Open label two-arm study to evaluate rilzabrutinib in IgG4-related disease patients

Phase 1

• Conditions: IgG4-related disease; MedDRA version: 20.0Level: PTClassification code 10077271Term: Immunoglobulin G4 related diseaseSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2022-002959-18-ES
• Lead Sponsor: Principia Biopharma Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-12-20
• Last Update Posted: 26 February 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 25

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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the safety and the ability of daily oral administration of rilzabrutinib to maintain glucocorticoid-free remission in participants with IgG4-RD for at least 24 weeks;Secondary Objective: - To evaluate the effect of rilzabrutinib on IgG4-RD disease activity over time<br>- To evaluate the effect of rilzabrutinib on specific protein biomarkers over time;Primary end point(s): 1. Proportion of participants who are without disease flare for at least 24 consecutive weeks following the first dose of rilzabrutinib. Disease flare is defined as an increase from screening IgG4-RD responder index (RI) >2 or initiation of rescue treatment. <br>2. Incidence of SAE, AE leading to discontinuation and possible glucocorticoid-related AE. <br>3. Number of participants with Potentially clinically significant abnormalities (PCSAs) for clinical laboratory tests, vital signs and ECG;Timepoint(s) of evaluation of this end point: 1. At Week 52<br>2, 3. Up to 68 weeks

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1. Proportion of participants with reduction from baseline IgG4-RD RI activity score =2 points at Week 12<br>2. Proportion of patients with an IgG4-RD RI activity score = 0 at Week 12<br>3. Level and change from baseline of each subclass of the serological markers<br>4. Proportion of participants achieving reduction in baseline serum IgG4 level of 10% at 12 weeks<br>5. Change from baseline in IgG4-RD RI over time<br>6. Proportion of participants with no disease flares between Week 4 and Week 12, and between Week 12 and Week 52 (or the end of the treatment extension period) among the participants who have treatment extension<br>7. Change from baseline over time in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI;Timepoint(s) of evaluation of this end point: 1, 2, 3. At Week 52<br>4. At Week 12<br>5, 7. From baseline to Week 52<br>6. Until Week 52