Clinical trial NCT04285723: Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
Official 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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: PIK3CA-Related Overgrowth Spectrum (PROS)
Keywords: alpelisib
PIK3CA
BYL719
PROS
Retrospective chart review study
EPIK-P1
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2020-02-26
Enrollment: 57
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients who participated in the compassionate use program and received alpelisib for the treatment of PIK3CA-related overgrowth spectrum (PROS).

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.

Detailed description: 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.

Eligibility criteria: 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)

Primary outcomes:
measure: Percentage of Patients Responders and Non-responders at Week 24
timeFrame: Index date and week 24 or 6 months (± 4 weeks)
Secondary outcomes:
measure: Percent Change in the Sum of Measurable Target Lesion Volume
timeFrame: Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
measure: Percent Change in the Sum of All Measurable Lesion Volume
timeFrame: Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
measure: Percent Change in the Sum of All Measurable Non-target Lesion Volume
timeFrame: Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
measure: Mean Duration of Response (DoR)
timeFrame: Up to 187 weeks
measure: Reasons for Discontinuation of Concomitant PROS-related Non-drug Treatments
timeFrame: Up to 187 weeks
measure: Participants With Concomitant PROS-related Medications Over Time
timeFrame: Index date, week 24 and end of study
measure: Number of PROS-related Completed Surgeries During the Study Period
timeFrame: Up to 187 weeks
measure: Number of Patients With Improvement in Most Frequent PROS-related Signs and Symptoms
timeFrame: Index date and week 24 or 6 months (± 4 weeks)
measure: Change in Performance Status Score
timeFrame: Index date and week 24 or 6 months (± 4 weeks)
measure: Functional Status - Mobility Assessment
timeFrame: Up to 187 weeks
measure: Change From Index Date in Functional Status - School Status During the Study Period
timeFrame: Index date, week 24 and end of study (up to 187 weeks)
measure: Change From Index Date in Functional Status - Work Status
timeFrame: Index date, week 24 and end of study
measure: Health Resource Utilization (HRU) - Number of Hospitalizations Per Patient
timeFrame: Up to 187 weeks
measure: Number of Patients With Grade 3/4 on Laboratory Assessments: Hematology
timeFrame: Up to 187 weeks
measure: Number of Patients With Grade 3/4 on Clinical Assessments - Clinical Chemistry
timeFrame: 187 weeks
measure: Notable Vital Sign Values During the Study Period - Pediatric Patients
timeFrame: End of study (up to week 187)
measure: Notable Vital Sign Values During the Study Period - Adult Patients
timeFrame: End of study (up to week 187)
measure: Number of Participants With Notable ECG Values.
timeFrame: Up to week 187
measure: Growth and Development in Pediatric Population
timeFrame: Week 24 or 6 months (± 4 weeks)
measure: Overview of Number of Patients With Adverse Events (AEs)
timeFrame: Up to 187 weeks
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Childrens Hospital
state: Massachusetts
city: Parkville
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Montpellier cedex 5
country: France
facility: Novartis Investigative Site
state: Herault
city: Dijon
country: France
facility: Novartis Investigative Site
city: Paris cedex 15
country: France
facility: Novartis Investigative Site
city: Dublin
country: Ireland
facility: Novartis Investigative Site
city: Madrid
country: Spain
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: