SMARCA4

Chr 19

SWI/SNF related BAF chromatin remodeling complex subunit ATPase 4

Also known as: BAF190, BAF190A, BRG1, CSS4, MRD16, OTSC12, RTPS2, SNF2

The protein functions as an ATPase and helicase component of the SWI/SNF chromatin remodeling complex, altering chromatin structure to enable transcriptional activation of normally repressed genes. Heterozygous loss-of-function mutations cause autosomal dominant Coffin-Siris syndrome 4 and rhabdoid tumor predisposition syndrome 2. The protein is intolerant to loss-of-function variants, with pathogenicity resulting from haploinsufficiency of the chromatin remodeling function.

GeneReviewsResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismLOEUF 0.06
Clinical SummarySMARCA4
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Gene-Disease Validity (ClinGen)
rhabdoid tumor predisposition syndrome 2 · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

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
32 unique Pathogenic / Likely Pathogenic· 192 VUS of 400 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 — SMARCA4
Authoritative clinical overview · Recommended first read
Open GeneReview ↗
Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.06LOEUF
pLI 1.000
Z-score 8.48
OE 0.01 (0.000.06)
Highly constrained

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

Missense Constraint
6.85Z-score
OE missense 0.40 (0.370.44)
423 obs / 1046.1 exp
Constrained

Extremely missense-constrained (top ~0.01%)

Observed / Expected Ratios
LoF OE0.01 (0.000.06)
00.351.4
Missense OE0.40 (0.370.44)
00.61.4
Synonymous OE1.12
01.21.6
LoF obs/exp: 1 / 85.7Missense obs/exp: 423 / 1046.1Syn Z: -2.00
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveSMARCA4-related Coffin SirisOTHERAD
definitiveSMARCA4-related rhabdoid tumor predisposition syndromeLOFAD
DN
0.2698th %ile
GOF
0.3391th %ile
LOF
0.84top 5%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function, gain-of-function and dominant-negative). Both the Badonyi & Marsh prediction and the broader genomic evidence point to loss-of-function as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOFprediction above median · 1 literature citation · 91% of P/LP variants are LoF · LOEUF 0.06
GOF1 literature citation
DN1 literature citation

Literature Evidence

DNMutations in SMARCB1, SMARCA4, and SMARCE1 are expected to exert dominant-negative or gain-of-function effects, whereas those in ARID1A are expected to exert loss-of-function effects.PMID:25168959
GOFWhereas germline heterozygous truncating mutations in SMARCA4 have been reported in individuals with rhabdoid tumor predisposition syndrome-2 (613235), all mutations in the CSS4 patients were nontruncating, implying that they exert gain-of-function or dominant-negative effects.PMID:22426308
LOFThis study suggests that SMARCA4 constitutional mutations associated with CSS are not necessarily non-truncating, and that haploinsufficiency may explain milder CSS phenotypes, as previously reported for haploinsufficient ARID1B.PMID:28608987

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

400 submitted variants in ClinVar

Classification Summary

Pathogenic16
Likely Pathogenic16
VUS192
Likely Benign121
Conflicting1
16
Pathogenic
16
Likely Pathogenic
192
VUS
121
Likely Benign
1
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
15
1
0
0
16
Likely Pathogenic
14
2
0
0
16
VUS
1
178
11
2
192
Likely Benign
1
2
60
58
121
Benign
0
0
0
0
0
Conflicting
1
Total311837160346

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

View in ClinVar →

Protein Context — Lollipop Plot

SMARCA4 · 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

Mantle Cell Lymphoma Refractory

Genetically Risk-Stratified Venetoclax, Ibrutinib, Rituximab (± Navitoclax) in Relapsed/Refractory Mantle Cell Lymphoma

ACTIVE NOT RECRUITING
NCT05864742Phase PHASE2Peter MacCallum Cancer Centre, AustraliaStarted 2023-09-07
IbrutinibVenetoclaxNavitoclax
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
Malignant Solid TumorsOther Neoplasms Solid TumorsPediatric Solid Tumor

Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors

RECRUITING
NCT03739827National Cancer Institute (NCI)Started 2019-01-28
SMARCA4-Deficient TumorLocally Advanced or Metastatic Non-Small Cell Lung Cancer

A Phase II Study of QL1706 and Platinum-Based Chemotherapy in Patients With SMARCA4-Deficient, Locally Advanced or Metastatic Non-Small Cell Lung Cancer.

NOT YET RECRUITING
NCT07200947Phase PHASE2Zhijie WangStarted 2025-12-01
QL1706, nab-paclitaxel, carboplatin
Solid Tumor MalignanciesClear Cell Endometrial CarcinomaOvarian Cancer

Efficacy and Safety of the Valemetostat in Patients With Selected Solid Tumors.

RECRUITING
NCT07303387Phase PHASE2Gustave Roussy, Cancer Campus, Grand ParisStarted 2026-03-05
Valemetostat Tosylate
Malignant Rhabdoid TumorRhabdoid Tumor of the KidneyEpithelioid Sarcoma

Study of Nivolumab and Ipilimumab in Children and Young Adults With INI1-Negative Cancers

ACTIVE NOT RECRUITING
NCT04416568Phase PHASE2Dana-Farber Cancer InstituteStarted 2020-08-14
NivolumabIpilimumab
Non Small Cell Lung Cancer

Efficacy and Safety Analysis of First-Line ABCP Therapy in Advanced SMARCA4-Mutated NSCLC

NOT YET RECRUITING
NCT07093762Fuzhou General HospitalStarted 2025-08-20
Atezolizumab+Bevacizumab+Carboplatin+Paclitaxel
Esophageal Squamous Cell CarcinomaGastric AdenocarcinomaGastric Squamous Cell Carcinoma

First-in-Human Study of PLX-61639 in Locally Advanced or Metastatic Solid Tumors

RECRUITING
NCT07284186Phase PHASE1Plexium, Inc.Started 2025-12-01
PLX-61639
Small Cell Lung Cancer

SMARCA4/2 Inhibitor for POU2F3-Positive SCLC

NOT YET RECRUITING
NCT07551635Phase PHASE2Dana-Farber Cancer InstituteStarted 2026-09-04
FHD-286
16P11.2 Deletion Syndrome16p11.2 Duplications1Q21.1 Deletion

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

RECRUITING
NCT01238250Simons SearchlightStarted 2010-10
Atypical Teratoid Rhabdoid TumorINI1 (SMARCB1)-Deficient Primary CNS Malignant TumorsSMARCA4-deficient Primary CNS Malignant Tumors

Tazemetostat+Nivo/Ipi in INI1-Neg/SMARCA4-Def Tumors

ACTIVE NOT RECRUITING
NCT05407441Phase PHASE1, PHASE2Susan Chi, MDStarted 2023-08-10
TazemetostatNivolumabIpilimumab
Advanced Malignant Solid NeoplasmAnn Arbor Stage III Non-Hodgkin LymphomaAnn Arbor Stage IV Non-Hodgkin Lymphoma

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

ACTIVE NOT RECRUITING
NCT03155620Phase PHASE2National Cancer Institute (NCI)Started 2017-07-31
Biopsy ProcedureBiospecimen CollectionBone Marrow Aspiration and Biopsy
Clinical Literature
Open Research Assistant →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
SMARCA4 9
Zhao R et al.·Zhongguo Fei Ai Za Zhi
2022
SMARCA4
Peng L et al.·Zhongguo Fei Ai Za Zhi
2024
Top 5 full-text resultsSearch PubTator3 ↗
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗