Trial · NCT04589650
Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum
NCT04589650, Phase 2, Active not recruiting, Novartis Pharmaceuticals; results first posted 2025-02-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum. Trials, record NCT04589650. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04589650
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum
- NCT number
- NCT04589650
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 236 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- PIK3CA-related Overgrowth Spectrum (PROS)
- Interventions
- Alpelisib, Placebo
- First posted
- 2020-10-19
- Last updated
- 2026-10-07
- Results first posted
- 2025-02-10
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Participants Randomized to Alpelisib With a Confirmed Objective Response by BIRC in Group 1 and Group 2 | Up to 48 weeks | A responder is defined by achieving a \>=20% reduction from baseline in the sum of target lesion volumes (via BIRC), provided that none of the individual target lesions have a \>=20% increase from baseline and in absence of progression of non target lesions and without new lesions. Confirmation of response requires a subsequent imaging assessment performed at least 4 weeks after the onset of response. Participants who permanently discontinued alpelisib prior to confirmation of response, and participants who received surgery as rescue therapy prior to confirmation of response are considered as non-responders. |
| Secondary | Key Secondary Objective: Proportion of Participants With Response at Week 16 by BIRC in Group 1 and Group 2 | Week 16 | A responder is defined by achieving a \>=20% reduction from baseline in the sum of target lesion volumes (via BIRC) at Week 16, provided that none of the individual target lesions have a \>=20% increase from baseline and in absence of progression of non-target lesions and without new lesions. Participants who permanently discontinued alpelisib prior to Week 16, participants who received surgery as rescue therapy prior to Week 16, and participants who had a missing/non-evaluable radiological assessment at Week 16 are considered as non-responders. |
| Secondary | Proportion of Participants With a Response at Week 24 (by BIRC) in Groups 1 and 2 | Week 24 | A responder is defined by achieving a \>=20% reduction from baseline in the sum of target lesion volumes (via BIRC) at Week 24, provided that none of the individual target lesions have a \>=20% increase from baseline and in absence of progression of non-target lesions and without new lesions. |
| Secondary | Frequency and Severity of Adverse Events in Groups 1 and 2 up to Week 16 | Up to Week 16 | An adverse event (AE) is any untoward medical occurrence (e.g., any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a participant or clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product. This includes events reported by the participant (or, when appropriate, by a caregiver, surrogate, or the participant's legally authorized representative). Treatment emergent Adverse Event (TEAEs) in this study are events that started after the first dose of study treatment and until 30 days after the last dose of study treatment, or events present prior to the first dose of treatment which increased in severity based on preferred term within 30 days after the last study treatment. |
| Secondary | Frequency and Severity of Adverse Events in All Groups of Participants Over Time | Up to approximately 5 years | Type, frequency, seriousness, and severity of treatment-emergent adverse events per CTCAE v4.03 criteria in participants with PROS over time. |
| Secondary | Change From Baseline to Week 16 in Brief Pain Inventory (BPI) Worst Pain Intensity in Group 1 and 2 | Baseline, Week 4, Week 8, Week 12, Week 16 | For adult and pediatric patients 12 years of age and older, the BPI item that assesses worst pain intensity in the past 24 hours was used. Patients respond to the item on an 11-point response scale ranging from 0 (no pain) to 10 (pain as bad as you can imagine). Worst pain intensity was averaged weekly over a 7-day period if a patient had completed the questionnaire for at least 4 days in the 7-day period. The weekly mean was calculated based on the available assessments. Clinically important change at Week 16 was defined as a 2-point reduction for patients who had a pain intensity score ≥ 4 at baseline. For patients with a baseline score \< 4, a 1-point reduction from baseline was also considered as a clinically important change. |
| Secondary | Number of Participants With Global Impression of Symptom Severity (PGIS) Score up to Week 16 | Week 4, Week 8, Week 16 | A Patient Global Impression of Symptom Severity item was used to understand the overall severity of symptoms experienced and clinical meaningfulness of treatment effects experienced during this study. This item included 5 response options: no symptoms, mild, moderate, severe, and very severe. |
| Secondary | Percentage Change From Baseline in Target and MRI-measurable Non- Target Lesion Volume in Group 1 and Group 2 | From Baseline up to approximately 5 years | Percentage change from baseline in the sum of target lesion volume, MRI-measurable non-target lesion volume and all MRI measurable (target an non-target) lesion volume as assessed by BIRC. Target lesions are defined as anatomically reproducibly defined tissue(s) masses, which may be composed of one or several tissue types, and can be accurately measured by imaging technique MRI. Target lesion(s) (up to 3) should be identified at screening, be at least 2 cm in the longest diameter at baseline (for each selected lesion) and may be further reproducibly assessed by MRI. MRI-measurable non-target lesions are defined as all anatomic lesions other than selected as target and may be measured at radiologic assessment (at least 2 cm in the longest diameter at baseline, the volume may be further reproducibly assessed by MRI). |
| Secondary | Proportion of Participants With Changes From Baseline in Other Non-target Lesions in Group 1 and Group 2 | From Baseline up to approximately 5 years | Percentage change from baseline in other non-target lesions (by BIRC). Non Target lesions are defined as: * Anatomic lesions, limb/trunkal areas affected by PROS, organomegaly when they may be measured only by caliper/ruler (e.g., circumference of changed limb or body part) * Truly non-measurable lesions (e.g., small lesions less than 2 cm on MRI, superficial visual lesions, masses, organomegaly, PROS-related enlargement of anatomic area identified by physical exam that is not measurable by reproducible imaging technique) |
| Secondary | Proportion of Participants With New Lesions in Group 1 and Group 2 | From Baseline up to approximately 5 years | The proportion of patients with new lesions (as assessed by BIRC) will be assessed throughout the study. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion of Participants Randomized to Alpelisib With a Confirmed Objective Response by BIRC in Group 1 and Group 2 | Up to 48 weeks | Full Analysis Set (FAS) - BYL719. All patients to whom alpelisib had been assigned by randomization. | % of confirmed responder by BIRC | NUMBER |
| SECONDARY | Key Secondary Objective: Proportion of Participants With Response at Week 16 by BIRC in Group 1 and Group 2 | Week 16 | Full Analysis Set (FAS). All patients to whom study treatment was assigned by randomization. | % of responder by BIRC | NUMBER |
| SECONDARY | Proportion of Participants With a Response at Week 24 (by BIRC) in Groups 1 and 2 | Week 24 | Full Analysis Set (FAS) - BYL719. All patients to whom alpelisib had been assigned by randomization. | % of confirmed responder by BIRC | NUMBER |
| SECONDARY | Frequency and Severity of Adverse Events in Groups 1 and 2 up to Week 16 | Up to Week 16 | Safety Set. All patients who received at least one dose of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Frequency and Severity of Adverse Events in All Groups of Participants Over Time | Up to approximately 5 years | |||
| SECONDARY | Change From Baseline to Week 16 in Brief Pain Inventory (BPI) Worst Pain Intensity in Group 1 and 2 | Baseline, Week 4, Week 8, Week 12, Week 16 | Full Analysis Set (FAS). Only participants with a value at both Baseline and post-baseline visit included. | Score on a scale | MEAN |
| SECONDARY | Number of Participants With Global Impression of Symptom Severity (PGIS) Score up to Week 16 | Week 4, Week 8, Week 16 | Full Analysis Set (FAS). Only participants with an available value for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage Change From Baseline in Target and MRI-measurable Non- Target Lesion Volume in Group 1 and Group 2 | From Baseline up to approximately 5 years | |||
| SECONDARY | Proportion of Participants With Changes From Baseline in Other Non-target Lesions in Group 1 and Group 2 | From Baseline up to approximately 5 years | |||
| SECONDARY | Proportion of Participants With New Lesions in Group 1 and Group 2 | From Baseline up to approximately 5 years |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group132
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion of Participants Randomized to Alpelisib With a Confirmed Objective Response by BIRC in Group 1 and Group 2 | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | 16.7 | 97.5% Confidence Interval 7.0 to 31.1 | % of confirmed responder by BIRC | |
| Proportion of Participants Randomized to Alpelisib With a Confirmed Objective Response by BIRC in Group 1 and Group 2 | Group 2 (6-17 Years) - Alpelisib Arm | 23.2 | 97.5% Confidence Interval 11.9 to 38.3 | % of confirmed responder by BIRC | |
| Key Secondary Objective: Proportion of Participants With Response at Week 16 by BIRC in Group 1 and Group 2 | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | 11.1 | 95% Confidence Interval 4.2 to 22.6 | % of responder by BIRC | |
| Key Secondary Objective: Proportion of Participants With Response at Week 16 by BIRC in Group 1 and Group 2 | Group 1 (Greater or Equal to 18 Years) - Placebo Arm | 0 | 95% Confidence Interval 0.0 to 12.8 | % of responder by BIRC | |
| Key Secondary Objective: Proportion of Participants With Response at Week 16 by BIRC in Group 1 and Group 2 | Group 2 (6-17 Years) - Alpelisib Arm | 8.9 | 95% Confidence Interval 3.0 to 19.6 | % of responder by BIRC | |
| Key Secondary Objective: Proportion of Participants With Response at Week 16 by BIRC in Group 1 and Group 2 | Group 2 (6-17 Years) - Placebo Arm | 0 | 95% Confidence Interval 0.0 to 12.3 | % of responder by BIRC | |
| Proportion of Participants With a Response at Week 24 (by BIRC) in Groups 1 and 2 | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | 16.7 | 97.5% Confidence Interval 7 to 31.1 | % of confirmed responder by BIRC | |
| Proportion of Participants With a Response at Week 24 (by BIRC) in Groups 1 and 2 | Group 2 (6-17 Years) - Alpelisib Arm | 19.6 | 97.5% Confidence Interval 9.2 to 34.3 | % of confirmed responder by BIRC | |
| Frequency and Severity of Adverse Events in Groups 1 and 2 up to Week 16 | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | Adverse Events (AEs) | 50 | Participants | |
| Frequency and Severity of Adverse Events in Groups 1 and 2 up to Week 16 | Group 1 (Greater or Equal to 18 Years) - Placebo Arm | Adverse Events (AEs) | 26 | Participants |
1 to 10 of 132Source ClinicalTrials.gov
See all 132 results by groupParticipant flow54
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | 54 | |
| Overall Study | STARTED | Group 1 (Greater or Equal to 18 Years) - Placebo Arm | 27 | |
| Overall Study | STARTED | Group 2 (6-17 Years) - Alpelisib Arm | 56 | |
| Overall Study | STARTED | Group 2 (6-17 Years) - Placebo Arm | 28 | |
| Overall Study | STARTED | Exploratory Group 4 (2-5 Years) All Pediatrics - Alpelisib FCT | 7 | |
| Overall Study | STARTED | Exploratory Group 5 (6-17 Years) All Pediatrics - Alpelisib FCT | 16 | |
| Overall Study | Completed Core | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | 52 | |
| Overall Study | Completed Core | Group 1 (Greater or Equal to 18 Years) - Placebo Arm | 26 | |
| Overall Study | Completed Core | Group 2 (6-17 Years) - Alpelisib Arm | 55 | |
| Overall Study | Completed Core | Group 2 (6-17 Years) - Placebo Arm | 26 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 participant flowAdverse events2,172
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | 0 | 54 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 1 (Greater or Equal to 18 Years) - Placebo Arm | 0 | 27 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 1 (Greater or Equal to 18 Years) Alpelisib Period/Alpelisib Arm | 0 | 54 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 1 (Greater or Equal to 18 Years) Alpelisib Period/Placebo Arm | 0 | 26 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 1 (Greater or Equal to 18 Years) All Adults in Alpelisib Period | 0 | 80 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 2 (6-17 Years) - Alpelisib Arm | 0 | 56 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 2 (6-17 Years) - Placebo Arm | 0 | 28 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 2 (6-17 Years) Alpelisib Period Alpelisib Arm | 0 | 56 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 2 (6-17 Years) Alpelisib Period Placebo Arm | 0 | 26 | |
| other | Abdominal lymphadenopathy | Blood and lymphatic system disorders | Group 2 (6-17 Years) All Pediatrics in Alpelisib Period | 0 | 82 |
1 to 10 of 2,172Source ClinicalTrials.gov
See all 2,172 adverse eventsAdverse event totals36
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Group 1 (Greater or Equal to 18 Years) - Alpelisib Arm | 0 | 54 |
| Total, all-cause mortality | Group 1 (Greater or Equal to 18 Years) - Placebo Arm | 0 | 27 |
| Total, all-cause mortality | Group 1 (Greater or Equal to 18 Years) Alpelisib Period/Alpelisib Arm | 0 | 54 |
| Total, all-cause mortality | Group 1 (Greater or Equal to 18 Years) Alpelisib Period/Placebo Arm | 0 | 26 |
| Total, all-cause mortality | Group 1 (Greater or Equal to 18 Years) All Adults in Alpelisib Period | 0 | 80 |
| Total, all-cause mortality | Group 2 (6-17 Years) - Alpelisib Arm | 0 | 56 |
| Total, all-cause mortality | Group 2 (6-17 Years) - Placebo Arm | 1 | 28 |
| Total, all-cause mortality | Group 2 (6-17 Years) Alpelisib Period Alpelisib Arm | 0 | 56 |
| Total, all-cause mortality | Group 2 (6-17 Years) Alpelisib Period Placebo Arm | 0 | 26 |
| Total, all-cause mortality | Group 2 (6-17 Years) All Pediatrics in Alpelisib Period | 0 | 82 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
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/NCT04589650
GET https://conductscience.org/api/trials/v1/NCT04589650/registered?page=2