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

Principia Biopharma

Status and phase: Completed; Phase 2

Conditions: Immunoglobulin G4 Related Disease

Treatments: Drug: Glucocorticoids; Drug: rilzabrutinib

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04520451

U1111-1260-3972 (Registry Identifier)

2022-002959-18 (EudraCT Number)

PRN1008-017 (Other Identifier)

ACT17125

Details and patient eligibility

About

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.

Full 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.

Enrollment: 27 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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27 participants in 2 patient groups

Rilzabrutinib + glucocorticoids

Experimental group

Description:

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)

Treatment:

Drug: rilzabrutinib

Drug: Glucocorticoids

Glucocorticoids

Active Comparator group

Description:

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

Treatment:

Drug: Glucocorticoids