A Phase 2 Multicenter Study of TL-895 in Subjects with Myelofibrosis, Indolent Systemic Mastocytosis, Monoclonal Mast Cell Activation Syndrome, or Non-Monoclonal Mast Cell Activation Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 150
- 试验地点
- 30
- 主要终点
- To determine the recommended phase 2 dose
研究概览
简要总结
Part A: Cohorts 1-4: To determine the recommended phase 2 dose and schedule of TL-895 in each cohort. Part A: Cohort 5: To determine the recommended phase 3 dose of TL-895. Part A: Cohort 6: To determine the recommended phase 3 dose of TL-895. Part B: Cohorts 1-4: Improvement in Total Symptom Score at Week 24 Part B: Cohort 5: To assess changes in ISM symptoms after treatment with TL-895. Part B Cohort 6: To assess changes in MCAS symptoms after treatment with TL-895.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 主要目的
- Cohort 6
- 盲法
- Double (Carer, Investigator, Subject)
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Cohorts 1-3: Adults ≥18 years of age
- •Cohorts 1-3: Confirmed diagnosis of PMF, post-PV MF, or post-ET MF, as assessed by treating physician according to the World Health Organization (WHO) criteria
- •Cohorts 1-3: Eastern Cooperative Oncology Group (ECOG) performance status of ≤2
- •Cohorts 1-3: Adequate hematologic, hepatic, and renal functions
- •Cohorts 1-3: MF symptoms as defined by having at least 2 symptoms with an average baseline (Day -7 to Day -1) score of at least 1 for each of the 2 symptoms per MFSAF v4.0
- •Cohort 3 only: Ineligibility for JAKi treatment with a platelet count of ≥ 25 and < 50 x 10^9/L
- •Cohort 5: Confirmed diagnosis of ISM as defined by WHO diagnostic criteria based on review of bone marrow biopsy pathology report results
- •Cohort 5: Subject must have moderate-to-severe symptoms
- •Cohort 6: Confirmed diagnosis of MCAS
排除标准
- •Cohorts 1-3, 5: Prior treatment with any BTK or BMX inhibitors
- •Cohorts 1-3: Prior treatment with JAKi within 28 days prior to study treatment
- •Cohorts 1-3: Prior splenectomy or splenic irradiation within 24 weeks prior to first dose of study treatment
- •Cohort 5: Prior treatment with Avapritinib, bezuclastinib, or BLU-263/elenestinib
- •Cohort 5: Diagnosis with another myeloproliferative disorder
- •Cohort 6: Treatment with certain prior therapies as defined in protocol
- •Cohort 6: A current diagnosis of cutaneous or systemic mastocytosis as defined by WHO criteria
结局指标
主要结局
To determine the recommended phase 2 dose
To determine the recommended phase 2 dose
To determine the recommended phase 3 dose
To determine the recommended phase 3 dose
Improvement in total symptom score at Week 24
Improvement in total symptom score at Week 24
Assess changes in ISM symptoms
Assess changes in ISM symptoms
Assess changes in MCAS symptoms
Assess changes in MCAS symptoms
次要结局
未报告次要终点
研究者
Regulatory Affairs
Scientific
Telios Pharma Inc.