A Phase 2, Open-Label, Monotherapy, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 47
- 试验地点
- 33
- 主要终点
- Percentage of Participants Who Achieved Complete Response (CR) as Determined by Investigator Assessment According to the Response Criteria for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of pemigatinib (INCB054828) in subjects with myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR) 1 rearrangement.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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
- •Documented lymphoid or myeloid neoplasm with 8p11 rearrangement known to lead to FGFR1 activation, based on standard diagnostic cytogenetic evaluation performed locally, before signing informed consent for this study.
- •Eligible subjects must:
- •Have relapsed after stem cell transplantation or after other disease modifying therapy, OR
- •Not be current candidates for stem cell transplantation or other disease modifying therapies.
- •Note: All relapsed/refractory subjects must have evidence of either cytogenetic or hematological disease and have no evidence of residual toxicity (eg, graft-versus-host disease requiring treatment).
- •Life expectancy ≥ 12 weeks.
- •Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
排除标准
- •Prior receipt of a selective FGFR inhibitor.
- •History and/or current evidence of ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, myocardia, or lung, except calcified lymph nodes and asymptomatic arterial or cartilage/tendon calcifications.
- •Current evidence of corneal disorder/keratopathy, including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis, as confirmed by ophthalmologic examination.
- •Use of any potent cytochrome P450 3A4 inhibitors or inducers within 14 days or 5 half-lives (whichever is shorter) before the first dose of study drug.
研究组 & 干预措施
Pemigatinib
干预措施: Pemigatinib (Drug)
结局指标
主要结局
Percentage of Participants Who Achieved Complete Response (CR) as Determined by Investigator Assessment According to the Response Criteria for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement
时间窗: up to 2513 days (120 21-day treatment cycles)
CR was defined as the presence of all of the following improvements: (1) bone marrow: ≤5% myeloblasts (including monocytic blast equivalent) and no lymphoblasts, with normal maturation of all cell lines, and return to age-adjusted normal cellularity; (2) osteomyelofibrosis absent or equal to "mild reticulin fibrosis" (Grade 1 or less fibrosis); (3) peripheral blood: white blood cells (WBC) ≤10 x 10\^9 cells/Liter (L); hemoglobin (Hgb) ≥11 grams per deciliter (g/dL); platelets ≥100 x 10\^9/L and ≤450 x 10\^9/L; neutrophils ≥1.0 x 10\^9/L; blasts = 0%; neutrophil precursors reduced to ≤2%; monocytes ≤1 x 10\^9/L; eosinophils ≤0.5 x 10\^9/L; (4) extramedullary disease: complete resolution of extramedullary disease present before therapy (e.g., lymphadenopathy), including palpable hepatosplenomegaly. Persistent low-level dysplasia was permitted given subjectivity of assignment of dysplasia. Response criteria by investigator assessment were the same for chronic phase (CP) and blast phase (BP).
次要结局
- Percentage of Participants Who Achieved a PCyR as Assessed by CRC Assessment(up to 2513 days (120 21-day treatment cycles))
- Duration of Response(up to 2513 days (120 21-day treatment cycles))
- Number of Participants With Any Treatment-emergent Adverse Event (TEAE)(up to 2543 days)
- Percentage of Participants Who Achieved a Partial Cytogenetic Response (PCyR) as Assessed by Local Analysis and Investigator Evaluation(up to 2513 days (120 21-day treatment cycles))
- Duration of Complete Response(up to 2513 days (120 21-day treatment cycles))
- Progression-free Survival (PFS)(up to 2513 days (120 21-day treatment cycles))
- Percentage of Participants Who Achieved a Best Overall Response of Complete Response (CR) or Partial Response (PR) as Determined by Investigator Assessment According to the Response Criteria for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement(up to 2513 days (120 21-day treatment cycles))
- Percentage of Participants Who Achieved a Complete Cytogenetic Response (CCyR) as Assessed by Local Analysis and Investigator Evaluation(up to 2513 days (120 21-day treatment cycles))
- Overall Survival(up to 2513 days (120 21-day treatment cycles))
- Number of Participants With Any ≥Grade 3 TEAE(up to 2543 days)