An Observational Study in Patients With Indolent Systemic Mastocytosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 150
- 试验地点
- 7
- 主要终点
- Baseline Demographics
研究概览
简要总结
This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings. The study will describe the demographic and clinical characteristics of ISM participants, including anaphylaxis and bone manifestations in ISM. Quality of life and disease control will be assessed through participant questionnaires. The study will also evaluate real world ISM treatment management, including use of avapritinib.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Male or female adult participants (≥ 18 years of age) with a diagnosis of ISM according to the World Health Organization (WHO) diagnostic criteria
- •Participant is currently being treated or plans to be treated with symptom-directed therapies and/or avapritinib for ISM.
排除标准
- •Participants with advanced systemic mastocytosis (AdvSM) or another associated hematologic neoplasm
- •Participants with smoldering systemic mastocytosis
- •Ongoing participation in interventional studies in systemic mastocytosis (SM) at the time of enrollment
- •Participants currently receiving treatment with a KIT inhibitor other than avapritinib at the time of enrollment.
研究组 & 干预措施
Participants With ISM
Patients who are currently being treated or plan to be treated for ISM
结局指标
主要结局
Baseline Demographics
时间窗: Baseline (Month 1)
Change From Baseline in Indolent Systemic Mastocytosis (ISM) Symptom-Directed Therapies
时间窗: Baseline up to Month 61
Change From Baseline in Patient-Reported Outcomes Scores
时间窗: Baseline up to Month 61
Change From Baseline in Serum KIT D816V Variant Allele Frequency (VAF)
时间窗: Baseline up to Month 61
Change From Baseline in Serum Tryptase
时间窗: Baseline up to Month 61
次要结局
未报告次要终点