FANCA

Chr 16AR

FA complementation group A

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

The FANCA protein functions in DNA repair, specifically in interstrand DNA cross-link repair and maintenance of chromosome stability as part of the Fanconi anemia nuclear protein complex. Mutations cause Fanconi anemia complementation group A, an autosomal recessive disorder characterized by cytogenetic instability, hypersensitivity to DNA crosslinking agents, increased chromosomal breakage, and defective DNA repair. FANCA mutations are the most common cause of Fanconi anemia among all complementation groups.

OMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismARLOEUF 1.372 OMIM phenotypes
Clinical SummaryFANCA
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Gene-Disease Validity (ClinGen)
Fanconi anemia complementation group A · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

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 OE1.15 (0.981.37)
00.351.4
Missense OE1.55 (1.471.62)
00.61.4
Synonymous OE1.57
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.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

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
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
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
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
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
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 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
Acute LeukemiaAdenomatous PolyposisAdrenocortical Carcinoma

Familial Investigations of Childhood Cancer Predisposition

RECRUITING
NCT03050268St. Jude Children's Research HospitalStarted 2017-04-06
Fanconi Anemia Complementation Group A

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

ACTIVE NOT RECRUITING
NCT04069533Phase PHASE2Rocket Pharmaceuticals Inc.Started 2019-11-28
RP-L102
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
Prostate Cancer Metastatic Castration-ResistantAbnormal DNA RepairMetastatic Prostate Carcinoma

Abiraterone/Prednisone, Olaparib, or Abiraterone/Prednisone + Olaparib in Patients With Metastatic Castration-Resistant Prostate Cancer With DNA Repair Defects

ACTIVE NOT RECRUITING
NCT03012321Phase PHASE2Northwestern UniversityStarted 2017-01-12
OlaparibAbiraterone AcetatePrednisone
Clinical Literature
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