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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04285723
Registration number
NCT04285723
Ethics application status
Date submitted
26/11/2019
Date registered
26/02/2020
Date last updated
9/02/2023
Titles & IDs
Public title
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
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Scientific title
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)
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Secondary ID [1]
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CBYL719F12002
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Universal Trial Number (UTN)
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Trial acronym
EPIK-P1
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
PIK3CA-Related Overgrowth Spectrum (PROS)
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Condition category
Condition code
Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Other interventions - alpelisib
alpelisib - Patients treated with alpelisib
Other interventions: alpelisib
Retrospective observational case-only study. There is no treatment allocation. Patients with severe or life-threatening PROS who have received alpelisib as part of a compassionate use program were invited to participate.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of Patients Responders and Non-responders at Week 24
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Assessment method [1]
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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.
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Timepoint [1]
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Index date and week 24 or 6 months (± 4 weeks)
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Secondary outcome [1]
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Percent Change in the Sum of Measurable Target Lesion Volume
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Assessment method [1]
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Percent change in the sum of measurable target lesion (1 to 3 lesions) volume, as assessed by a central review of imaging scans, as measured by the change between the index date (or up to 24 weeks prior) and key time-points following the index date.
The index date: (baseline) was defined as the date of alpelisib initiation End of study: patients were followed up to a maximum of 187 weeks.
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Timepoint [1]
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Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
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Secondary outcome [2]
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Percent Change in the Sum of All Measurable Lesion Volume
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Assessment method [2]
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Percent change in the sum of all measurable (target and non-target) lesion volume, as assessed by a central review of imaging scans, as measured by the change between the index date (or up to 24 weeks prior) and key time-points following the index date.
The index date (baseline) was defined as the date of alpelisib initiation. End of study: patients were followed up to a maximum of 187 weeks As no measurable non-target lesions were identified, the results for changes in the sum of all measurable (target and non-target) lesion volume were identical to the results presented for measurable target lesion.
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Timepoint [2]
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Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
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Secondary outcome [3]
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Percent Change in the Sum of All Measurable Non-target Lesion Volume
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Assessment method [3]
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Percent change in the sum of all measurable non-target lesion volume, as assessed by a central review of imaging scans, as measured by the change between the index date (or up to 24 weeks prior) and key time-points following the index date.
The index date (baseline) was defined as the date of alpelisib initiation.
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Timepoint [3]
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Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
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Secondary outcome [4]
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Mean Duration of Response (DoR)
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Assessment method [4]
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Duration of response is defined as the time from first documented response, to the date of the first documented disease progression or death due to any cause.
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.
Disease progression is defined as an increase of any individual target lesions of = 20% in volume from previous assessment, progression of non-target PIK3CA Related Overgrowth Spectrum (PROS) lesions or appearance of a new PROS lesion.
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Timepoint [4]
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Up to 187 weeks
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Secondary outcome [5]
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Reasons for Discontinuation of Concomitant PROS-related Non-drug Treatments
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Assessment method [5]
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Number of participants with concomitant PIK3CA Related Overgrowth Spectrum (PROS)-related non-drug treatments and other medical interventions by reason for discontinuation.
A patient may have multiple information, but was counted only once in type of other supportive non-drug treatment if more than one type in this category has been reported.
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Timepoint [5]
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Up to 187 weeks
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Secondary outcome [6]
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Participants With Concomitant PROS-related Medications Over Time
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Assessment method [6]
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Number of participants with at least one concomitant utilization of PIK3CA Related Overgrowth Spectrum (PROS)-related medication.
End of study: patients were followed up to a maximum of 187 weeks. Results are provided for the full study population as planned and documented in the protocol and SAP, and not by age group.
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Timepoint [6]
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Index date, week 24 and end of study
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Secondary outcome [7]
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Number of PROS-related Completed Surgeries During the Study Period
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Assessment method [7]
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Number of surgeries by reason of surgery. A patient may have multiple anatomical sites of surgery and types of surgery on the same day.
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Timepoint [7]
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Up to 187 weeks
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Secondary outcome [8]
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Number of Patients With Improvement in Most Frequent PROS-related Signs and Symptoms
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Assessment method [8]
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Improvement in PIK3CA Related Overgrowth Spectrum (PROS) signs and symptoms was defined based on Common Toxicity Criteria (CTC) grade reduction or resolution of the event from index date.
CTC is a set of criteria for the standardized classification of adverse effects of drugs used in cancer therapy. The Common Terminology Criteria for Adverse Events (CTCAE) system is a product of the US National Cancer Institute (NCI). For this study, version 4.03 was used
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Timepoint [8]
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Index date and week 24 or 6 months (± 4 weeks)
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Secondary outcome [9]
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Change in Performance Status Score
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Assessment method [9]
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Participants with a change in performance status score (ECOG, Karnofsky, Lansky) between the index date and week 24. Patients completed one questionnaire (ECOG, Karnofsky or Lansky) based on physicians choice.
The Eastern Cooperative Oncology Group (ECOG) score runs from 0 to 5, with 0 denoting perfect health and 5 death. The Karnofsky Performance Score (KPS) and Lansky score run from 0 to 100, where 0 is death and 100 is perfect health.
For the ECOG scale, "improvement" is defined as a decrease by at least 1 point and and "worsening" is defined as an increase by at least 1 point. For the Karnofsky and Lansky scale, "improvement" is defined as an increase by at least 20 points and worsening is defined as a decrease by at least 20 points.
If neither of the criteria for improvement or worsening is met, then it is considered stable.
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Timepoint [9]
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Index date and week 24 or 6 months (± 4 weeks)
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Secondary outcome [10]
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Functional Status - Mobility Assessment
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Assessment method [10]
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Change in functional status: Mobility severity assessment measured up to the judgment of the investigator.
A patient may have multiple information
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Timepoint [10]
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Up to 187 weeks
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Secondary outcome [11]
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Change From Index Date in Functional Status - School Status During the Study Period
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Assessment method [11]
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Change in functional status: School status during the study period (no attendance, part-time or full time).
Improvement is defined as a shift from reporting of "No attendance" to "Part-time" as well as , "Part-time" or "No attendance" to "Full-time"; Worsening is defined as a shift from reporting of "Part-time" to "No attendance", as well as from "Full-time" to "Part-time" or "No attendance".
If neither of the criteria for improvement or worsening is met, then it is considered stable.
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Timepoint [11]
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Index date, week 24 and end of study (up to 187 weeks)
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Secondary outcome [12]
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Change From Index Date in Functional Status - Work Status
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Assessment method [12]
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Change in functional status: Work status assessment during the study period (no attendance, part-time, full-time or unemployed).
Improvement is defined as a shift from reporting of "No attendance" to "Part-time" or "Full time", from "Part-time" to "Full-time", as well as from "Unemployed" to "Part-time" or "Full-time" work.
Worsening is defined as a shift from reporting of "Part-time" to "No attendance", "Full-time" to "Part-time" or "No attendance" as well as "Part-time" or "Full-time" to "Unemployed" status.
Change in score is only applicable if both index date and post-index date information are available.
If a patient has more than 1 score reported within the same time window, the worst is considered.
If neither of the criteria for improvement or worsening is met, then it is considered stable.
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Timepoint [12]
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Index date, week 24 and end of study
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Secondary outcome [13]
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Health Resource Utilization (HRU) - Number of Hospitalizations Per Patient
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Assessment method [13]
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Hospitalizations starting on or after the start of study treatment (index date) or starting prior to and continuing after the start of study treatment are summarized.
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Timepoint [13]
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Up to 187 weeks
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Secondary outcome [14]
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Number of Patients With Grade 3/4 on Laboratory Assessments: Hematology
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Assessment method [14]
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Worst post-index date hematology abnormalities based on Common Toxicity Criteria (CTC) grades during the study period.
Grade refers to the severity of the AE: Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated.
Patients are counted only for the worst grade observed post-index date values. Laboratory assessments performed more than 30 days after last study treatment administration date are not summarized.
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Timepoint [14]
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Up to 187 weeks
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Secondary outcome [15]
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Number of Patients With Grade 3/4 on Clinical Assessments - Clinical Chemistry
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Assessment method [15]
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Worst post-index date biochemistry abnormalities based on Common Toxicity Criteria (CTC) grades.
Grade refers to the severity of the AE: Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated.
Patients are counted only for the worst grade observed post-index date values. Laboratory assessments performed more than 30 days after last study treatment administration date are not summarized.
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Timepoint [15]
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187 weeks
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Secondary outcome [16]
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Notable Vital Sign Values During the Study Period - Pediatric Patients
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Assessment method [16]
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Low/High: Indicating abnormal value/change from index date.
High systolic blood pressure: =95th percentile of the age and height group Low Systolic blood pressure: =5th percentile of the age and height group. High diastolic blood pressure: =95th percentile of the age and height group Low diastolic blood pressure: =5th percentile of the age and height group. High pulse rate (bpm): 2-3 years: >128; 3-4 years: >123; 4-6 years: >117; 6-8 years: >111; 8-12years: >103; 12-15 years: >96; =15 years: >92 Low pulse rate (bpm): 2-3 years: <92; 3-4 years: <86; 4-6 years: <81; 6-8 years: <74; 8-12 years: <67; 12-15 years: <62; =15 years: <58.
High weight: increase from baseline of =2 Body mass index (BMI) for age percentile categories Low weight: decrease from baseline of =2 BMI-for-age percentile categories
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Timepoint [16]
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End of study (up to week 187)
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Secondary outcome [17]
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Notable Vital Sign Values During the Study Period - Adult Patients
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Assessment method [17]
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Low/High: Indicating abnormal value/change from index date.
High systolic blood pressure: =180 mmHg and increase =20 mmHg Low systolic blood pressure: =90 mmHg and decrease =20 mmHg. High diastolic blood pressure: =105 mmHg and increase =15 mmHg Low diastolic blood pressure: =50 mmHg and decrease =15 mmHg. High pulse rate: =120 bpm and increase =15 bpm Low pulse rate: = 50 bpm and decrease =15 bpm. High weight: Increase =10% Low weight: Decrease =10%
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Timepoint [17]
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End of study (up to week 187)
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Secondary outcome [18]
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Number of Participants With Notable ECG Values.
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Assessment method [18]
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Notable Electrocardiogram (ECG) values during the study period
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Timepoint [18]
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Up to week 187
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Secondary outcome [19]
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Growth and Development in Pediatric Population
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Assessment method [19]
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Assessed in patients who were aged <18 years at the time of alpelisib initiation.
SDS (standard deviation scores) for height and BMI are obtained from the WHO Growth Charts, and SDS for height and weight velocity are obtained from Baumgartner et al. (1986). Height or weight velocity is a variable derived from the measurement of height or weight at different times and represents the increase in height or weight during a fixed period.
SD- scores are used to describe how far a measurement is from the median (average).
Weight-for-age reference data are not available beyond age 10.
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Timepoint [19]
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Week 24 or 6 months (± 4 weeks)
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Secondary outcome [20]
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Overview of Number of Patients With Adverse Events (AEs)
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Assessment method [20]
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A patient with multiple severity grades for an AE is only counted under the maximum grade.
All grades includes any AEs with missing grade. Grade refers to the severity of the AE: Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated.
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Timepoint [20]
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Up to 187 weeks
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Eligibility
Key inclusion criteria
- 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)
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Minimum age
2
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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Study design
Purpose
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Duration
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Selection
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Timing
Retrospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
9/06/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
16/04/2021
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Sample size
Target
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Accrual to date
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Final
57
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Novartis Investigative Site - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Massachusetts
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Country [2]
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France
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State/province [2]
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Herault
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Country [3]
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France
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State/province [3]
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Dijon
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Country [4]
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France
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State/province [4]
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Paris cedex 15
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Country [5]
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Ireland
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State/province [5]
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Dublin
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Country [6]
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Spain
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State/province [6]
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Madrid
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04285723
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Novartis Pharmaceuticals
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Address
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Novartis Pharmaceuticals
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04285723
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