PIK3CA

Chr 3

phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha

Also known as: CCM4, CLAPO, CLOVE, CWS5, HMH, MCAP, MCM, MCMTC

The protein encoded by PIK3CA is the catalytic subunit of phosphatidylinositol 3-kinase that phosphorylates phosphoinositides using ATP, functioning in critical cellular signaling pathways. Somatic gain-of-function mutations cause multiple overgrowth and vascular malformation syndromes including CLOVE syndrome, megalencephaly-capillary malformation-polymicrogyria syndrome, and CLAPO syndrome, while germline mutations cause Cowden syndrome 5 with autosomal dominant inheritance. The pathogenic mechanism involves hyperactivation of the PI3K-AKT-mTOR pathway leading to increased cell growth and proliferation.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, Mechanism
GOFmechanismLOEUF 0.1215 OMIM phenotypes
VCEP Guidelines: Brain MalformationsReleased
View SpecificationsClinGen Panel
Clinical SummaryPIK3CA
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Gene-Disease Validity (ClinGen)
hereditary breast carcinoma · ADRefuted

Refuted — evidence has disproved this relationship

3 total gene-disease associations curated

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
6 unique Pathogenic / Likely Pathogenic· 92 VUS of 200 total submissions
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available
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GeneReview available — PIK3CA
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.12LOEUF
pLI 1.000
Z-score 7.21
OE 0.05 (0.020.12)
Highly constrained

Among the most LoF-intolerant genes (~top 3%)

Missense Constraint
5.60Z-score
OE missense 0.34 (0.300.38)
191 obs / 565.7 exp
Constrained

Extremely missense-constrained (top ~0.01%)

Observed / Expected Ratios
LoF OE0.05 (0.020.12)
00.351.4
Missense OE0.34 (0.300.38)
00.61.4
Synonymous OE0.78
01.21.6
LoF obs/exp: 3 / 66.4Missense obs/exp: 191 / 565.7Syn Z: 2.36
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitivePIK3CA-related overgrowth spectrum disorder with or without megalencephaly, capillary malformation, polymicrogyria and lipomatous overgrowthGOFAD
DN
0.3097th %ile
GOF
0.4776th %ile
LOF
0.69top 10%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median · LOEUF 0.12

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

200 submitted variants in ClinVar

Classification Summary

Pathogenic2
Likely Pathogenic4
VUS92
Likely Benign72
2
Pathogenic
4
Likely Pathogenic
92
VUS
72
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
1
1
0
2
Likely Pathogenic
0
4
0
0
4
VUS
1
90
0
1
92
Likely Benign
0
1
3
68
72
Benign
0
0
0
0
0
Total196469170

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

PIK3CA · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Lymphoma, Non-HodgkinMultiple MyelomaAdvanced Solid Tumors

Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR)

RECRUITING
NCT03297606Phase PHASE2Canadian Cancer Trials GroupStarted 2018-03-23
OlaparibDasatinibNivolumab plus Ipilimumab
Polycythemia VeraEssential ThrombocythaemiaMyelofibrosis

Prevalence Of Germline Gene Mutations In Patients With Myeloproliferative Neoplasms With Family History

NOT YET RECRUITING
NCT06923670Phase NAFondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2025-05-21
NGS testingNGS analysis for mutations in genes involved in familial predisposition to hematological malignancies
Lung Cancer

Feasibility of Targeted Bronchial Washing for Molecular Testing by Next Generation Sequencing in Early-stage Lung Cancer

ACTIVE NOT RECRUITING
NCT06301295Phase NAPusan National University HospitalStarted 2024-05-29
Ultarthin bronchoscopy with intratumoral washing
Slow-Flow Vascular MalformationFast-Flow Vascular MalformationVascular Malformations

A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

RECRUITING
NCT05983159Phase PHASE2Murdoch Childrens Research InstituteStarted 2024-09-13
AlpelisibMirdametinib
Locally Advanced Breast CancerMetastatic Breast Cancer

A Study to Evaluate the Effectiveness and Safety of Inavolisib in Participants With Endocrine-resistant, PIK3CA-mutated, Hormone Receptor-positive, HER2-negative Locally Advanced or Metastatic Breast Cancer

RECRUITING
NCT07347600Hoffmann-La RocheStarted 2026-01-21
InavolisibPalbociclibFulvestrant
Breast Cancer

Elacestrant and Exemestane for Patients With Pretreated HR+/HER2- Metastatic Breast Cancer and [18F] FES-avid Lesions (COMBINE)

NOT YET RECRUITING
NCT07395336Phase PHASE2European Institute of OncologyStarted 2026-04
elacestrant and exemestane
HER2-positive Metastatic Breast CancerFirst-line Treatment

Disitamab Vedotin + Pyrotinib Versus THP in the First-line Treatment for HER2+ Advanced Breast Cancer Clinical Trial

RECRUITING
NCT06278870Phase PHASE3Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityStarted 2023-09-06
disitamab vedotinPyrotinibtrastuzumab
Breast Cancer

A Study of LOXO-783 in Patients With Breast Cancer/Other Solid Tumors

ACTIVE NOT RECRUITING
NCT05307705Phase PHASE1Eli Lilly and CompanyStarted 2022-05-11
LOXO-783FulvestrantImlunestrant
HER 2 Negative Breast Cancer

A Multicenter, Single-Arm, Phase II Exploratory Study of Eribulin in Combination With Anlotinib for HER2-Negative Recurrent/Metastatic Breast Cancer Previously Treated With Antibody-Drug Conjugates

RECRUITING
NCT07520760Phase PHASE2Sun Yat-sen UniversityStarted 2026-04-01
Eribulin in Combination with Anlotinib
Endometrial Cancer

Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel and a Substudy With an Insulin-Suppressing Diet in Patients With Advanced/Recurrent Endometrial Cancer

RECRUITING
NCT06463028Phase PHASE2Faeth TherapeuticsStarted 2024-12-12
SapanisertibSerabelisibPaclitaxel
Breast CancerER-positive Breast CancerHER2-negative Breast Cancer

Levels of Circulating Tumor DNA as a Predictive Marker for Early Switch in Treatment for Patients With Metastatic (Stage IV) Breast Cancer

ACTIVE NOT RECRUITING
NCT05826964Phase PHASE2University of MiamiStarted 2023-06-12
AI+CDK4/6iSERD+CDK4/6imTOR inhibitor + AI
Recurrent Endometrial CarcinomaRecurrent Endometrial Clear Cell AdenocarcinomaRecurrent Endometrial Endometrioid Adenocarcinoma

Testing Different Amounts of the Combination of Drugs M1774 and ZEN-3694 for the Treatment of Recurrent Ovarian and Endometrial Cancer

RECRUITING
NCT05950464Phase PHASE1National Cancer Institute (NCI)Started 2023-12-18
BET Bromodomain Inhibitor ZEN-3694Biopsy ProcedureBiospecimen Collection
Clinical Literature
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