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Profile Name PIK3CA mutant
Gene Variant Detail

PIK3CA mutant (unknown)

Relevant Treatment Approaches

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
PIK3CA mutant breast cancer sensitive VS-5584 Preclinical Actionable In a preclinical study, PIK3CA mutant breast cancer cells showed increased sensitivity to VS-5584 compared to PIK3CA wild-type cells in culture (PMID: 23270925). 23270925
PIK3CA mutant breast cancer no benefit GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 was well-tolerated and resulted in some efficacy in patients with breast cancer harboring a PIK3CA mutation including stable disease in 22% (2/9), however, objective response rate was not associated with PIK3CA mutations when compared to those without PIK3CA mutations (PMID: 26603258). 26603258
PIK3CA mutant Advanced Solid Tumor sensitive Bevacizumab + Pegylated liposomal-doxorubicin + Temsirolimus Clinical Study Actionable In a clinical study, the combination of Torisel (temisirolimus) plus Avastin (bevacizumab) and Doxil (pegylated liposomal-doxirubicin) resulted in stable disease in patients with advanced solid tumors harboring PIK3CA mutations (PMID: 21216929). 21216929
PIK3CA mutant Advanced Solid Tumor sensitive Bevacizumab + Pegylated liposomal-doxorubicin + Temsirolimus Clinical Study Actionable In a clinical study, the combination of Torisel (temsirolimus) plus Avastin (bevacizumab) and Doxil (pegylated liposomal-doxorubicin) resulted in a partial response in patients with advanced solid tumors harboring PIK3CA mutations (PMID: 21216929). 21216929
PIK3CA mutant cervical cancer decreased response Cetuximab Phase II Actionable In a Phase II clinical trial, Erbitux (cetuximab) treatment, in addition to radiochemotherapy, did not result in any complete responses (0/8) and demonstrated a worse disease free survival when compared to radiochemotherapy alone in cervical cancer patients harboring PIK3CA mutations (PMID: 25724520). 25724520
PIK3CA mutant Advanced Solid Tumor sensitive BAY1082439 Preclinical Actionable In a preclinical study, BAY1082439 induced regression in xenograft models of advanced solid tumors with PIK3CA mutations (Cancer Res April 15, 2012 72; 2799). detail...
PIK3CA mutant ovarian cancer not predictive Perifosine Phase II Actionable In a Phase II trial , Perifosine (KRX-0401) treatment was well-tolerated, resulted in stable disease as best response in patients with recurrent ovarian cancer, no significant difference in disease control rate (12.5%, 2/16 vs 40%, 2/5, p=0.22), median progression-free survival (HR=0.57, p=0.32), or median overall survival (HR=1.37, p=0.58) was identified between PIK3CA wild-type and PIK3CA mutant patients (PMID: 28864978). 28864978
PIK3CA mutant Advanced Solid Tumor sensitive VS-5584 Preclinical Actionable In preclinical studies, VS-5584 demonstrated efficacy in a variety of cancer cell lines, particularly those harboring PIK3CA mutations (PMID: 23270925). 23270925
PIK3CA mutant Her2-receptor negative breast cancer no benefit Capivasertib + Paclitaxel Phase II Actionable In a Phase II trial, the addition of AZD5363 to Taxol (paclitaxel) therapy did not improve progression-free survival compared to placebo (10.9 vs 10.8 months) in patients with PIK3CA-mutant, Esr1-positive, Erbb2 (Her2)-negative breast cancer (PMID: 30860570; NCT01625286). 30860570
PIK3CA mutant hepatocellular carcinoma decreased response Sorafenib Clinical Study - Cohort Actionable In a clinical case study, Nexavar (sorafenib) treatment of patients with hepatocellular carcinoma harboring Mtor pathway mutations in PIK3CA, PTEN, TSC2, or TSC1 (n=12), resulted in a lower disease control rate (8.3% vs. 40.2%), shorter progression-free survival (1.9 months vs. 5.3 months) and shorter overall survival (10.4 months vs. 17.9 months) compared to patients without mutations in this pathway (n=67) (PMID: 30373752; NCT01775072). 30373752
PIK3CA mutant colorectal cancer predicted - sensitive MS417 + Pictilisib Preclinical - Cell culture Actionable In a preclinical study, Pictilisib (GDC-0941) and MS417 in combination resulted in improved growth inhibition and increased cell death compared to either agent alone in colorectal cancer cell lines harboring PIK3CA mutations in culture (PMID: 26058079). 26058079
PIK3CA mutant estrogen-receptor positive breast cancer predicted - sensitive Letrozole Phase III Actionable In a Phase III trial, estrogen-receptor positive breast cancer patients harboring a PIK3CA mutation demonstrated a greater benefit when treated with Femara (letrozole) compared to Nolvadex (tamoxifen) (PMID: 29902286; NCT00004205). 29902286
PIK3CA mutant endometrial cancer no benefit Temsirolimus Phase II Actionable In a retrospective study of a Phase II trial, mutation status of PIK3CA was not associated with progression-free survival or response rate in advanced endometrial cancer patients treated with Torisel (temsirolimus) (PMID: 27016228). 27016228
PIK3CA mutant colon cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 demonstrated efficacy in promoting apoptosis of colon cancer cells harboring a PIK3CA mutation in culture (PMID: 22543857). 22543857
PIK3CA mutant lung non-small cell carcinoma sensitive PF-04691502 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-04691502 inhibited tumor growth in non-small cell lung cancer cell line xenograft models harboring PIK3CA mutations (PMID: 21750219). 21750219
PIK3CA mutant Her2-receptor negative breast cancer sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in a disease control rate of 60.9% (14/23, 0 complete response (CR), 1 partial response (PR), 13 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 17.4% (4/23) in patients with ER-positive, ERBB2 (HER2)-negative breast cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA mutant liposarcoma predicted - sensitive Copanlisib Case Reports/Case Series Actionable In a Phase II trial (NCI MATCH EAY131-Z1F), Aliqopa (copanlisib) treatment resulted in a partial response in a patient with liposarcoma harboring PIK3CA mutation (J Clin Oncol 38: 2020 (suppl; abstr 3506); NCT02465060). detail...
PIK3CA mutant hematologic cancer sensitive CUDC-907 Preclinical - Cell line xenograft Actionable In a preclinical study, CUDC-907 inhibited PIK3CA, blocked downstream AKT pathway activation, and induced apoptosis and cell cycle arrest in both PIK3CA wild-type and PIK3CA mutant human cancer cell lines and xenograft models of multiple solid and hematologic cancers (PMID: 22693356). 22693356
PIK3CA mutant endometrial cancer sensitive GDC-0980 Phase II Actionable In a Phase II trial, Apitolisib (GDC-0980) was poorly tolerated, but demonstrated efficacy in endometrial cancer patients harboring mutations in PIK3CA, PTEN, or AKT1 (J Clin Oncol 32:5s, 2014 (suppl; abstr 5513)). detail...
PIK3CA mutant triple-receptor negative breast cancer predicted - sensitive Ipatasertib + Paclitaxel Phase II Actionable In a Phase II trial, Ipatasertib (GDC-0068) in combination with Abraxane (paclitaxel) resulted in improved progression free survival (6.2 vs 4.9 months) compared to placebo in triple-receptor negative breast cancer patients harboring mutations in PIK3CA, AKT1, or PTEN (J Clin Oncol 35, 2017 (suppl; abstr 1009)). detail...
PIK3CA mutant endometrial cancer sensitive Pictilisib Preclinical Actionable In a preclinical study, endometrioid endometrial cancer cell lines harboring PIK3CA mutations demonstrated increased sensitivity to GDC-0941 (Pictilisib) induced growth inhibition comparing to PIK3CA wild-type cells in culture (PMID: 23674493). 23674493
PIK3CA mutant colon cancer sensitive PWT33597 Preclinical Actionable In a preclinical study, PWT33597 decreased signaling of mTOR and PIK3 pathways in colon cancer cells with Pik3ca mutations (Cancer Res 2011;71(8 Suppl):Abstract nr 4485). detail...
PIK3CA mutant invasive bladder transitional cell carcinoma predicted - resistant Cisplatin + Gemcitabine + Sorafenib Phase II Actionable In a Phase II trial, PIK3CA mutations were more frequent in muscle-invasive urothelial bladder cancer patients that did not respond to Nexavar (sorafenib), Platinol (cisplatin) and Gemzar (gemcitabine) combination therapy than those who did respond (J Clin Oncol 35, 2017 (suppl 6S; abstract 345)). detail...
PIK3CA mutant triple-receptor negative breast cancer sensitive Pictilisib Preclinical - Cell culture Actionable In a preclinical study, triple-receptor negative breast cancer cell lines harboring PIK3CA mutations demonstrated increased sensitivity to Pictilisib (GDC-0941) in culture (PMID: 27196766). 27196766
PIK3CA mutant clear cell adenocarcinoma predicted - sensitive Copanlisib Case Reports/Case Series Actionable In a Phase II trial (NCI MATCH EAY131-Z1F), Aliqopa (copanlisib) treatment resulted in a partial response in a patient with clear cell carcinoma of anterior abdominal wall harboring PIK3CA mutation (J Clin Oncol 38: 2020 (suppl; abstr 3506); NCT02465060). detail...
PIK3CA mutant breast cancer sensitive Letrozole + Taselisib Phase Ib/II Actionable In a Phase Ib trial, Taselisib (GDC-0032) and Femara (letrozole) combination therapy resulted in an overall response rate of 38% in breast cancer patients with PIK3CA mutations, compared to 9% in patients with wild-type PIK3CA (Cancer Res May 1, 2015 75; PD5-2). detail...
PIK3CA mutant ovarian cancer sensitive CH5132799 Preclinical Actionable In a preclinical study, CH5132799 inhibited proliferation of ovarian cancer cells with PIK3CA mutations in culture (PMID: 21558396). 21558396
PIK3CA mutant stomach cancer sensitive BAY1082439 Preclinical Actionable In a preclinical study, BAY1082439 inhibited growth of gastric cancer cells harboring PIK3CA mutations in culture and xenograft models (AACR; 2015. Abstract nr 2674). detail...
PIK3CA mutant lung non-small cell carcinoma sensitive PWT33597 Preclinical Actionable In a preclinical study, PWT33597 decreased signaling of mTOR and PIK3 pathways in non-small cell lung cancer cells with Pik3ca mutations (Cancer Res 2011;71(8 Suppl):Abstract nr 4485). detail...
PIK3CA mutant breast cancer sensitive Pictilisib Preclinical Actionable In a preclinical study, GDC-0941 inhibited growth and induced apoptosis in breast cancer cells expressing PIK3CA mutations (PMID: 24601221). 24601221
PIK3CA mutant Advanced Solid Tumor sensitive MLN1117 Preclinical Actionable In a preclinical study, MLN1117 inhibited proliferation of tumor cells harboring oncogenic PIK3CA mutations in culture and inhibited tumor growth in PIK3CA mutant xenograft models (Cancer Ther 2011;10(11 Suppl):Abstract nr A171). detail...
PIK3CA mutant Advanced Solid Tumor sensitive MLN1117 Phase I Actionable In a Phase I trial, MLN1117 demonstrated safety and preliminary efficacy in a variety of patients with advanced solid tumors carrying PIK3CA mutations (Journal of Clinical Oncology, 2015 ASCO Annual Meeting Vol 33, No 15_suppl., 2015: 2501). detail...
PIK3CA mutant gastric adenocarcinoma sensitive Fluorouracil + PI-103 Preclinical Actionable In a preclinical study, PI-103 enhanced sensitivity to 5-FU in gastric cancer cells with mutant PIK3CA (PMID: 22336586). 22336586
PIK3CA mutant cervical cancer not predictive Perifosine Phase II Actionable In a Phase II trial , Perifosine (KRX-0401) treatment was well-tolerated, resulted in stable disease as best response in patients with recurrent cervical cancer, no significant difference in disease control rate (11.1%, 2/18 vs 25%, 2/8, p=0.56), median progression-free survival (HR=1.06, p=0.81), or median overall survival (HR=1.66, p=0.26) was identified between PIK3CA wild-type and PIK3CA mutant patients (PMID: 28864978). 28864978
PIK3CA mutant ovarian cancer sensitive Temsirolimus Phase I Actionable In a Phase I clinical trial, Torisel (temsirolimus) demonstrated limited efficacy in ovarian cancer patients with PIK3CA mutations (PMID: 22271473). 22271473
PIK3CA mutant ovarian cancer sensitive Temsirolimus Case Reports/Case Series Actionable In a clinical study, an ovarian cancer patient harboring a PIK3CA E542 mutation demonstrated stable disease when treated with Torisel (temsirolimus) (PMID: 21216929). 21216929
PIK3CA mutant ovarian cancer sensitive Buparlisib + Olaparib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Buparlisib (BKM120) and Lynparza (olaparib) worked synergistically to induce apoptosis and inhibit growth of ovarian cancer cell lines harboring PIK3CA mutations in culture, and induced apoptosis and decreased proliferation of PIK3CA-mutant ovarian cancer cell line xenograft models (PMID: 26909613). 26909613
PIK3CA mutant Advanced Solid Tumor sensitive AT-7867 Preclinical Actionable In a preclinical study, AT-7867 inhibited proliferation in several human tumor cell lines harboring PIK3CA mutations in culture (PMID: 20423992). 20423992
PIK3CA mutant Advanced Solid Tumor sensitive A-443654 Preclinical Actionable In a preclinical study, cells expressing mutant PIK3CA demonstrated increased sensitivity to A-443654 compared to cells expressing wild-type PIK3CA (PMID: 19208828). 19208828
PIK3CA mutant head and neck squamous cell carcinoma predicted - sensitive Carboplatin + Paclitaxel + Temsirolimus Phase II Actionable In a Phase II trial, two patients with head and neck squamous cell carcinoma harboring a PIK3CA mutation who had stable disease demonstrated some tumor regression when treated with the combination of Torisel (temsirolimus), Paraplatin (carboplatin), and Taxol (paclitaxel) (PMID: 28961834). 28961834
PIK3CA mutant endometrial cancer sensitive Alpelisib Case Reports/Case Series Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in 1 complete response and 1 partial response in patients with endometrial cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA mutant uterine cancer predicted - sensitive Copanlisib Case Reports/Case Series Actionable In a Phase II trial (NCI MATCH EAY131-Z1F), Aliqopa (copanlisib) treatment resulted in a partial response in a patient with uterine cancer harboring PIK3CA mutation (J Clin Oncol 38: 2020 (suppl; abstr 3506); NCT02465060). detail...
PIK3CA mutant endometrial cancer sensitive CH5132799 Preclinical Actionable In a preclinical study, CH5132799 inhibited proliferation of endometrial cancer cells with PIK3CA mutations in culture (PMID: 21558396). 21558396
PIK3CA mutant breast cancer sensitive CH5132799 Preclinical Actionable In a preclinical study, breast cancer cells with PIK3CA mutations, both with and without ERBB2 (HER2) amplification, demonstrated sensitivity to CH5132799 in culture (PMID: 21558396). 21558396
PIK3CA mutant stomach cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 demonstrated efficacy in promoting apoptosis of gastric cancer cells harboring a PIK3CA mutation in culture (PMID: 22543857). 22543857
PIK3CA mutant endometrioid ovary carcinoma sensitive Capivasertib Phase I Actionable In a Phase I clinical trial, AZD5363 demonstrated safety and efficacy in patients with advanced solid tumors, including prolonged stable disease in one patient with a PIK3CA-mutant endometrioid ovarian cancer (American Association for Cancer Research; 06 Apr 2013-10 Apr 2013; Abstract LB-66). detail...
PIK3CA mutant colorectal cancer sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in a disease control rate of 34.3% (12/35, 0 complete response (CR), 2 partial response (PR), 10 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 8.6% (3/35) in patients with colorectal cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA mutant triple-receptor negative breast cancer predicted - sensitive Capivasertib + Paclitaxel Phase II Actionable In a Phase II trial (PAKT), Capivasertib (AZD5363) in combination with Taxol (paclitaxel) as first-line therapy resulted in improved median progression-free survival (9.3 vs 3.7 months, HR=0.30, p=0.01) compared to placebo in patients with metastatic triple-negative breast cancer harboring PIK3CA, AKT1, and/or PTEN mutations, but not in patients with wile-type PIK3CA, AKT1, and PTEN (5.3 vs 4.4 months, HR=1.13, p=0.61) (PMID: 31841354; NCT02423603). 31841354
PIK3CA mutant female reproductive organ cancer predicted - sensitive Capivasertib Phase I Actionable In a Phase I trial, treatment with AZD5363 was well-tolerated and resulted in tumor shrinkage in 56% (14/25) patients with PIK3CA-mutant gynecological cancers, however, the response rate was modest with confirmed RECIST responses in 8% (2/26) of patients (PMID: 29066505; NCT01226316). 29066505
PIK3CA mutant ovarian cancer sensitive OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 inhibited tumor growth in ovarian cancer cell line xenograft models harboring PIK3CA mutations (PMID: 21673091). 21673091
PIK3CA mutant Advanced Solid Tumor sensitive M2698 Preclinical Actionable In a preclinical study, MSC2363318A demonstrated anti-proliferative activity against many solid tumor cell lines with PI3K pathway genomic alterations, and inhibited tumor growth in several human cancer xenograft models of breast, pancreatic, glioblastoma and ovarian cancers (Mol Cancer Ther 2013;12(11 Suppl):A162). detail...
PIK3CA mutant estrogen-receptor positive breast cancer no benefit Fulvestrant Phase II Actionable In a Phase II trial, Falsodex (fulvestrant) treatment resulted in similar progression free survival in ER positive breast cancer patients harboring PIK3CA mutations and those who were PIK3CA wild-type (PMID: 27174596). 27174596
PIK3CA mutant stomach cancer predicted - sensitive MLN1117 Case Reports/Case Series Actionable In a Phase I trial, MLN1117 (INK1117) treatment resulted in a partial response in one patient with gastric cancer harboring a PIK3CA mutation (PMID: 28490463; NCT01449370). 28490463
PIK3CA mutant Advanced Solid Tumor predicted - sensitive Uprosertib Phase I Actionable In a Phase I clinical trial, GSK2141795 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with 4 patients with PIK3CA mutations and/or PTEN loss achieving stable disease for greater than 6 months (J Clin Oncol 29: 2011 (suppl; abstr 3003)). detail...
PIK3CA mutant breast adenocarcinoma sensitive Acalisib Preclinical - Cell line xenograft Actionable In a preclinical study, Acalisib (GS-9820) resulted in tumor growth inhibition in xenograft models of breast adenocarcinoma harboring a PIK3CA mutation (Mol Cancer Ther 2009;8(12 Suppl):B136). detail...
PIK3CA mutant Her2-receptor positive breast cancer sensitive CCT128930 Preclinical - Cell line xenograft Actionable In a preclinical study, CCT128930 induced growth arrest and inhibited tumor growth in a xenograft model of a ERBB2 (HER2)-positive human breast cancer cell line harboring a PIK3CA mutation (PMID: 21191045). 21191045
PIK3CA mutant Advanced Solid Tumor predicted - sensitive Copanlisib Phase II Actionable In a Phase II trial (NCI MATCH EAY131-Z1F), Aliqopa (copanlisib) treatment resulted in an objective response rate of 11% (3/28, 3 partial response) and a clinical benefit rate of 32% (9/28) in patients with advanced solid tumors harboring PIK3CA mutations (J Clin Oncol 38: 2020 (suppl; abstr 3506); NCT02465060). detail...
PIK3CA mutant breast cancer sensitive CUDC-907 Preclinical Actionable In a preclinical study, CUDC-907 inhibited growth of human breast cancer cell lines harboring PIK3CA mutations in culture (PMID: 22693356). 22693356
PIK3CA mutant prostate cancer sensitive CH5132799 Preclinical Actionable In a preclinical study, CH5132799 inhibited proliferation of prostate cancer cells with PIK3CA mutations in culture (PMID: 21558396). 21558396
PIK3CA mutant Advanced Solid Tumor no benefit Alpelisib + Infigratinib Phase Ib/II Actionable In a Phase Ib trial, treatment with the combination of Piqray (alpelisib) and Infigratinib (BGJ398) resulted in partial response (PR) in 9.7% (6/62) and disease control (PR or stable disease) in 61.3% (34/62) of patients with advanced solid tumors harboring a PIK3CA mutation with or without an FGFR alteration, however, the efficacy was not deemed great enough to pursue further development (JCO Precision Oncology 2019 :3, 1-13). detail...
PIK3CA mutant breast cancer sensitive AT13148 Preclinical - Cell line xenograft Actionable In a preclinical study, a ERBB3 (HER3) positive breast cancer cell line xenograft model harboring a PIK3CA mutation was demonstrated decreased tumor growth when treated with AT13148 (PMID: 22781553). 22781553
PIK3CA mutant breast cancer predicted - sensitive GDC-0077 Preclinical - Pdx & cell culture Actionable In a preclinical study, GDC-0077 inhibited proliferation and induced apoptosis in PIK3CA-mutant breast cancer cell lines in culture, and induced apoptosis and tumor regression in PIK3CA-mutant breast cancer cell line and patient-derived xenograft models (AACR; Cancer Res 2017;77(13 Suppl):Abstract nr 156). detail...
PIK3CA mutant Advanced Solid Tumor sensitive CUDC-907 Preclinical - Cell line xenograft Actionable In a preclinical study, CUDC-907 inhibited PIK3CA, blocked downstream AKT pathway activation, and induced apoptosis and cell cycle arrest in both PIK3CA wild-type and PIK3CA mutant human cancer cell lines and xenograft models of multiple tumor types (PMID: 22693356). 22693356
PIK3CA mutant Advanced Solid Tumor sensitive GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in partial response in 21% (3/14) and stable disease in 57% (8/14) of patients with advanced solid tumors harboring PIK3CA mutations (PMID: 26787751). 26787751
PIK3CA mutant Advanced Solid Tumor sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) demonstrated safety and efficacy in patients with advanced solid tumor harboring PIK3CA mutations, resulted in a disease control rate of 58.2% (78/134, 1 complete response (CR), 7 partial response (PR), 70 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 15.7% (21/134) (PMID: 29401002; NCT01219699). 29401002
PIK3CA mutant Advanced Solid Tumor sensitive Alpelisib Preclinical - Cell line xenograft Actionable In a preclinical study, PIK3CA mutations were associated with sensitivity to Alpelisib (BYL719) in human tumor cell lines in culture and in xenograft models (PMID: 24608574). 24608574
PIK3CA mutant Advanced Solid Tumor sensitive Onatasertib Phase I Actionable In a Phase I trial, CC-223 demonstrated safety and preliminary efficacy in patients with solid tumors, including stable disease for greater than 110 days in 2 patients with PIK3CA mutation or PTEN deletion (PMID: 26177599). 26177599 detail...
PIK3CA mutant endometrial cancer not predictive Perifosine Phase II Actionable In a Phase II trial , Perifosine (KRX-0401) treatment was well-tolerated, resulted in stable disease as best response in patients with recurrent endometrial cancer, no significant difference in disease control rate (47.1%, 8/17 vs 14.3%, 1/7, p=0.19), median progression-free survival (HR=1.71, p=0.30), or median overall survival (HR=0.92, p=0.88) was identified between PIK3CA wild-type and PIK3CA mutant patients (PMID: 28864978). 28864978
PIK3CA mutant Advanced Solid Tumor sensitive Carboxyamidotriazole Orotate Phase I Actionable In a Phase I clinical trial, carboxyamidotriazole orotate treatment demonstrated some efficacy in patients with various solid tumors, including patients harboring PIK3CA mutations (J Clin Oncol 31, 2013 (suppl; abstr 2518)). detail...
PIK3CA mutant head and neck squamous cell carcinoma sensitive Radiotherapy + Taselisib Preclinical - Pdx & cell culture Actionable In a preclinical study, Taselisib (GDC-0032) enhanced the effects of radiation induced apoptosis in HNSCC cell lines, cell line xenograft models, and a patient derived xenograft (PDX) model, all with PIK3CA mutations (PMID: 26589432). 26589432
PIK3CA mutant breast cancer no benefit MK2206 Phase II Actionable In a Phase II trial, MK2206 demonstrated limited clinical efficacy in patients with advanced breast cancer harboring mutations in PIK3CA (n=14) or AKT1 (n=4), resulted in an objective response rate of 5.6% (1/18, 1 partial response) and a 6-month progression-free survival rate of 5.6%, with no significant target inhibition in tumor biopsies (PMID: 31277699; NCT01277757). 31277699
PIK3CA mutant squamous cell carcinoma sensitive Carboxyamidotriazole Orotate Phase I Actionable In a Phase I clinical trial, carboxyamidotriazole orotate treatment demonstrated some efficacy in patients with various solid tumors including squamous cell carcinomas (tonsil) with PI3KCA and NRAS mutations (J Clin Oncol 31, 2013 (suppl; abstr 2518)). detail...
PIK3CA mutant head and neck cancer sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in a disease control rate of 68.4% (13/19, 0 complete response (CR), 0 partial response (PR), 13 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 10.5% (2/19) in patients with head and neck cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA mutant lung squamous cell carcinoma no benefit Capivasertib Case Reports/Case Series Actionable In a Phase II trial (NLMT), Capivasertib (AZD5363) treatment did not result in a conformed response (0/4) or durable clinical benefit (0/4) in patients with squamous cell lung cancer harboring PIK3CA mutations, thus the cohort was closed due to futility (PMID: 32669708, NCT02664935). 32669708
PIK3CA mutant cervical cancer predicted - sensitive Alpelisib Case Reports/Case Series Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in partial response in 3 patients with cervical cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA mutant lung non-small cell carcinoma predicted - sensitive Sulindac Preclinical - Cell culture Actionable In a preclinical study, non-small cell lung cancer cell lines harboring either a PIK3CA mutation or PIK3CA amplification demonstrated increased sensitivity to Clinoril (sulindac) compared to cell lines with wild-type PIK3CA, resulting in decreased proliferation in culture (PMID: 30683736). 30683736
PIK3CA mutant triple-receptor negative breast cancer sensitive Palbociclib + Pictilisib Preclinical Actionable In a preclinical study, Ibrance (palbociclib) and Pictilisib (GDC-0941) worked synergistically to inhibit the survival of triple-receptor negative breast cancer cell lines harboring PIK3CA mutations in culture (PMID: 27020857). 27020857
PIK3CA mutant estrogen-receptor positive breast cancer no benefit Fulvestrant + Pictilisib Phase II Actionable In a Phase II trial, Falsodex (fulvestrant) and Pictilisib (GDC-0941) combination treatment resulted in similar progression free survival in ER positive breast cancer patients harboring PIK3CA mutations and those who were PIK3CA wild-type (PMID: 27174596). 27174596
PIK3CA mutant lung squamous cell carcinoma no benefit Taselisib Phase I Actionable In a Phase I (SWOG S1400B) trial, Taselisib (GDC-0032) treatment did not meet primary endpoint in patients with lung squamous cell carcinoma harboring PIK3CA mutations, with only 1 responder in the primary analysis population (n=21) (PMID: 31158500; NCT02785913). 31158500
PIK3CA mutant breast cancer predicted - sensitive CYH33 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cell lines harboring PIK3CA mutations demonstrated increased sensitivity to CYH33 compared to cell lines with wild-type PIK3CA in culture (p<0.01) (PMID: 30003928). 30003928
PIK3CA mutant Her2-receptor negative breast cancer no benefit Everolimus Phase III Actionable In a retrospective analysis of a Phase III trial (BOLERO-2), the presence of PIK3CA mutations did not significantly affect the efficacy of Afinitor (everolimus) in patients with hormone receptor-positive, Erbb2 (Her2)-negative breast cancer, with a HR for progression-free survival of 0.37 in PIK3CA wild-type group and a HR of 0.51 (p=0.35) in patients with PIK3CA mutations (PMID: 26503204; NCT00863655). 26503204
PIK3CA mutant Advanced Solid Tumor sensitive Sapanisertib Preclinical Actionable In a preclinical study, human cancer cell lines harboring PIK3CA mutations were sensitive to Sapanisertib (MLN0128) as demonstrated by significant growth inhibition (PMID: 25261369). 25261369
PIK3CA mutant breast cancer no benefit Temsirolimus Phase II Actionable In a Phase II trial, Torisel (temsirolimus) treatment resulted in stable disease in 9.7% (3/31) of breast cancer patients positive for ER, PR or ERBB2 (HER2), however there was no association between PIK3CA mutational status and clinical benefits (PMID: 22245973). 22245973
PIK3CA mutant breast cancer predicted - sensitive Capivasertib Preclinical Actionable In a preclinical study, AZD5363 inhibited growth in breast cancer cell lines and xenograft models expressing PIK3CA mutations (PMID: 22294718). 22294718
PIK3CA mutant breast cancer predicted - sensitive Capivasertib Phase I Actionable In a Phase I trial, treatment with AZD5363 was well-tolerated and resulted in tumor shrinkage in 46% (12/26) patients with PIK3CA-mutant breast cancer, with confirmed RECIST responses in 4% (1/28) of patients (PMID: 29066505; NCT01226316). 29066505
PIK3CA mutant breast cancer sensitive Sapanisertib Preclinical Actionable In a preclinical study, Sapanisertib (MLN0128) demonstrated efficacy in PIK3CA mutant breast cancer xenograft models (PMID: 23085766). 23085766
PIK3CA mutant lung adenocarcinoma no benefit Capivasertib Case Reports/Case Series Actionable In a Phase II trial (NLMT), Capivasertib (AZD5363) treatment did not result in a conformed response (0/8) or durable clinical benefit (0/8) in patients with lung adenocarcinoma harboring mutations in PIK3CA, PTEN, or AKT, thus the cohort was closed due to futility (PMID: 32669708, NCT02664935). 32669708
PIK3CA mutant Her2-receptor positive breast cancer not predictive Ado-trastuzumab emtansine Phase III Actionable In a retrospective analysis of a Phase III trial (KATHERINE), adjuvant Kadcyla (ado-trastuzumab emtansine) treatment demonstrated similar invasive disease-free survival benefit over Herceptin (trastuzumab) in PIK3CA mutant (HR=0.54) and PIK3CA wild-type (HR=0.48) patients with Erbb2 (Her2)-positive breast cancer who had residual invasive disease after neoadjuvant therapy (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 502-502; NCT01772472). detail...
PIK3CA mutant Advanced Solid Tumor sensitive PX-866 Phase I Actionable In a Phase I trial, PX-866 demonstrated efficacy and was well tolerated in patients with advanced solid tumor with both PIK3CA wild-type and mutant status (PMID: 22693357). 22693357
PIK3CA mutant Advanced Solid Tumor sensitive Taselisib Phase I Actionable In a Phase I trial, Taselisib (GDC-0032) demonstrated activity in patients with PIK3CA-mutant advanced solid tumors, in the absence of alterations in the MAPK or PTEN pathways (Cancer Res October 1, 2014 74:915). detail...
PIK3CA mutant breast cancer sensitive Alpelisib + GSK2334470 Preclinical - Cell line xenograft Actionable In a preclinical study, GSK2334470 resensitized Alpelisib (BYL719)-resistant breast cancer cell lines harboring PIK3CA mutations to Alpelisib (BYL719) in culture and in cell line xenograft models (PMID: 27451907). 27451907
PIK3CA mutant breast cancer sensitive AZD8835 Preclinical Actionable In a preclinical study, AZD8835 inhibited tumor growth in breast cancer xenograft models harboring PIK3CA mutations (AACR; 2015. Abstract nr 2665). detail...
PIK3CA mutant breast cancer sensitive OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 induced tumor regression in breast cancer cell line xenograft models harboring PIK3CA mutations (PMID: 21673091). 21673091
PIK3CA mutant breast cancer predicted - sensitive MLN1117 Case Reports/Case Series Actionable In a Phase I trial, MLN1117 (INK1117) treatment resulted in partial response in 3 patients with breast cancer harboring PI3CA mutations (PMID: 28490463; NCT01449370). 28490463
PIK3CA mutant endometrial carcinoma no benefit Copanlisib Phase II Actionable In a Phase II (NRG-GY008) trial, Aliqopa (copanlisib) was well tolerated but demonstrated limited efficacy, resulted in stable disease as best response in 54.5% (6/11) of patients with endometrial carcinoma harboring PIK3CA mutations, with a median progression-free survival of 2.8 months and a median overall survival of 15.2 months (PMID: 31934607). 31934607
PIK3CA mutant estrogen-receptor positive breast cancer sensitive AZD8835 Preclinical Actionable In a preclinical study, AZD8835 inhibited proliferation of estrogen receptor (ER)-positive breast cancer cells harboring PIK3CA mutations in culture and suppressed tumor growth in xenograft models (PMID: 26839307). 26839307
PIK3CA mutant Advanced Solid Tumor sensitive XL147 Phase I Actionable In a Phase I clinical trial, XL147 (SAR24508) was shown to be safe and potentially efficacious, with clinical activity seen irrespective of tumor PI3K pathway molecular alterations (PMID: 24166903). 24166903
PIK3CA mutant Advanced Solid Tumor sensitive XL147 Preclinical Actionable In a preclinical study, tumor cell lines with PIK3CA mutations demonstrated sensitivity to XL147 in culture (PMID: 25637314). 25637314
PIK3CA mutant Advanced Solid Tumor sensitive AZD8835 Preclinical Actionable In a preclinical study, AZD8835 inhibited growth of a variety of advanced solid tumor models with Pik3ca mutations in culture (PMID: 26839307). 26839307
PIK3CA mutant endometrial cancer no benefit MK2206 Phase II Actionable In a Phase II trial, MK2226 demonstrated limited benefit in endometrial cancer patients regardless of PIK3CA status, with partial responses in 6% (2/37) of patients, 19% (7/37; 5 with serous histology) remaining progression-free for at least 6 mo, and a median progression-free survival (PFS) of 2.0 mo in the overall population, and partial responses in 11% (11/9), 33% (3/9) progression-free for 6 mo, and a median PFS of 1.7 mo in the group of patients with PIK3CA mutations (PMID: 31714586; NCT01307631). 31714586
Clinical Trial Phase Therapies Title Recruitment Status
NCT03842228 Phase I Copanlisib + Durvalumab + Olaparib Copanlisib, Olaparib, and Durvalumab in Treating Patients With Metastatic or Unresectable Solid Tumors Recruiting
NCT01219699 Phase I Alpelisib Alpelisib + Fulvestrant A Study of BYL719 in Adult Patients With Advanced Solid Malignancies, Whose Tumors Have an Alteration of the PIK3CA Gene Completed
NCT02576444 Phase II Adavosertib + Olaparib Olaparib Capivasertib + Olaparib AZD6738 + Olaparib OLAParib COmbinations (OLAPCO) Active, not recruiting
NCT03056755 Phase II Alpelisib + Fulvestrant Alpelisib + Letrozole Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole, Based on Prior Endocrine Therapy, in Patients With PIK3CA Mutation With Advanced Breast Cancer Who Have Progressed on or After Prior Treatments (BYLieve) Recruiting
NCT04191499 Phase II Fulvestrant + Palbociclib Fulvestrant + GDC-0077 + Palbociclib A Study Evaluating the Efficacy and Safety of GDC-0077 + Palbociclib + Fulvestrant vs Placebo + Palbociclib + Fulvestrant in Patients With PIK3CA-Mutant, Hormone Receptor-Positive, Her2-Negative, Locally Advanced or Metastatic Breast Cancer Recruiting
NCT02465060 Phase II Erdafitinib Copanlisib Trametinib Crizotinib Sunitinib Sapanisertib Nivolumab AZD4547 Dasatinib Pertuzumab + Trastuzumab Dabrafenib + Trametinib Binimetinib Adavosertib Osimertinib Palbociclib Afatinib Capivasertib Defactinib GSK2636771 Vismodegib Ado-trastuzumab emtansine Larotrectinib Taselisib Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial) Recruiting
NCT01776008 Phase II Goserelin + MK2206 Akt Inhibitor MK-2206 and Anastrozole With or Without Goserelin Acetate in Treating Patients With Stage II-III Breast Cancer Terminated
NCT01791478 Phase I Alpelisib + Letrozole BYL719 and Letrozole in Post-Menopausal Patients With Hormone Receptor-Positive Metastatic Breast Cancer Active, not recruiting
NCT02124148 Phase I Cisplatin + Prexasertib Cetuximab + Prexasertib Pemetrexed Disodium + Prexasertib LY3023414 + Prexasertib Fluorouracil + Leucovorin + Prexasertib A Study of Prexasertib (LY2606368) With Chemotherapy or Targeted Agents in Participants With Advanced Cancer Completed
NCT01226316 Phase I Capivasertib Safety, Tolerability & Potential Anti-cancer Activity of Increasing Doses of AZD5363 in Different Treatment Schedules Active, not recruiting
NCT02189174 Phase Ib/II CLR457 Study of CLR457 Administered Orally in Adult Patients With Advanced Solid Malignancies Terminated
NCT02167854 Phase I Alpelisib + Elgemtumab + Trastuzumab Open-Label Study Evaluating the Safety and Tolerability of LJM716, BYL719 and Trastuzumab in Patients With Metastatic HER2+ Breast Cancer Active, not recruiting
NCT01470209 Phase I Buparlisib + Everolimus A Phase I Study of BKM120 and Everolimus in Advanced Solid Malignancies Completed
NCT03218826 Phase I AZD8186 + Docetaxel PI3Kbeta Inhibitor AZD8186 and Docetaxel in Treating Patients Advanced Solid Tumors With PTEN or PIK3CB Mutations That Are Metastatic or Cannot Be Removed by Surgery Recruiting
NCT02987959 Phase II Sapanisertib Study of TAK-228 (MLN0128) in Soft Tissue Sarcomas Active, not recruiting
NCT04208178 Phase III Pertuzumab + Trastuzumab Alpelisib + Pertuzumab + Trastuzumab Study of Alpelisib (BYL719) in Combination With Trastuzumab and Pertuzumab as Maintenance Therapy in Patients With HER2-positive Advanced Breast Cancer With a PIK3CA Mutation Recruiting
NCT03297606 Phase II Bosutinib Palbociclib Vismodegib Ipilimumab + Nivolumab Cobimetinib + Vemurafenib Temsirolimus Olaparib Erlotinib Crizotinib Sunitinib Afatinib Dasatinib Pertuzumab + Trastuzumab Axitinib Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR) (CAPTUR) Recruiting
NCT03675893 Phase II Abemaciclib + LY3023414 Abemaciclib + Letrozole + LY3023414 A Phase 2 Study of LY3023414 and Abemaciclib With or Without Letrozole in Endometrial Cancer Recruiting
NCT02340221 Phase III Fulvestrant + Taselisib Fulvestrant A Study of Taselisib + Fulvestrant Versus Placebo + Fulvestrant in Participants With Advanced or Metastatic Breast Cancer Who Have Disease Recurrence or Progression During or After Aromatase Inhibitor Therapy (SANDPIPER) Active, not recruiting
NCT02861300 Phase Ib/II Capecitabine + Telaglenastat CB-839 + Capecitabine in Solid Tumors and Fluoropyrimidine Resistant PIK3CA Mutant Colorectal Cancer Recruiting
NCT03006172 Phase I GDC-0077 + Letrozole + Palbociclib GDC-0077 Fulvestrant + GDC-0077 GDC-0077 + Letrozole To Evaluate the Safety, Tolerability, and Pharmacokinetics of GDC-0077 Single Agent in Participants With Solid Tumors and in Combination With Endocrine and Targeted Therapies in Participants With Breast Cancer Recruiting
NCT01306045 Phase II Erlotinib Lapatinib MK2206 Sunitinib Selumetinib Molecular Profiling and Targeted Therapy for Advanced Non-Small Cell Lung Cancer, Small Cell Lung Cancer, and Thymic Malignancies Active, not recruiting
NCT02728258 Phase II Copanlisib Copanlisib in Treating Patients With Persistent or Recurrent Endometrial Cancer Active, not recruiting
NCT02315625 Phase II Everolimus Sunitinib Study of Mutation-Targeted Therapy With Sunitinib or Everolimus in People With Advanced Low- or Intermediate-Grade Neuroendocrine Tumors of the Gastrointestinal Tract and Pancreas With or Without Cytoreductive Surgery Completed
NCT02961283 Phase I ASN003 Study of ASN003 in Subjects With Advanced Solid Tumors Terminated
NCT03834740 Phase I Everolimus + Ribociclib Ph0/2 Ribociclib & Everolimus Recruiting
NCT01437566 Phase II GDC-0980 Fulvestrant Pictilisib Study of GDC-0941 or GDC-0980 With Fulvestrant Versus Fulvestrant in Advanced or Metastatic Breast Cancer in Patients Resistant to Aromatase Inhibitor Therapy Completed
NCT04192981 Phase I GDC-0084 GDC-0084 With Radiation Therapy for People With PIK3CA-Mutated Solid Tumor Brain Metastases or Leptomeningeal Metastases Recruiting
NCT03767335 Phase I CH5132799 + Fulvestrant + Trastuzumab CH5132799 + Trastuzumab MEN1611 With Trastuzumab (+/- Fulvestrant) in Metastatic Breast Cancer (B-PRECISE-01) Recruiting
NCT02644122 Phase II SF1126 SF1126 in Recurrent or Progressive SCCHN and Mutations in PIK3CA Gene and/or PI-3 Kinase Pathway Genes Terminated
NCT03337724 Phase II Ipatasertib + Paclitaxel Paclitaxel A Study of Ipatasertib in Combination With Paclitaxel as a Treatment for Participants With PIK3CA/AKT1/PTEN-Altered, Locally Advanced or Metastatic, Triple-Negative Breast Cancer or Hormone Receptor-Positive, HER2-Negative Breast Cancer (IPATunity130) Recruiting
NCT00770263 Phase I Erlotinib + Temsirolimus Erlotinib and Temsirolimus for Solid Tumors Completed
NCT03032406 Phase II Everolimus + Hydroxychloroquine Everolimus Hydroxychloroquine CLEVER Pilot Trial: A Phase II Pilot Trial of HydroxyChLoroquine, EVErolimus or the Combination for Prevention of Recurrent Breast Cancer Active, not recruiting
NCT01928459 Phase I Alpelisib + Infigratinib Phase 1b Trial of BGJ398/BYL719 in Solid Tumors Completed
NCT01971515 Phase I M2698 First-in-Human Dose Escalation Trial in Subjects With Advanced Malignancies Completed
NCT02646319 Phase I Nab-Rapamycin Nanoparticle Albumin-Bound Rapamycin in Treating Patients With Advanced Cancer With mTOR Mutations Completed
NCT03065062 Phase I Gedatolisib + Palbociclib Study of the CDK4/6 Inhibitor Palbociclib (PD-0332991) in Combination With the PI3K/mTOR Inhibitor Gedatolisib (PF-05212384) for Patients With Advanced Squamous Cell Lung, Pancreatic, Head & Neck and Other Solid Tumors Recruiting
NCT01708161 Phase I Alpelisib + Ganitumab A Phase Ib/II Study of the Combination of BYL719 Plus AMG 479 in Adult Patients With Selected Solid Tumors Terminated
NCT01396408 Phase II Temsirolimus Sunitinib A Phase II Study of Sunitinib or Temsirolimus in Patients With Advanced Rare Tumours Active, not recruiting