A Phase 2 Study of Ibrutinib in Advanced Systemic Mastocytosis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Overall Response Rate (ORR)
研究概览
简要总结
This phase 2 trial studies ibrutinib to see how well it works in treating patients with systemic (affecting the entire body) mastocytosis that has spread to other parts of the body and usually cannot be cured or controlled with treatment (advanced). Systemic mastocytosis is a disease in which too many mast cells (a type of immune system cell) are found throughout the body. Mast cells give off chemicals such as histamine that can cause flushing (a hot, red face), itching, abdominal cramps, muscle pain, nausea, vomiting, diarrhea, low blood pressure, and shock. Ibrutinib may stop the growth of mast cells by blocking some of the enzymes needed for cell growth.
详细描述
PRIMARY OBJECTIVE:
Evaluate the response rate to ibrutinib in patients with advanced systemic mastocytosis (SM) (aggressive systemic mastocytosis [ASM] or mast cell leukemia [MCL], or SM-associated hematologic non-mast cell disorder [AHNMD]) by the end of 6 cycles (6 months).
SECONDARY OBJECTIVES:
- Evaluate the tolerability and safety profile of ibrutinib in patients with advanced SM.
- Evaluate the pharmacokinetic (PK) profile of ibrutinib in a subset of patients with advanced SM.
- Evaluate changes in histopathology (blood and bone marrow) of patients with advanced SM in response to ibrutinib therapy.
- Evaluate changes in mastocytosis related symptom scores and quality-of-life (QOL) using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF).
- Evaluate the duration of response (DoR) and time to response (TTR).
- Evaluate progression-free survival (PFS) and overall survival.
OUTLINE:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non 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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Ibrutinib 420 mg/day
Participants receive ibrutinib daily on days 1 to 28, at 420 mg/day in 28-day cycles
干预措施: Ibrutinib (Drug)
Ibrutinib 560 mg/day
Participants receive ibrutinib daily on days 1 to 28, at 560 mg/day in 28-day cycles
干预措施: Ibrutinib (Drug)
结局指标
主要结局
Overall Response Rate (ORR)
时间窗: Up to 6 months
Overall response rate (ORR) is reported as the sum of the rates of participants achieving complete remission (CR), partial remission (PR), \& clinical improvement (CI). A clinical response is a response with duration of ≥ 12 weeks. CR is defined as all 4 criteria: * No presence of compact neoplastic mast cell aggregates * Serum tryptase level \< 20 ng/mL * Peripheral blood count remission defined as absolute neutrophil count (ANC) ≥1 x 10e9/L + normal differential, Hb ≥11 g/dL, \& platelet count ≥100x10e9/L * Complete resolution of palpable hepatosplenomegaly \& all biopsy-proven or suspected SM-related organ damage PR is defined as all 3 criteria with response duration ≥12 weeks, that is not CR or progressive disease: * ≥ 50% reduction in neoplastic mast cells * Serum tryptase level reduced ≥50% * Resolution of 1+ biopsy-proven or suspected systemic mastocytosis (SM)-related organ damage findings CI is defined as any improvement in any of the above measures.
次要结局
- Number of Participants With Adverse Events(30 days)
- Ibrutinib Pharmacokinetics (PK)(28 days)
- Change of Mast Cell Burden(2 years)
- Serum Tryptase Levels(2 years)
- Total Symptom Score (TSS)(30 days)
- Change in Quality of Life (QoL)(30 days)
- Duration of Response (DoR)(2 years)
- Time-to-Response (TTR)(2 years)
- Progression-free Survival (PFS)(2 years)
- Overall Survival (OS)(26 months)
研究者
Jason Robert Gotlib
Principal Investigator
Stanford University