Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Have Received Alpelisib as Part of a Compassionate Use Program (EPIK-P1)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 57
- 试验地点
- 2
- 主要终点
- Percentage of Patients Responders and Non-responders at Week 24
研究概览
简要总结
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the cut-off date at a MAP site. The study cut-off date was 09-Mar-2020.
Patient-level data were abstracted from medical charts of all eligible patients at all participating sites. Study completion date refers to the last date data was extracted.
Information from patients treated with alpelisib was used to describe the efficacy and safety of alpelisib in PROS patients.
详细描述
The index date (baseline) is defined as the date of alpelisib initiation. The study period is the period from the index date up to the most recent data available at the time of the cut-off date.
The maximum follow-up was 187 weeks.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient (adult or pediatric) is ≥ 2 years of age
- •Patient has a physician confirmed/documented diagnosis of PROS
- •Patient has a documented evidence of a mutation in the PIK3CA gene
- •Patient's condition was assessed by the treating physician as severe or life threatening and treatment was deemed necessary
- •Patient has been treated with at least one dose of alpelisib, initiated on or before 23-Sep-2019 (i.e. at least 24 weeks before the cut-off date of the 09-Mar-2020)
- •Patient has medical chart history available during enrollment in the Novartis MAP
- •Patient (or parent/guardian in case of pediatric patient) consented to participate in the study (as required by local ethics regulations) Inclusion criteria for MAP enrollment (assessed at the time of alpelisib initiation)
排除标准
- 未提供
结局指标
主要结局
Percentage of Patients Responders and Non-responders at Week 24
时间窗: Index date and week 24 or 6 months (± 4 weeks)
Response is defined by achieving at least 20% reduction from index date in the sum of measurable target lesion volume (1 to 3 lesions, via central review of imaging scans), provided that none of the individual target lesions have ≥ 20% increase from index date and in absence of progression of non-target lesions and without new lesions.
次要结局
- Percent Change in the Sum of All Measurable Lesion Volume(Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187))
- Percent Change in the Sum of Measurable Target Lesion Volume(Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187))
- Percent Change in the Sum of All Measurable Non-target Lesion Volume(Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187))
- Number of Participants With Notable ECG Values.(Up to week 187)
- Mean Duration of Response (DoR)(Up to 187 weeks)
- Participants With Concomitant PROS-related Medications Over Time(Index date, week 24 and end of study)
- Number of Patients With Improvement in Most Frequent PROS-related Signs and Symptoms(Index date and week 24 or 6 months (± 4 weeks))
- Functional Status - Mobility Assessment(Up to 187 weeks)
- Change in Performance Status Score(Index date and week 24 or 6 months (± 4 weeks))
- Change From Index Date in Functional Status - School Status During the Study Period(Index date, week 24 and end of study (up to 187 weeks))
- Reasons for Discontinuation of Concomitant PROS-related Non-drug Treatments(Up to 187 weeks)
- Change From Index Date in Functional Status - Work Status(Index date, week 24 and end of study)
- Number of Patients With Grade 3/4 on Clinical Assessments - Clinical Chemistry(187 weeks)
- Number of PROS-related Completed Surgeries During the Study Period(Up to 187 weeks)
- Health Resource Utilization (HRU) - Number of Hospitalizations Per Patient(Up to 187 weeks)
- Number of Patients With Grade 3/4 on Laboratory Assessments: Hematology(Up to 187 weeks)
- Notable Vital Sign Values During the Study Period - Pediatric Patients(End of study (up to week 187))
- Notable Vital Sign Values During the Study Period - Adult Patients(End of study (up to week 187))
- Overview of Number of Patients With Adverse Events (AEs)(Up to 187 weeks)
- Growth and Development in Pediatric Population(Week 24 or 6 months (± 4 weeks))