An Open Label, Two-Arm Study to Evaluate the Effect of Rilzabrutinib (PRN1008/SAR444671) on Safety and Disease Activity in ParticipantsWith IgG4-Related Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 27
- 试验地点
- 9
- 主要终点
- Proportion of participants who are without disease flare following the first dose of rilzabrutinib until the end of treatment
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
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)
干预措施: rilzabrutinib (Drug)
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)
干预措施: Glucocorticoids (Drug)
Glucocorticoids
Glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 12 weeks on study)
干预措施: Glucocorticoids (Drug)
结局指标
主要结局
Proportion of participants who are without disease flare following the first dose of rilzabrutinib until the end of treatment
时间窗: 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
时间窗: Up to 68 weeks
Number of participants with Potentially clinically significant abnormalities (PCSAs) for clinical laboratory tests, vital signs and ECG
时间窗: Up to 68 weeks
次要结局
- Proportion of participants with reduction from baseline IgG4-RD RI activity score by ≥2 points over time(At Week 52)
- Proportion of patients with an IgG4-RD RI activity score = 0 at over time(Up to 64 weeks)
- Level and change from baseline of each subclass of the serological markers over time(Up to 64 weeks)
- 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 in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI over time(From baseline up to 64 weeks)