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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04285723
Ethics application status
Date submitted
26/11/2019
Date registered
26/02/2020
Date last updated
30/10/2020

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
alpelisib - Patients treated with alpelisib

Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of patients with response (yes/no) at Week 24 (+/- 4 weeks) - Proportion of patients with response (yes/no) at Week 24 (+/- 4 weeks), 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.
Timepoint [1] 0 0
24 weeks (+/- 4 weeks)
Secondary outcome [1] 0 0
Percent change in the sum of measurable target lesion volume as measured by the change between the index date and key time-points following the index date - 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 12 weeks prior) and key time-points following the index date
Timepoint [1] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [2] 0 0
Percent change in the sum of all measurable (target and non-target) lesion volume between the index date (or up to 12 weeks prior) and key time-points following the index date - 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 12 weeks prior) and key time-points following the index date
Timepoint [2] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [3] 0 0
Description of medications and non-drug therapy received at key time points - Description of medications and non-drug therapy received at key time points
PROS-related treatment(s) other than alpelisib
Medication(s) (e.g., concomitant PROS-related medications including medication for the management of PROS related complications as well as medications to manage complications secondary to alpelisib)
Non-drug treatment(s) (e.g., feeding tube, ketogenic diet, non-invasive device for sleep apnea, sclerotherapy, endovascular occlusive procedures)
Alpelisib treatment (e.g., dose, dose adjustments, duration of treatment, dose interruptions, discontinuation)
PROS-related surgeries (e.g., de-bulking or vascular surgery as well as the intended site of the procedure)
Timepoint [3] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [4] 0 0
Change in PROS symptoms and complications over time - Change in PROS symptoms and complications over time
Overgrowth lesions, including number, girth, size, and color
Life-threatening complications (e.g., stroke, pulmonary embolism)
Chronic bleeding/leaking
Infection episodes
Hypotonia
Sleep disturbances
Seizures
Thrombotic events
Thromboembolic events
Pain
Cognitive impairment
Fatigue
Migraines
Depression/anxiety
Timepoint [4] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [5] 0 0
Change in functional status (work/school attendance) - Change in functional status
o Work/school/pre-school attendance
Timepoint [5] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [6] 0 0
Change in health care resource use - Change in health care resource use
Non-medical resource use (e.g., physical therapy, occupational therapy, home care services)
Hospitalizations (including relevant medical interventions undertaken if related to PROS)
ER visits (including relevant medical interventions undertaken
Timepoint [6] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [7] 0 0
Change in laboratory assessments - Change in laboratory assessments (e.g., D-dimer, fibrinogen, hemoglobin, renal function, albumin, protein)
Timepoint [7] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [8] 0 0
Type, frequency, seriousness, and severity per CTCAE v4.03 criteria and causality assessments of treatment-emergent adverse events - Type, frequency, seriousness, and severity per CTCAE v4.03 criteria and causality assessments of treatment-emergent adverse events
o Adverse events, including start and end date, grade, seriousness, relation to treatment, action taken with study treatment, and outcome
Timepoint [8] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [9] 0 0
Percent change in the sum of all measurable non-target lesion volume between the index date (or up to 12 weeks prior) and key time-points following the index date - 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 12 weeks prior) and key time-points following the index date
Timepoint [9] 0 0
4, 12, 24, 3, 52 weeks after starting the study treatment (index date)
Secondary outcome [10] 0 0
Change in functional status (mobility) - Change in functional status
o Mobility measured with an Investigator developed scale.
Timepoint [10] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [11] 0 0
Change in functional status (performance status) - Change in functional status
o ECOG performance status
Timepoint [11] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [12] 0 0
Change in assessments of cardiac function - Change in cardiac assessments (e.g. ECG)
Timepoint [12] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [13] 0 0
Change in clinical assessments (height) - Change in clinical assessments
o Vital signs (height)
Timepoint [13] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [14] 0 0
Change in clinical assessments (blood pressure) - Change in clinical assessments
o Vital signs (blood pressure)
Timepoint [14] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [15] 0 0
Change in clinical assessments (weight) - Change in clinical assessments
o Vital signs (weight)
Timepoint [15] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [16] 0 0
Change in clinical assessments (resting pulse) - Change in clinical assessments
o Vital signs (resting pulse)
Timepoint [16] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [17] 0 0
Change in functional status (performance status) - Change in functional status
o Lansky performance status
Timepoint [17] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [18] 0 0
Change in functional status (performance status) - Change in functional status
o Karnofsky performance status
Timepoint [18] 0 0
4, 12 , 24, 36, 52 weeks after starting the study treatment (index date)
Secondary outcome [19] 0 0
Duration of response - Duration of response defined as the time from first documented response, to the date of the first documented disease progression or death due to any cause.
Timepoint [19] 0 0
At the time of the cutoff date 9 March 2020

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
Gender
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
The study is a site-based retrospective non-interventional medical chart review of pediatric
and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS).
Patient-level data are abstracted from medical charts of all eligible patients at all
participating sites.

Information from patients treated with alpelisib will be used to describe the efficacy and
safety of alpelisib in PROS patients.
Trial website
https://clinicaltrials.gov/show/NCT04285723
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
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
novartis.email@novartis.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04285723