names

PRN1008/SAR444671

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Massachusetts General Hospital

OTHER

Sponsor Role: collaborator

Principia Biopharma, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

New York, New York, United States

Investigational Site Number 84036

Portland, Oregon, United States

Investigational Site Number 12403

Vancouver, , Canada

Investigational Site Number 25013

Marseille, , France

Investigational Site Number 38016

Milan, , Italy

Investigational Site Number 72415

Barcelona, , Spain

Countries

United States; Canada; France; Italy; Spain

References

Wallace ZS, Zhang Y, Perugino CA, Naden R, Choi HK, Stone JH; ACR/EULAR IgG4-RD Classification Criteria Committee. Clinical phenotypes of IgG4-related disease: an analysis of two international cross-sectional cohorts. Ann Rheum Dis. 2019 Mar;78(3):406-412. doi: 10.1136/annrheumdis-2018-214603. Epub 2019 Jan 5.

Reference Type: BACKGROUND

PMID: 30612117 (View on PubMed)

Other Identifiers

PRN1008-017

Identifier Type: OTHER

Identifier Source: secondary_id

U1111-1260-3972

Identifier Type: REGISTRY

Identifier Source: secondary_id

2022-002959-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ACT17125

Identifier Type: -

Identifier Source: org_study_id