Aspergillosis

disease
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Summary

Aspergillosis (MONDO:0005657) is a disease with 2 GWAS associations across 5 studies and 52 clinical trials. Top therapeutic interventions include voriconazole, caspofungin, and micafungin. A subtype of opportunistic mycosis — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

  • Prevalence: Unknown (Worldwide)
  • GWAS associations: 2
  • Phenotypes (HPO): 56
  • Clinical trials: 52

Clinical features

Signs & symptoms

Clinical features (HPO)

56 HPO clinical features (Orphanet curated; top 50 by frequency):

HPO IDTermFrequency
HP:0012735CoughVery frequent (80-99%)
HP:0020153Positive blood 1,3 beta glucan testVery frequent (80-99%)
HP:0001875Decreased total neutrophil countFrequent (30-79%)
HP:0001880EosinophiliaFrequent (30-79%)
HP:0001945FeverFrequent (30-79%)
HP:0002105HemoptysisFrequent (30-79%)
HP:0002113Pulmonary infiltratesFrequent (30-79%)
HP:0002721ImmunodeficiencyFrequent (30-79%)
HP:0006528Chronic lung diseaseFrequent (30-79%)
HP:0025179Ground-glass opacification on pulmonary HRCTFrequent (30-79%)
HP:0030878Abnormality on pulmonary function testingFrequent (30-79%)
HP:0032016Abnormal sputumFrequent (30-79%)
HP:0032177Parenchymal consolidationFrequent (30-79%)
HP:0100749Chest painFrequent (30-79%)
HP:0000077Abnormality of the kidneyOccasional (5-29%)
HP:0000246SinusitisOccasional (5-29%)
HP:0000491KeratitisOccasional (5-29%)
HP:0000505Visual impairmentOccasional (5-29%)
HP:0000620DacryocystitisOccasional (5-29%)
HP:0001626Abnormality of the cardiovascular systemOccasional (5-29%)
HP:0001742Nasal congestionOccasional (5-29%)
HP:0002031Abnormal esophagus morphologyOccasional (5-29%)
HP:0002090PneumoniaOccasional (5-29%)
HP:0002094DyspneaOccasional (5-29%)
HP:0002099AsthmaOccasional (5-29%)
HP:0002102PleuritisOccasional (5-29%)
HP:0002206Pulmonary fibrosisOccasional (5-29%)
HP:0002207Diffuse reticular or finely nodular infiltrationsOccasional (5-29%)
HP:0002315HeadacheOccasional (5-29%)
HP:0003212Increased circulating IgE levelOccasional (5-29%)
HP:0004377Hematological neoplasmOccasional (5-29%)
HP:0006510Chronic pulmonary obstructionOccasional (5-29%)
HP:0006516Hypersensitivity pneumonitisOccasional (5-29%)
HP:0011355Localized skin lesionOccasional (5-29%)
HP:0012115HepatitisOccasional (5-29%)
HP:0020103Invasive pulmonary aspergillosisOccasional (5-29%)
HP:0031417RhinorrheaOccasional (5-29%)
HP:0100326Immunologic hypersensitivityOccasional (5-29%)
HP:0200026Ocular painOccasional (5-29%)
HP:0000629Periorbital fullnessVery rare (<1-4%)
HP:0000772Abnormal rib morphologyVery rare (<1-4%)
HP:0000925Abnormality of the vertebral columnVery rare (<1-4%)
HP:0001250SeizureVery rare (<1-4%)
HP:0001287MeningitisVery rare (<1-4%)
HP:0001297StrokeVery rare (<1-4%)
HP:0002110BronchiectasisVery rare (<1-4%)
HP:0002170Intracranial hemorrhageVery rare (<1-4%)
HP:0002202Pleural effusionVery rare (<1-4%)
HP:0002383Infectious encephalitisVery rare (<1-4%)
HP:0002693Abnormality of the skull baseVery rare (<1-4%)

Identifiers

Disease identifiers

FieldValue
Canonical nameaspergillosis
Mondo IDMONDO:0005657
EFOEFO:0007157
MeSHD001228
Orphanet1163
DOIDDOID:13564
ICD-10-CMB44
ICD-111913468488
NCITC2886
SNOMED CT65553006
UMLSC0004030
MedGen13939
GARD0005856
MedDRA10003488
NORD814
Is cancer (heuristic)no

Data availability: 2 GWAS associations (5 studies).

Disease family

This is a subtype of opportunistic mycosis. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by etiologic mechanism › disease of primarily extrinsic mechanism › infectious diseasefungal infectious diseaseopportunistic mycosisaspergillosis

Related subtypes (11): phaeohyphomycosis, candidiasis, maple bark strippers’ lung, cryptococcosis, geotrichosis, microsporidiosis, fusariosis, pneumocystosis, Zygomycosis, opportunistic systemic mycosis, hyalohyphomycosis

Subtypes (4): pulmonary aspergilloma, neuroaspergillosis, allergic bronchopulmonary aspergillosis, aspergillus niger infection

Genetics & variants

GWAS landscape

2 GWAS associations across 5 studies. Top hits map to 3 distinct genes (as reported by GWAS).

Top associations by p-value

rsIDp-valueGeneRisk alleleOdds ratio
rs1139939608e-23CFTR?6.13
rs65026328e-08DRC3, ATPAF2?

Top studies (by case count)

StudyLead authorYearCasesControlsTitle
GCST90473087UK Biobank Whole-Genome Sequencing Consortium2025456457,984Whole-genome sequencing of 490,640 UK Biobank participants.
GCST90481465Verma A2024424450,659Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90651804Liu TY2025299223,436Diversity and longitudinal records: Genetic architecture of disease associations and polygenic risk in the Taiwanese Han population.
GCST90435562Zhou W2018163406,301Efficiently controlling for case-control imbalance and sample relatedness in large-scale genetic association studies.
GCST90837483Koyama S202500Genetics and context for precision health in Greater Boston.

Variant details and genetic-evidence tiers

Tier distribution (top 50 variants)

TierVariants
Tier 1: coding1
Tier 2: splice/UTR0
Tier 3: regulatory0
Tier 4: intronic/intergenic1

MAF distribution

BucketVariants
common (>=0.05)2
low_freq (0.01-0.05)0
rare (<0.01)0
unknown0

Functional consequences

ConsequenceCount
inframe_insertion1
intron_variant1

Top variants

rsIDChrPosAllelesMAFConsequenceGenep-valueTier
rs1139939607117559591ATCT>A,ATCTTCT0.05inframe_insertionCFTR8e-23Tier 1: coding
rs65026321718009743T>A,C0.05intron_variantDRC3, ATPAF28e-08Tier 4: intronic/intergenic

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

Drugs indicated for this disease

4 approved, 5 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

DrugDevelopment status
Amphotericin BApproved (phase 4)
ItraconazoleApproved (phase 4)
PosaconazoleApproved (phase 4)
VoriconazoleApproved (phase 4)
AnidulafunginPhase 3 (in late-stage trials)
FluconazolePhase 3 (in late-stage trials)
IsavuconazolePhase 3 (in late-stage trials)
OlorofimPhase 3 (in late-stage trials)
PyrrolePhase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Caspofungin, INTERFERON GAMMA-1B, Ibrexafungerp, Micafungin.

Clinical trials & evidence

Clinical trials

Clinical trials: 52.

Phase distribution (across all retrieved trials)

PhaseTrials
Not specified20
PHASE211
PHASE310
PHASE45
PHASE2/PHASE34
PHASE12

Top trials by phase / activity

NCTPhaseStatusTitle
NCT00423163PHASE4WITHDRAWNA Study to Evaluate the Effectiveness of Voriconazole + Micafungin Versus Voriconazole Alone for Invasive Aspergillosis
NCT00620074PHASE4TERMINATEDStudy to Test the Combination of Voriconazole and Anidulafungin in Patients Who Have, or Are Thought to Have, Invasive Aspergillosis and Who Are Unable to Take a Common Antifungal Therapy (Polyene)
NCT00647907PHASE4COMPLETEDA Study of the Efficacy and Safety of Voriconazole for the Treatment of Fungal Infections
NCT00940017PHASE4COMPLETEDA Study To Assess The Anidulafungin And Voriconazole Concentration In Lung Following Intravenous Administration In Healthy Subjects
NCT02646800PHASE4TERMINATEDPost Marketing Study to Evaluate the Safety and Efficacy of Micafungin Against Fungal Infections Caused by Candida Spp or Aspergillus Spp
NCT00001646PHASE3COMPLETEDVoriconazole vs. Amphotericin B in the Treatment of Invasive Aspergillosis
NCT00001810PHASE3COMPLETEDAn Open Label, Non-Comparative, Multicenter, Phase III Trial of the Efficacy, Safety and Toleration of Voriconazole in the Primary or Secondary Treatment of Invasive Fungal Infections
NCT00001937PHASE3COMPLETEDComparing the Effectiveness of Fluconazole and a New Medicine (FK463) in Preventing Fungal Infections in Bone Marrow Transplant Patients
NCT00037206PHASE2/PHASE3COMPLETEDA Safety & Effectiveness Study of Intravenous Anidulafungin With AmBisome® for Treatment of Invasive Aspergillosis (IA).
NCT00159822PHASE2/PHASE3COMPLETEDVoriconazole For Chronic Bronchopulmonary Aspergillosis
NCT00263315PHASE2/PHASE3COMPLETEDInhalation of Liposomal Amphotericin B to Prevent Invasive Aspergillosis
NCT00361517PHASE3COMPLETEDTo Determine Whether Galactomannan Test Will Help to Detect Fungal Infections Early and Hence Start Treatment Early
NCT00412893PHASE3COMPLETEDIsavuconazole (BAL8557) for Primary Treatment of Invasive Aspergillosis
NCT00531479PHASE3COMPLETEDAnidulafungin Plus Voriconazole Versus Voriconazole For The Treatment Of Invasive Aspergillosis
NCT00634049PHASE3COMPLETEDIsavuconazole in the Treatment of Renally Impaired Aspergillosis and Rare Fungi
NCT00784368PHASE3COMPLETEDA Pharmacokinetic Study of JK1211(Itraconazole [Itrizole]) Oral Solution in Participants With Deep Mycosis and Those With Febrile Neutropenia Suspected of Fungal Infection
NCT01188759PHASE3WITHDRAWNVoriconazole And Anidulafungin Combination For Invasive Aspergillosis In Pediatric Subjects
NCT02180165PHASE3COMPLETEDAssessing the Safety and Efficacy of MK-5592 (Posaconazole) in Japanese Participants With Fungal Infection (MK-5592-101)
NCT04966234PHASE2/PHASE3UNKNOWNA New Posaconazole Dosing Regimen for Paediatric Patients With Cystic Fibrosis and Aspergillus Infection
NCT05653193PHASE2RECRUITINGInterferon-gamma as Adjunctive Therapy in Chronic Pulmonary Aspergillosis: a Randomised Feasibility Study
NCT00005668PHASE2COMPLETEDA Multicenter, Prospective, Randomized, Double-Blind Clinical Trial Comparing Itraconazole Oral Solution in Cyclodextrin to Placebo in the Treatment of Aspergilloma
NCT00036166PHASE2COMPLETEDStudy of FK463 for the Treatment of Invasive Aspergillosis
NCT00047827PHASE2TERMINATEDTrial of Micafungin (FK463) in Combination With Liposomal Amphotericin B (AmBisome) for Aspergillosis
NCT00076869PHASE2COMPLETEDMK0991 in Combination With Standard Antifungal Agent(s) for the Treatment of Salvage Invasive Aspergillosis (0991-037)
NCT00082524PHASE2COMPLETEDDocumented Candida or Aspergillus Infections in Pediatric Patients (0991-043)
NCT01165320PHASE2COMPLETEDA Study of Caspofungin (MK-0991) in Japanese Children and Adolescents With Documented Candida or Aspergillus Infections (MK-0991-074)
NCT01207128PHASE2WITHDRAWNTrial of Combination Antifungal Therapy (Vori+Mica vs. Vori+Placebo) in Invasive Aspergillosis
NCT01887457PHASE2SUSPENDEDIndividualisation of Voriconazole Antifungal Therapy Antifungal Therapy
NCT03870841PHASE2TERMINATEDThe Effect of PC945 on Aspergillus Fumigatus Lung Infection in Patients With Cystic Fibrosis
NCT03905447PHASE2TERMINATEDThe Effect of Early Treatment of PC945 on Aspergillus Fumigatus Lung Infection in Lung Transplant Patients.
NCT00005912PHASE1COMPLETEDVoriconazole to Prevent Systemic Fungal Infections in Children
NCT02715570PHASE1COMPLETEDA Study to Investigate the Safety, Tolerability and Pharmacokinetics of Single and Repeat Doses of PC945
NCT03828773Not specifiedRECRUITINGPTX3-targeted Antifungal Prophylaxis
NCT05045391Not specifiedACTIVE_NOT_RECRUITINGPulmonary Aspergillosis in Tuberculosis Patients
NCT07080359Not specifiedNOT_YET_RECRUITINGIsavuconazole in Critically Ill Patients: Efficacy and Safety
NCT00388167Not specifiedCOMPLETEDAcetato de Caspofungin (Cancidas®) in the Treatment of Fungal Infection
NCT00460330Not specifiedUNKNOWNEvaluate Three Methods for Diagnosis of Invasive Fungal Infection in Chinese Patients After HSCT
NCT00473252Not specifiedCOMPLETEDSurveillance of Fungal Infections During Construction Activity
NCT00854607Not specifiedCOMPLETEDAn Observational Study of Fungal Biomarkers (MK-0000-089)
NCT01315925Not specifiedUNKNOWNPre-hospital Risk Factors for Invasive Fungal Infection

Drugs tested across these trials (top 30)

MoleculeMax phaseTrials referencing
VORICONAZOLE414
CASPOFUNGIN46
MICAFUNGIN46
ANIDULAFUNGIN45
POSACONAZOLE44
AMPHOTERICIN B42
ITRACONAZOLE42
DEOXYCHOLIC ACID41
FLUCONAZOLE41
ISAVUCONAZOLE33
OPELCONAZOLE32
SILVER31
CHEMBL376004905
CHEMBL475094102
CHEMBL49980802
CHEMBL526720501
DEOXYCHOLATE-11
HYOSCYAMINE-11