Trial · NCT04980833
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)
NCT04980833, Phase 2, Active not recruiting, Novartis Pharmaceuticals
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. 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). Trials, record NCT04980833. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04980833
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 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)
- NCT number
- NCT04980833
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 41 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- PIK3CA-related Overgrowth Spectrum (PROS)
- Interventions
- Alpelisib
- First posted
- 2021-07-28
- Last updated
- 2026-10-14
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Prospective period only: Proportion of participants with new or worsening grade ≥3 treatment emergent adverse events (AEs) | From first interventional dose administration in the prospective period (Day 1) until treatment discontinuation, assessed for at least 5 years; participants not entering a Novartis rollover study undergo a 30-day safety follow-up after the last dose. | Incidence of new or worsening grade ≥3 treatment emergent AEs (by system organ class and preferred term) |
| Secondary | Retrospective period: Proportion of participants with Adverse Events (AEs) | From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period (Day -1) | Incidence, type and severity per CTCAE v4.03 criteria, causality assessments of AEs, including changes in laboratory values, vital signs and cardiac function during the retrospective period. |
| Secondary | Prospective period: Proportion of participants with AEs | From first interventional dose administration in the prospective period (Day 1) until treatment discontinuation, assessed for at least 5 years | Incidence, type and severity per CTCAE v4.03 criteria, causality assessments of AEs, including changes in laboratory values, vital signs, cardiac function, and growth, sexual maturation, and bone/dental development (for applicable ages). |
| Secondary | Retrospective and prospective period: Overall clinical assessment as assessed by the investigator | Retrospective: From 10-Mar-2020 to Day -1. Prospective: From Day 1 until treatment discontinuation, assessed for at least 5 years. | Proportion of participants with overall clinical assessment reported as improvement, stable or worsened, as assessed by the investigator. |
| Secondary | Retrospective and prospective period: Incidence of PROS-related symptoms and complications/comorbidities among participants with symptoms and complications/comorbidities. | Retrospective: From 10-Mar-2020 to Day -1. Prospective: From Day 1 until treatment discontinuation, assessed for at least 5 years. | Incidence of symptoms and complications/comorbidities associated with PROS among affected participants. |
| Secondary | Retrospective and prospective period: Proportion of participants with healthcare visits/hospitalizations due to PROS. | Retrospective: From 10-Mar-2020 to Day -1. Prospective: From Day 1 until treatment discontinuation, assessed for at least 5 years | Proportion of participants with healthcare visits/hospitalizations due to PROS. |
| Secondary | Retrospective and prospective period: Proportion of participants requiring PROS-related treatment(s) other than alpelisib | Retrospective: From 10-Mar-2020 to Day -1. Prospective: From Day 1 until treatment discontinuation, assessed for at least 5 years | Proportion of participants requiring PROS-related treatment(s) other than alpelisib, including medications (concomitant PROS-related medications including medication for the management of PROS related complications as well as medications to manage complications secondary to alpelisib) and non-drug treatments (e.g., feeding tube, ketogenic diet, non-invasive device for sleep apnea, sclerotherapy, endovascular occlusive procedures) |
| Secondary | Retrospective and prospective period: Proportion of participants with dose adjustments of alpelisib | Retrospective: From 10-Mar-2020 to Day -1. Prospective: From Day 1 until treatment discontinuation, assessed for at least 5 years | Proportion of participants requiring dose increase, reductions and interruptions of alpelisib |
| Secondary | Retrospective and prospective period: Proportion of participants with PROS-related surgeries | Retrospective: From 10-Mar-2020 to Day -1. Prospective: From Day 1 until treatment discontinuation, assessed for at least 5 years | Proportion of participants requiring surgery due to PROS |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40603171 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04980833
GET https://conductscience.org/api/trials/v1/NCT04980833/registered