SMARCA4

Chr 19AD

SWI/SNF related BAF chromatin remodeling complex subunit ATPase 4

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

The protein encoded by this gene is a member of the SWI/SNF family of proteins and is similar to the brahma protein of Drosophila. Members of this family have helicase and ATPase activities and are thought to regulate transcription of certain genes by altering the chromatin structure around those genes. The encoded protein is part of the large ATP-dependent chromatin remodeling complex SNF/SWI, which is required for transcriptional activation of genes normally repressed by chromatin. In addition, this protein can bind BRCA1, as well as regulate the expression of the tumorigenic protein CD44. Mutations in this gene cause rhabdoid tumor predisposition syndrome type 2. Multiple transcript variants encoding different isoforms have been found for this gene. [provided by RefSeq, May 2012]

Primary Disease Associations & Inheritance

?Otosclerosis 12MIM #620792
AD
{Rhabdoid tumor predisposition syndrome 2}MIM #613325
AD
Coffin-Siris syndrome 4MIM #614609
AD
542
ClinVar variants
49
Pathogenic / LP
1.00
pLI score· haploinsufficient
12
Active trials
Clinical SummarySMARCA4
🧬
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.
📋
ClinVar Variants
49 Pathogenic / Likely Pathogenic· 301 VUS of 542 total submissions
💊
Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint?LOEUF (Loss-of-function Observed/Expected Upper bound Fraction) is the upper bound of the 90% CI for LoF OE — the preferred gnomAD v4 metric. Lower = more intolerant to LoF. LOEUF < 0.35 = highly constrained.
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?Missense Z-score: standard deviations fewer missense variants observed vs. expected. Z > 3.09 (p < 0.001) = gene does not tolerate missense variation. OE missense < 0.6 is also considered constrained.
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?Shaded band = 90% confidence interval. Vertical tick = point estimate. Grey threshold line = gnomAD constraint cutoff for that variant class.
LoF OE?Ratio of observed to expected LoF variants. Upper CI bound (LOEUF) ≤ 0.35 = strong LoF constraint signal.0.01 (0.000.06)
00.351.4
Missense OE?Ratio of observed to expected missense variants. OE ≤ 0.6 = fewer missense variants than expected by chance.0.40 (0.370.44)
00.61.4
Synonymous OE?Control metric — synonymous variants are largely neutral and expected near OE = 1.0. Significant deviation may indicate annotation issues.1.12
01.21.6
LoF obs/exp: 1 / 85.7Missense obs/exp: 423 / 1046.1Syn Z: -2.00

ClinVar Variant Classifications

542 submitted variants in ClinVar

Classification Summary

Pathogenic29
Likely Pathogenic20
VUS301
Likely Benign187
Conflicting5
29
Pathogenic
20
Likely Pathogenic
301
VUS
187
Likely Benign
5
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
13
1
15
0
29
Likely Pathogenic
15
3
2
0
20
VUS
2
268
29
2
301
Likely Benign
1
3
59
124
187
Benign
0
0
0
0
0
Conflicting
5
Total31275105126542

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.

Gene2Phenotype Curations

SMARCA4-related Coffin Siris

definitive
ADUndeterminedAltered Gene Product Structure
Dev. DisordersSkeletal
G2P ↗
missense variantinframe deletioninframe insertion

SMARCA4-related rhabdoid tumor predisposition syndrome

definitive
ADLoss Of FunctionAbsent Gene Product, Altered Gene Product Structure
Dev. DisordersCancer
G2P ↗

Gene2Phenotype curations · DECIPHER consortium patient cohort (public variants) · deciphergenomics.org

OMIM — Genotype-Phenotype Relationships

1 OMIM entry

?Otosclerosis 12

MIM #620792

Molecular basis of disorder known

Autosomal dominant

{Rhabdoid tumor predisposition syndrome 2}

MIM #613325

Molecular basis of disorder known

Autosomal dominant

Coffin-Siris syndrome 4

MIM #614609

Molecular basis of disorder known

Autosomal dominant
📖
GeneReview available — SMARCA4
Authoritative clinical overview · NCBI Bookshelf · Recommended first read
Open GeneReview ↗
Clinical Literature
Landmark / reviewRecent case evidence

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

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
SMARCA4-Deficient TumorLocally Advanced or Metastatic Lung Cancer

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

NOT YET RECRUITING
NCT07307443Phase PHASE2Zhijie WangStarted 2026-02-01
Anlotinib, nab-paclitaxel, carboplatin
Metastatic Solid TumorAdvanced Solid TumorNon-small Cell Lung Cancer

A Study of LY4050784 in Participants With Advanced or Metastatic Solid Tumors

RECRUITING
NCT06561685Phase PHASE1Eli Lilly and CompanyStarted 2024-09-19
LY4050784PembrolizumabCisplatin
Solid TumorARID1A Gene Mutation

Phase II Study of Tazemetostat in Solid Tumors Harboring an ARID1A Mutation

RECRUITING
NCT05023655Phase PHASE2Prisma Health-UpstateStarted 2022-01-06
Tazemetostat
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
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
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
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
Atypical Teratoid/Rhabdoid Tumors (AT/RTs)Central Nervous System (CNS) Tumors

Feasibility of Using Bortezomib With or Without Chemotherapy in Patients With Atypical Teratoid/Rhabdoid Tumors

ACTIVE NOT RECRUITING
NCT06853080Phase PHASE2Taipei Medical UniversityStarted 2020-12-02
Bortezomib
Atypical Teratoid Rhabdoid TumorINI1 (SMARCB1)-Deficient Primary CNS Malignant TumorsSMARCA4-deficient Primary CNS Malignant Tumors

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

RECRUITING
NCT05407441Phase PHASE1, PHASE2Susan Chi, MDStarted 2023-08-10
TazemetostatNivolumabIpilimumab
Solid Tumor MalignanciesClear Cell Endometrial CarcinomaOvarian Cancer

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

NOT YET RECRUITING
NCT07303387Phase PHASE2Gustave Roussy, Cancer Campus, Grand ParisStarted 2026-02-28
Valemetostat Tosylate
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