FANCA

Chr 16AR

FA complementation group A

Also known as: FA, FA-H, FA1, FAA, FACA, FAH, FANCH

The Fanconi anemia complementation group (FANC) currently includes FANCA, FANCB, FANCC, FANCD1 (also called BRCA2), FANCD2, FANCE, FANCF, FANCG, FANCI, FANCJ (also called BRIP1), FANCL, FANCM and FANCN (also called PALB2). The previously defined group FANCH is the same as FANCA. Fanconi anemia is a genetically heterogeneous recessive disorder characterized by cytogenetic instability, hypersensitivity to DNA crosslinking agents, increased chromosomal breakage, and defective DNA repair. The members of the Fanconi anemia complementation group do not share sequence similarity; they are related by their assembly into a common nuclear protein complex. This gene encodes the protein for complementation group A. Alternative splicing results in multiple transcript variants encoding different isoforms. Mutations in this gene are the most common cause of Fanconi anemia. [provided by RefSeq, Jul 2008]

GeneReviewsOMIMResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

Fanconi anemia, complementation group AMIM #227650
AR
Fanconi anemia, complementation group AMIM #227650
AR

Clinical highlights

Gene-disease validity (ClinGen)
Fanconi anemia complementation group A · ARDefinitivesufficient evidence for diagnostic panels
Interpreting a novel variant
Loss of function is the curated mechanism (Gene2Phenotype), though the gene is not strongly LoF-constrained in the population — weigh truncating variants against that tolerance.Curated gene-level mechanism — a prior for triage, not a per-variant call.
12
Active trials
89
Pubs (1 yr)
P/LP submissions
P/LP missense
1.37
LOEUF
LOF
Mechanism· G2P
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GeneReview available — FANCA
Authoritative clinical overview · Recommended first read
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint?
1.37LOEUF
pLI 0.000
Z-score -1.29
OE 1.15 (0.981.37)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint?
-5.41Z-score
OE missense 1.55 (1.471.62)
1202 obs / 777.3 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios?
LoF OE?1.15 (0.981.37)
00.351.4
Missense OE?1.55 (1.471.62)
00.61.4
Synonymous OE?1.57
01.21.6
LoF obs/exp: 96 / 83.3Missense obs/exp: 1202 / 777.3Syn Z: -8.03

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

FANCA · 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

Bone Marrow Failure SyndromesErythrocyte DisorderLeukocyte Disorder

Investigation of the Genetics of Hematologic Diseases

RECRUITING
NCT02720679St. Jude Children's Research HospitalStarted 2016-06-17
Fanconi Anemia Complementation Group A

Gene Therapy for Fanconi Anemia, Complementation Group A

ACTIVE NOT RECRUITING
NCT04248439Phase PHASE2Rocket Pharmaceuticals Inc.Started 2020-07-15
RP-L102
Metastatic Pancreatic Ductal AdenocarcinomaHomologous Recombination Deficiency (HRD)

A Study of Pembrolizumab and Olaparib for People With Metastatic Pancreatic Ductal Adenocarcinoma and Homologous Recombination Deficiency or Exceptional Treatment Response to Platinum-Based Therapy

ACTIVE NOT RECRUITING
NCT04666740Phase PHASE2Memorial Sloan Kettering Cancer CenterStarted 2020-12-18
PembrolizumabOlaparib
Acute LeukemiaAdenomatous PolyposisAdrenocortical Carcinoma

Familial Investigations of Childhood Cancer Predisposition

RECRUITING
NCT03050268St. Jude Children's Research HospitalStarted 2017-04-06
Advanced or Metastatic Solid TumorsBreast CancerOvarian Cancer

A Study of PARG Inhibitor IDE161 in Participants With Advanced Solid Tumors

ACTIVE NOT RECRUITING
NCT05787587Phase PHASE1IDEAYA BiosciencesStarted 2023-04-18
IDE-161Pembrolizumab
Metastatic Breast CancerInvasive Breast CancerSomatic Mutation Breast Cancer (BRCA1)

Olaparib In Metastatic Breast Cancer

ACTIVE NOT RECRUITING
NCT03344965Phase PHASE2Beth Israel Deaconess Medical CenterStarted 2018-04-01
Olaparib
Bone Marrow Failure DisordersVEXAS SyndromeHemoglobinurea, Paroxysmal

Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study

ENROLLING BY INVITATION
NCT07102849National Heart, Lung, and Blood Institute (NHLBI)Started 2025-09-09
Fanconi Anemia Complementation Group AFanconi Anemia

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

ACTIVE NOT RECRUITING
NCT04437771Rocket Pharmaceuticals Inc.Started 2020-06-01
Safety and efficacy assessments
Acute Lymphoblastic Leukemia in RemissionAcute Myeloid Leukemia in RemissionMyelodysplastic Syndromes

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

ACTIVE NOT RECRUITING
NCT05236764Phase NABaylor College of MedicineStarted 2023-12-06
CliniMACS
Advanced Solid TumorsEwing SarcomaHepatocellular Carcinoma (HCC)

A Phase 1/1B Study of ST-01156, a Small Molecule RBM39 Degrader, in Patients With Advanced Solid Malignancies

RECRUITING
NCT07197554Phase PHASE1SEED Therapeutics, Inc.Started 2025-12-01
ST-01156
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
Lymphoma, Non-HodgkinMultiple MyelomaAdvanced Solid Tumors

TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer

RECRUITING
NCT02693535Phase PHASE2American Society of Clinical OncologyStarted 2016-03-14
SunitinibTemsirolimusTrastuzumab and Pertuzumab