Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)

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)

ClinicalTrials.gov Identifier: NCT04980833

Novartis Reference Number: CBYL719F12401

Last Update: May 02, 2022

See if you pre-qualify

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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.

Condition 
PIK3CA-related Overgrowth Spectrum (PROS)
Phase 
Phase 2
Overall status 
Recruiting
Start date 
Jan 27, 2022
Completion date 
May 27, 2027
Gender 
All
Age(s)
2 Years and older (Child, Adult, Older Adult)

Interventions

Drug
Alpelisib
Participant's treatment plan has been established by the treating physician, within the global compassionate use framework. Doses permitted are 50, 125, 200 and 250 mg.

Eligibility Criteria

Inclusion Criteria:

Participants who had previously participated in the study EPIK-P1.
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.

Exclusion Criteria:

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-2020.

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

Study Locations

France
Novartis Investigative Site
Recruiting
Dijon, 21000
-
France
Novartis Investigative Site
Recruiting
Montpellier Cedex, 34295
-
France
Novartis Investigative Site
Recruiting
Paris cedex 15, 75015
-
France
Ireland
Novartis Investigative Site
Recruiting
Dublin, 12
-
Ireland
Spain
Novartis Investigative Site
Recruiting
Madrid, 28046
-
Spain

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

Have a question?

Call 1-888-669-6682 or email [email protected]