A Phase II Study to Evaluate the Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 41
- 试验地点
- 7
- 主要终点
- Prospective period only: Proportion of participants with new or worsening grade ≥3 treatment emergent adverse events (AEs)
研究概览
简要总结
This is a prospective interventional Phase II multi center study, open label, preceded by a retrospective non-interventional period, to assess the long-term safety and efficacy of alpelisib, in pediatric and adult participants with PROS.
详细描述
The study will enroll males and females aged ≥2 years who previously participated in EPIK-P1 study and who continued to receive treatment with alpelisib after the cut-off date used in EPIK-P1 (NCT04285723).
The study has an initial retrospective period and a subsequent prospective period.
The retrospective period, is a non-interventional study period and will start one day after the EPIK-P1 data cut-off date (i.e. 10-Mar-2020). It will end the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Key safety and efficacy information that was previously collected as per local medical practice and recorded in the medical charts of eligible participants will be longitudinally abstracted.
The prospective period, is an interventional study period and will start on the day of the first interventional dose administration in the prospective period. It will end after all participants have completed at least 5 years of treatment in the prospective period of the study or discontinued earlier, whichever occurs earlier. During this study period, safety and efficacy data will be prospectively collected following a structured plan that is common to all participants.
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
- 2 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants who had previously participated in the study EPIK-P
- •Signed informed consent form and assent (when applicable) from the participant, parent, or guardian must be obtained prior to any study related screening procedures being performed.
- •Participant is treated with at least one dose of alpelisib after the EPIK-P1 study data cut- off date of 09-Mar-2020.
排除标准
- •For participants in the retrospective period
- •- All EPIK-P1 participants who permanently discontinued the investigational drug on or prior to the cut-off date 09-Mar-
- •For participants in the prospective period
- •Previous alpelisib treatment discontinuation (after 09-Mar-2020) due to any of the following adverse events:
- •Grade 4 skin and subcutaneous tissue disorders
- •Stevens-Johnson-Syndrome (SJS)/ Toxic Epidermal Necrolysis (TEN) or other SJS/TEN-like severe skin reactions (any grade)
- •Grade 4 hyperglycemia without confounding factors
- •Pneumonitis (any grade)
- •Grade 4 stomatitis
- •Grade 4 pancreatitis
- •Recurrent grade 4 thrombocytopenia
- •Grade 3 or 4 serum creatinine increase
- •Grade 4 isolated total bilirubin elevation
- •Recurrent grade 3 or 4 QT interval corrected by Fridericia's formula prolongation (>500 ms or >60 ms change from baseline)
- •Known impairment of GI function due to concomitant disease that may significantly alter the absorption of the study drug (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection) at time of informed consent.
- •Participant with uncontrolled diabetes mellitus (Type I or II) at time of informed consent.
- •Other inclusion/exclusion criteria may apply
研究组 & 干预措施
Alpelisib
All participants will receive alpelisib once a day
干预措施: Alpelisib (Drug)
结局指标
主要结局
Prospective period only: Proportion of participants with new or worsening grade ≥3 treatment emergent adverse events (AEs)
时间窗: From date of first interventional dose administration in the prospective period (Day 1) to 30 days after last dose of study drug, assessed up to 5 years.
Incidence of new or worsening grade ≥3 treatment emergent AEs (by system organ class and preferred term)
次要结局
- Retrospective period: Proportion of participants with Adverse Events (AEs)(From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1))
- Prospective period: Proportion of participants with AEs(From Day 1 up to 5 years)
- Retrospective and prospective period: Overall clinical assessment as assessed by the investigator(Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years)
- Retrospective and prospective period: Incidence of PROS-related symptoms and complications/comorbidities among participants with symptoms and complications/comorbidities.(Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years)
- Retrospective and prospective period: Proportion of participants with healthcare visits/hospitalizations due to PROS.(Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years)
- Retrospective and prospective period: Proportion of participants requiring PROS-related treatment(s) other than alpelisib(Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years)
- Retrospective and prospective period: Proportion of participants with dose adjustments of alpelisib(Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years)
- Retrospective and prospective period: Proportion of participants with PROS-related surgeries(Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years)