18 and older, female only, with Breast Cancer. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Pathological Complete Response (pCR) Per Investigator Assessment for Alpelisib vs. Placebo for PIK3CA Mutant CohortPrimary· After 24 weeks of treatment
Pathologic complete response (pCR) defined as absence of any residual invasive cancer on hematoxylin and eosin evaluation of the resected breast specimen and all sampled ipsilateral lymph nodes following completion of 24 weeks of treatment by local assessment (ypT0/Tis ypN0). Patients who experienced progression of disease while undergoing neoadjuvant therapy, or who did not receive surgery for any reason, or received antineoplastic treatment other than study drug(s) before surgery were considered as non-responders for the calculation of pCR rate.
Group
Value
95% CI
Alpelisib + Letrozole
1.7
0.2 – 6.3
Placebo + Letrozole
3.0
0.8 – 7.7
Pathological Complete Response (pCR) Per Investigator Assessment for Alpelisib vs. Placebo for PIK3CA Wild-type CohortPrimary· After 24 weeks of treatment
Pathologic complete response (pCR) defined as absence of any residual invasive cancer on hematoxylin and eosin evaluation of the resected breast specimen and all sampled ipsilateral lymph nodes following completion of 24 weeks of treatment by local assessment (ypT0/Tis ypN0). Patients who experienced progression of disease while undergoing neoadjuvant therapy, or who did not receive surgery for any reason, or received antineoplastic treatment other than study drug(s) before surgery were considered as non-responders for the calculation of pCR rate.
Group
Value
95% CI
Alpelisib + Letrozole
2.8
0.8 – 7.3
Placebo + Letrozole
1.7
0.2 – 6.4
Objective Response Rate Per Investigator Assessment According to RECIST 1.1 for Alpelisib vs. Placebo - PIK3CA Mutant CohortPrimary· After 24 weeks of treatment
Objective Response Rate (ORR) defined as the proportion of patients with a Best Overall Response (BOR) of Complete Response (CR) or Partial Response (PR) based on local investigator's assessment according to Response Evaluation Criteria In Solid Tumors Criteria (RECIST) 1.1.
BOR was assessed per MRI or Ultrasound and defined as per RECIST 1.1 as CR for a disappearance of all non-nodal target lesions (TL)/non-target lesions (NTL) and a reduction in short axis to \< 10 mm of any pathological lymph nodes assigned as TL/NTL and no new lesion; as PR if not qualifying for CR but with a decrease fro
Group
Value
95% CI
Alpelisib + Letrozole
43.3
34.6 – 52.4
Placebo + Letrozole
44.8
36.5 – 53.3
Objective Response Rate According to RECIST 1.1 Per Investigator Assessment for Alpelisib vs. Placebo - PIK3CA Wild-type CohortPrimary· After 24 weeks of treatment
Objective Response Rate (ORR) defined as the proportion of patients with a Best Overall Response (BOR) of Complete Response (CR) or Partial Response (PR) based on local investigator's assessment according to Response Evaluation Criteria In Solid Tumors Criteria (RECIST) 1.1.
BOR was assessed per MRI or Ultrasound and defined as per RECIST 1.1 as CR for a disappearance of all non-nodal target lesions (TL)/non-target lesions (NTL) and a reduction in short axis to \< 10 mm of any pathological lymph nodes assigned as TL/NTL and no new lesion; as PR if not qualifying for CR but with a decrease fro
Group
Value
95% CI
Alpelisib + Letrozole
63.4
55.1 – 71.0
Placebo + Letrozole
61.0
51.8 – 69.6
Rate of Breast Conserving Surgery for Alpelisib vs. Placebo - PIK3CA Mutant CohortSecondary· After 24 weeks of treatment
Breast conserving surgery is defined for participants who underwent surgery and did not have a mastectomy. The row "no surgery" provides the number of patients who did not undergo surgery at all for various reasons.
Breast conserving surgery
Group
Value
95% CI
Alpelisib + Letrozole
56.7
47.6 – 65.4
Placebo + Letrozole
50.7
42.2 – 59.2
No Surgery
Group
Value
95% CI
Alpelisib + Letrozole
15.0
9.3 – 22.6
Placebo + Letrozole
9.0
4.8 – 15.2
Rate of Breast Conserving Surgery for Alpelisib vs. Placebo - PIK3CA Wild-type CohortSecondary· After 24 weeks of treatment
Breast conserving surgery is defined as the percentage of participants with no mastectomy following completion of 24 weeks of treatment. Breast conserving surgery is defined for participants who underwent surgery and did not have a mastectomy. The row "no surgery" provides the number of patients who did not undergo surgery at all for various reasons.
Breast conserving surgery
Group
Value
95% CI
Alpelisib + Letrozole
50.7
42.5 – 58.9
Placebo + Letrozole
62.7
53.6 – 71.2
No Surgery
Group
Value
95% CI
Alpelisib + Letrozole
18.3
12.5 – 25.6
Placebo + Letrozole
8.5
4.5 – 15.2
Association Between pCR and Changes in Ki67 From Baseline for Alpelisib vs. Placebo - PIK3CA Mutant Cohort: Responders as Per pCRSecondary· Baseline, Cycle 1 Day 15 (each cycle is 28 days) and surgery (End of Treatment (EOT) expected after 24 weeks of treatment)
Association Between pCR and Changes in Ki67 From Baseline for Alpelisib vs. Placebo - PIK3CA Mutant Cohort: Responders as Per pCR
Baseline
Group
Value
95% CI
Alpelisib + Letrozole
5.0
5 – 5
Placebo + Letrozole
11.0
5 – 17
C1D15: % change from Baseline
Group
Value
95% CI
Alpelisib + Letrozole
-80.0
-80 – -80
Placebo + Letrozole
80.0
80 – 80
Association Between pCR and Changes in Ki67 From Baseline for Alpelisib vs. Placebo - PIK3CA Mutant Cohort: Non-responders as Per pCRSecondary· Baseline, Cycle 1 Day 15 (each cycle is 28 days ) and surgery (End of Treatment (EOT) expected after 24 weeks of treatment)
Association Between pCR and Changes in Ki67 From Baseline for Alpelisib vs. Placebo - PIK3CA Mutant Cohort: Non-responders as Per pCR.
Baseline
Group
Value
95% CI
Alpelisib + Letrozole
14.0
0 – 82
Placebo + Letrozole
13.0
3 – 89
C1D15 % change from Baseline
Group
Value
95% CI
Alpelisib + Letrozole
-62.5
-97 – 467
Placebo + Letrozole
-60.0
-96 – 733
EOT % change from Baseline
Group
Value
95% CI
Alpelisib + Letrozole
-51.2
-94 – 400
Placebo + Letrozole
-60.0
-97 – 223
Association Between pCR and Changes in Ki67 From Baseline for Alpelisib vs. Placebo - PIK3CA Wild-type Cohort: Responders as Per pCRSecondary· Baseline, Cycle 1 Day 15 (each cycle is 28 days) and surgery (End of Treatment (EOT) expected after 24 weeks of treatment)
Association between pCR and changes in Ki67 from baseline for alpelisib vs. placebo - PIK3CA wild-type cohort: responders as per pCR
Baseline
Group
Value
95% CI
Alpelisib + Letrozole
16.5
3 – 30
Placebo + Letrozole
20.0
20 – 20
C1D15: % change from Baseline
Group
Value
95% CI
Alpelisib + Letrozole
-80.0
-80 – -80
EOT % change from Baseline
Group
Value
95% CI
Alpelisib + Letrozole
-45.0
-90 – 0
Association Between pCR and Changes in Ki67 From Baseline for Alpelisib vs. Placebo - PIK3CA Wild-type Cohort: Non-responders as Per pCRSecondary· Baseline, Cycle 1 Day 15 (each cycle is 28 days) and surgery (End of Treatment (EOT) expected after 24 weeks of treatment)
Association between pCR and changes in Ki67 from baseline for alpelisib vs. placebo - PIK3CA wild-type cohort: non-responders as per pCR
Baseline
Group
Value
95% CI
Alpelisib + Letrozole
16.0
3 – 60
Placebo + Letrozole
18.0
4 – 85
C1D15 % change from Baseline
Group
Value
95% CI
Alpelisib + Letrozole
-60.0
-97 – 220
Placebo + Letrozole
-52.0
-93 – 50
EOT % change from Baseline
Group
Value
95% CI
Alpelisib + Letrozole
-60.0
-90 – 190
Placebo + Letrozole
-71.1
-100 – 250
Preoperative Endocrine Prognostic Index (PEPI) Response as Per Central Assessment for Alpelisib vs. Placebo - PIK3CA Mutant CohortSecondary· At the time of surgery (expected after 24 weeks of treatment)
Preoperative endocrine prognostic index (PEPI) response as per central assessment for alpelisib vs. placebo - PIK3CA mutant cohort. The total PEPI score assigned to each patient is the sum of the risk points derived from the pT stage, pN stage, Ki67 level, and ER status of the surgical specimen (Ellis et al, Contemp Clin Trials 2008). The PEPI score ranges from 0 to to 12, and a higher score means a worse outcome. PEPI response is defined as a PEPI score of 0.
Group
Value
95% CI
Alpelisib + Letrozole
1
Placebo + Letrozole
0
Preoperative Endocrine Prognostic Index (PEPI) Response as Per Central Assessment for Alpelisib vs. Placebo - PIK3CA Wild-type CohortSecondary· At the time of surgery (expected after 24 weeks of treatment)
Preoperative endocrine prognostic index (PEPI) response as per central assessment for alpelisib vs. placebo - PIK3CA wild-type cohort. The total PEPI score assigned to each patient is the sum of the risk points derived from the pT stage, pN stage, Ki67 level, and ER status of the surgical specimen (Ellis et al, Contemp Clin Trials 2008). The PEPI score ranges from 0 to to 12, and a higher score means a worse outcome. PEPI response is defined as a PEPI score of 0.
Group
Value
95% CI
Alpelisib + Letrozole
1
Placebo + Letrozole
1
Adverse events — posted to ClinicalTrials.gov
Time frame: All Adverse Events reported in this record are on-treatment events (from first dose of study treatment to last dose of study treatment + 30 days, duration expected to be a total of approximately 25 weeks)..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The purpose of the study was to determine whether treatment with a PI3K inhibitor plus letrozole led to an increase in pathologic clinical response and Objective Response Rate compared to treatment with placebo plus letrozole in patients with Breast cancer.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04285723 — Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
· completed
NCT04251533 — Study Assessing the Efficacy and Safety of Alpelisib + Nab-paclitaxel in Subjects With Advanced TNBC Who Carry Either a
· Phase 3
· terminated
NCT02077933 — Study of Safety and Efficacy of Alpelisib With Everolimus or Alpelisib With Everolimus and Exemestane in Advanced Breast
· Phase 1
· completed
NCT04085653 — Managed Access Programs for BYL719, Alpelisib
· available
NCT03706573 — Managed Access Program to Provide Alpelisib for Patients With HR+ Advanced or Metastatic Breast Cancer
· no longer available
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 14 September 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01923168.