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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04285723




Registration number
NCT04285723
Ethics application status
Date submitted
26/11/2019
Date registered
26/02/2020

Titles & IDs
Public title
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
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)
Secondary ID [1] 0 0
CBYL719F12002
Universal Trial Number (UTN)
Trial acronym
EPIK-P1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
PIK3CA-Related Overgrowth Spectrum (PROS) 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
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.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Patients Responders and Non-responders at Week 24
Timepoint [1] 0 0
Index date and week 24 or 6 months (± 4 weeks)
Secondary outcome [1] 0 0
Percent Change in the Sum of Measurable Target Lesion Volume
Timepoint [1] 0 0
Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
Secondary outcome [2] 0 0
Percent Change in the Sum of All Measurable Lesion Volume
Timepoint [2] 0 0
Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
Secondary outcome [3] 0 0
Percent Change in the Sum of All Measurable Non-target Lesion Volume
Timepoint [3] 0 0
Index date, week 4, 12 , 24, 52 and end of study (up to a maximum of week 187)
Secondary outcome [4] 0 0
Mean Duration of Response (DoR)
Timepoint [4] 0 0
Up to 187 weeks
Secondary outcome [5] 0 0
Reasons for Discontinuation of Concomitant PROS-related Non-drug Treatments
Timepoint [5] 0 0
Up to 187 weeks
Secondary outcome [6] 0 0
Participants With Concomitant PROS-related Medications Over Time
Timepoint [6] 0 0
Index date, week 24 and end of study
Secondary outcome [7] 0 0
Number of PROS-related Completed Surgeries During the Study Period
Timepoint [7] 0 0
Up to 187 weeks
Secondary outcome [8] 0 0
Number of Patients With Improvement in Most Frequent PROS-related Signs and Symptoms
Timepoint [8] 0 0
Index date and week 24 or 6 months (± 4 weeks)
Secondary outcome [9] 0 0
Change in Performance Status Score
Timepoint [9] 0 0
Index date and week 24 or 6 months (± 4 weeks)
Secondary outcome [10] 0 0
Functional Status - Mobility Assessment
Timepoint [10] 0 0
Up to 187 weeks
Secondary outcome [11] 0 0
Change From Index Date in Functional Status - School Status During the Study Period
Timepoint [11] 0 0
Index date, week 24 and end of study (up to 187 weeks)
Secondary outcome [12] 0 0
Change From Index Date in Functional Status - Work Status
Timepoint [12] 0 0
Index date, week 24 and end of study
Secondary outcome [13] 0 0
Health Resource Utilization (HRU) - Number of Hospitalizations Per Patient
Timepoint [13] 0 0
Up to 187 weeks
Secondary outcome [14] 0 0
Number of Patients With Grade 3/4 on Laboratory Assessments: Hematology
Timepoint [14] 0 0
Up to 187 weeks
Secondary outcome [15] 0 0
Number of Patients With Grade 3/4 on Clinical Assessments - Clinical Chemistry
Timepoint [15] 0 0
187 weeks
Secondary outcome [16] 0 0
Notable Vital Sign Values During the Study Period - Pediatric Patients
Timepoint [16] 0 0
End of study (up to week 187)
Secondary outcome [17] 0 0
Notable Vital Sign Values During the Study Period - Adult Patients
Timepoint [17] 0 0
End of study (up to week 187)
Secondary outcome [18] 0 0
Number of Participants With Notable ECG Values.
Timepoint [18] 0 0
Up to week 187
Secondary outcome [19] 0 0
Growth and Development in Pediatric Population
Timepoint [19] 0 0
Week 24 or 6 months (± 4 weeks)
Secondary outcome [20] 0 0
Overview of Number of Patients With Adverse Events (AEs)
Timepoint [20] 0 0
Up to 187 weeks

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)
Minimum age
2 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose
Duration
Selection
Timing
Retrospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/06/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
16/04/2021
Sample size
Target
Accrual to date
Final
57
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
France
State/province [2] 0 0
Herault
Country [3] 0 0
France
State/province [3] 0 0
Dijon
Country [4] 0 0
France
State/province [4] 0 0
Paris cedex 15
Country [5] 0 0
Ireland
State/province [5] 0 0
Dublin
Country [6] 0 0
Spain
State/province [6] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04285723