NCT07190196招募中3 期
A Randomized, Phase 3, Double-blind, 52-week Study to Evaluate the Efficacy and Safety of Rilzabrutinib (SAR444671) Compared to Placebo in Adult Participants With Active IgG4-related Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Sanofi
- 入组人数
- 124
- 试验地点
- 92
- 主要终点
- Time to first adjudicated clinical disease flare treated by the investigator during the Blinded Treatment period
研究概览
简要总结
This is a Phase 3, parallel group, 2-arm, randomized, double blind, placebo-controlled, 52-week treatment study to assess the efficacy and safety of rilzabrutinib as a treatment for adult patients with active IgG4-RD.
The purpose of this study is to measure time to adjudicated IgG4-RD clinical disease flare, and other relevant efficacy endpoints including flare-free rate, control of IgG4-RD disease activity, use of GC rescue and safety parameters such as treatment-emergent adverse events, clinical laboratory values and electrocardiograms (ECG) in participants aged 18 years and above, diagnosed with IgG4-RD and treated with rilzabrutinib tablets over a 52-week placebo-controlled period.
Study details include:
The study duration will be up to 60 weeks, including a Screening period of 4 to 6 weeks, a 52-week double blind treatment period, and 2 weeks of follow up (plus an optional OLE of 108 weeks).
The number of visits will be 16 (plus an optional 9 visits during the OLE).
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Participants must have a clinical diagnosis of IgG4-RD confirmed by the Adjudication Committee.
- •Participants meeting Step 1 Entry criteria of 2019 ACR/EULAR classification criteria for IgG4-RD and Total inclusion points are ≥20
- •Participants with active disease in at least 1 organ system, excluding lymph nodes, with active disease defined by an IgG4-RD Responder Index organ/site activity score ≥2 based on the manifestations of disease activity in the last 28 days.
- •Participants with history or current involvement of at least 1 organ/site (excluding lymph nodes) affected with IgG4-RD.
- •Participants with active IgG4-RD controlled for at least 2 weeks while on a stable dose of GC.
- •Participants willing to taper off GC after starting IMP.
- •Participants willing and able to participate in repeated study protocol mandated or clinically indicated imaging procedures to assess IgG4-RD such as computed tomography (CT), magnetic resonance imaging (MRI), positron emission tomography (PET), or ultrasound.
- •Participants who have an up-to-date vaccination status as per local guidelines. The last dose of live vaccines should be received at least 30 days before Day
- •Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
排除标准
- •Meet any Step 2 Exclusion criteria from the 2019 ACR/EULAR classification criteria for IgG4-RD.
- •History of retroperitoneal fibrosis, sclerosing mesenteritis, fibrosing mediastinitis, or other overwhelmingly fibrotic expression of IgG4-RD that is the sole disease manifestation.
- •Active malignancy or history of malignancy within 5 years before Day 1, except completely treated in situ carcinoma of the cervix, completely treated, and resolved nonmetastatic squamous or basal cell carcinoma of the skin.
- •Known or suspected immunodeficiency, including history of invasive opportunistic infections (eg, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystosis, and aspergillosis) despite infection resolution, or otherwise recurrent infections of abnormal frequency or prolonged duration suggesting an immune compromised status, as judged by the Investigator.
- •History of serious infections with the potential for recurrence (as judged by the Investigator), with less than 4 weeks interval between resolution of serious infection and first dose of study drug, or currently active moderate to severe infection at Screening (Grade 2 or higher).
- •Current or chronic history of liver disease unrelated to IgG4-RD.
- •Refractory nausea and vomiting, malabsorption, external biliary shunt, bariatric surgery, or significant bowel resection that would preclude adequate rilzabrutinib/placebo absorption.
- •History of solid organ transplant.
- •Planned major surgical procedure during the participation in this study.
- •History of drug abuse within the previous 12 months.
- •Alcoholism or excessive alcohol use, defined as regular consumption of more than approximately 3 standard drinks per day.
- •Prior participation in any rilzabrutinib studies or other BTK inhibitor studies.
- •History of treatment with an investigational drug within 6 months or 5 half-lives of the investigational drug, whichever is longer.
- •Laboratory abnormalities at the screening visit identified by the central laboratory The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
研究组 & 干预措施
Placebo
Placebo Comparator
Placebo
干预措施: Placebo (Drug)
Placebo
Placebo Comparator
Placebo
干预措施: Glucocorticoid (Drug)
Rilzabrutinib
Experimental
Rilzabrutinib
干预措施: Rilzabrutinib (Drug)
Rilzabrutinib
Experimental
Rilzabrutinib
干预措施: Glucocorticoid (Drug)
结局指标
主要结局
Time to first adjudicated clinical disease flare treated by the investigator during the Blinded Treatment period
时间窗: Until Week 52
The Adjudication Committee will include internationally recognized independent experts in diagnosis and management of patients with IgG4-RD, blinded to participant, investigator, and site identifiers
次要结局
- Annualized rate of clinical disease flares(At Week 52)
- Change in IgG4-RD RI total activity scores from baseline to Week 52(From baseline to Week 52)
- Change in IgG4-RD RI total activity scores from baseline to Week 12(From baseline to Week 12)
- Proportion of participants with reduction of ≥2 points from the baseline IgG4-RD RI total activity score(At Week 12, Week 24, and Week 52)
- Cumulative glucocorticoid dose for treatment of IgG4-RD(At Week 52)
- Proportion of participants with potentially clinically significant abnormalities in laboratory tests, vital signs, and electrocardiograms in the Safety Population(Until Week 160)
- Proportion of participants with TEAEs, AESIs, SAEs in the Safety Population(Until Week 160)
- Proportion of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids and immunomodulators(At Week 52)
- Proportion of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids(At Week 52)
- Proportion of participants in complete remission at Week 52(At Week 52)
- Percent change in IgG4-RD RI total activity scores from baseline to Week 52(From baseline to Week 52)
- Percent change in IgG4-RD RI total activity scores from baseline at Week 12(From baseline to Week 12)
- Cumulative glucocorticoid dose for treatment of IgG4-RD(At Week 52)
- Percentage of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids and immunomodulators(At Week 52)
- Percentage of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids(At Week 52)
- Annualized rate of clinical disease flares(At Week 52)
- Change in IgG4-RD RI total activity scores from baseline to Week 52(From baseline to Week 52)
- Percent change in IgG4-RD RI total activity scores form baseline to Week 52(From baseline to Week 52)
- Change in IgG4-RD RI total activity scores from baseline to Week 12(From baseline to Week 12)
- Proportion of participants with reduction of ≥2 points from the baseline IgG4-RD RI total activity score(At Week 12, Week 24, and Week 52)
- Proportion of participants in complete remission(At Week 52)
- Proportion of participants with potentially clinically significant abnormalities in laboratory tests, vital signs, and electrocardiograms in the Safety Population(Until Week 160)
- Proportion of participants with TEAEs, AESIs, SAEs in the Safety Population(Until Week 160)
研究者
研究点 (92)
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