PIK3CA

Chr 3

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

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

Phosphatidylinositol 3-kinase is composed of an 85 kDa regulatory subunit and a 110 kDa catalytic subunit. The protein encoded by this gene represents the catalytic subunit, which uses ATP to phosphorylate PtdIns, PtdIns4P and PtdIns(4,5)P2. This gene has been found to be oncogenic and has been implicated in cervical cancers. A pseudogene of this gene has been defined on chromosome 22. [provided by RefSeq, Apr 2016]

GeneReviewsOMIMResearchGenerating clinical summary…
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

2 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
112 unique Pathogenic / Likely Pathogenic· 662 VUS of 1609 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 OE?0.05 (0.020.12)
00.351.4
Missense OE?0.34 (0.300.38)
00.61.4
Synonymous OE?0.78
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

This gene — mechanism propensity

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

1609 submitted variants in ClinVar

Classification Summary

Pathogenic52
Likely Pathogenic60
VUS662
Likely Benign714
Benign50
Conflicting25
52
Pathogenic
60
Likely Pathogenic
662
VUS
714
Likely Benign
50
Benign
25
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
6
46
0
0
52
Likely Pathogenic
5
53
2
0
60
VUS
20
605
32
5
662
Likely Benign
2
6
147
559
714
Benign
0
2
48
0
50
Conflicting
25
Total337122295641,563

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

View in ClinVar →

27 pathogenic / likely-pathogenic (of 36) ClinVar copy-number / structural variants overlap PIK3CA — these span large chromosomal regions, not the gene specifically, and are excluded from the counts above. Explore in CNV tools →

Protein Context — Lollipop Plot

PIK3CA · protein map & ClinVar variants

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

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Head Neck CancerOral Cancer

Newer Therapeutic Targets in Head and Neck Cancers

RECRUITING
NCT05382585Banaras Hindu UniversityStarted 2017-04-01
Next generation Sequencing
Advanced LymphomaAdvanced Malignant Solid NeoplasmRefractory Lymphoma

Testing JNJ-42756493 (Erdafitinib) as Potentially Targeting Treatment in Cancers With FGFR Amplifications (MATCH-Subprotocol K1)

ACTIVE NOT RECRUITING
NCT06308822Phase PHASE2National Cancer Institute (NCI)Started 2018-08-20
Biopsy ProcedureBiospecimen CollectionComputed Tomography
Endometrial Cancer

A Study of Targeted Agents for Patients With Recurrent or Persistent Endometrial Cancer

RECRUITING
NCT04486352Phase PHASE1, PHASE2Alliance Foundation Trials, LLC.Started 2021-10-20
Atezolizumab - 28 Day CycleBevacizumabIpatasertib
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
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
Non Small Cell Lung CancerEGFR Gene MutationNon Small Cell Lung Cancer Stage IIIB

Osimertinib and Etoposide as First-Line Treatment in Osimertinib-Resistant Advanced EGFR-Mutant NSCLC

NOT YET RECRUITING
NCT06436144Phase PHASE2Daping Hospital and the Research Institute of Surgery of the Third Military Medical UniversityStarted 2024-06
Osimertinib
HER-2 Positive Breast CancerEstrogen Receptor Positive Breast Cancer

Randomized, Open Label, Clinical Study of the Targeted Therapy, Palbociclib, to Treat Metastatic Breast Cancer

ACTIVE NOT RECRUITING
NCT02947685Phase PHASE3Alliance Foundation Trials, LLC.Started 2017-06-21
palbociclibtrastuzumabpertuzumab
Lymphatic MalformationLymphatic AbnormalityLymphangioma

Institution of an Italian Registry and Biobank for Biological Sample Collection

RECRUITING
NCT06892964Phase NAFondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2024-06-27
Genetic profiling of all eligible patients
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
Triple Negative Breast CancerBreast Cancer

Serial Circulating Tumor DNA (ctDNA) Monitoring During Adjuvant Capecitabine in Early Triple-negative Breast Cancer

RECRUITING
NCT04768426Phase PHASE2Stanford UniversityStarted 2021-02-03
Capecitabine
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 Cancer - ER+, HER2-, PIK3CA Gene Mutation

PI3K Pathway Activation Markers in ER-Positive, HER2-Negative Breast Cancer: A Clinicopathologic Study

NOT YET RECRUITING
NCT07387861Assiut UniversityStarted 2026-03
Not applicable- observational study