Classic familial adenomatous polyposis

Summary

Classic familial adenomatous polyposis (MONDO:0021055) is a disease with 2 cohort genes and 89 clinical trials. Top therapeutic interventions include sulindac, eflornithine, and guselkumab.

At a glance

• Prevalence: 1-9 / 100 000 (Europe) [Orphanet-validated]
• Cohort genes: 2
• ClinVar variants: 213
• Phenotypes (HPO): 45
• Clinical trials: 89

Clinical features

Epidemiology

Prevalence records

12 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Point prevalence	1-9 / 100 000	6	Europe	Validated
Annual incidence	1-5 / 10 000	19	Denmark	Validated
Annual incidence	1-9 / 100 000	8.6	Sweden	Validated
Annual incidence	1-5 / 10 000	15.8	Finland	Validated
Annual incidence	1-9 / 1 000 000	0.24	Australia	Validated
Point prevalence	1-9 / 100 000	4.65	Denmark	Validated
Point prevalence	1-9 / 100 000	3.2	Sweden	Validated
Point prevalence	1-9 / 100 000	2.63	Finland	Validated
Point prevalence	1-9 / 100 000	5.3	United Kingdom	Validated
Point prevalence	1-9 / 100 000	2.8	Australia	Validated
Prevalence at birth	1-5 / 10 000	11.6	United Kingdom	Validated
Point prevalence	1-9 / 100 000		Worldwide	Not yet validated

Signs & symptoms

Clinical features (HPO)

45 HPO clinical features (Orphanet curated; top 45 by frequency):

HPO ID	Term	Frequency
HP:0003003	Colon cancer	Very frequent (80-99%)
HP:0005227	Adenomatous colonic polyposis	Very frequent (80-99%)
HP:0007378	Neoplasm of the gastrointestinal tract	Very frequent (80-99%)
HP:0007649	Congenital hypertrophy of retinal pigment epithelium	Very frequent (80-99%)
HP:0100245	Desmoid tumors	Very frequent (80-99%)
HP:0200063	Colorectal polyposis	Very frequent (80-99%)
HP:0000164	Abnormality of the dentition	Frequent (30-79%)
HP:0002014	Diarrhea	Frequent (30-79%)
HP:0002019	Constipation	Frequent (30-79%)
HP:0004394	Multiple gastric polyps	Frequent (30-79%)
HP:0004783	Duodenal polyposis	Frequent (30-79%)
HP:0025388	Thyroid nodule	Frequent (30-79%)
HP:0100246	Osteoma	Frequent (30-79%)
HP:0000706	Unerupted tooth	Occasional (5-29%)
HP:0000820	Abnormality of the thyroid gland	Occasional (5-29%)
HP:0002895	Papillary thyroid carcinoma	Occasional (5-29%)
HP:0006771	Duodenal adenocarcinoma	Occasional (5-29%)
HP:0008256	Adrenocortical adenoma	Occasional (5-29%)
HP:0010615	Angiofibromas	Occasional (5-29%)
HP:0011068	Odontoma	Occasional (5-29%)
HP:0011069	Supernumerary tooth	Occasional (5-29%)
HP:0100631	Neoplasm of the adrenal gland	Occasional (5-29%)
HP:0100717	Abnormality of the cementum	Occasional (5-29%)
HP:0030434	Pilomatrixoma	Very rare (<1-4%)
HP:0030692	Brain neoplasm	Very rare (<1-4%)
HP:0031459	Soft tissue neoplasm	Very rare (<1-4%)
HP:0040274	Adenocarcinoma of the small intestine	Very rare (<1-4%)
HP:0100575	Neoplasm of the gallbladder	Very rare (<1-4%)
HP:0100646	Thyroiditis	Very rare (<1-4%)
HP:0200040	Epidermoid cyst	Very rare (<1-4%)
HP:0000821	Hypothyroidism	Very rare (<1-4%)
HP:0000853	Goiter	Very rare (<1-4%)
HP:0001733	Pancreatitis	Very rare (<1-4%)
HP:0002884	Hepatoblastoma	Very rare (<1-4%)
HP:0002885	Medulloblastoma	Very rare (<1-4%)
HP:0002888	Ependymoma	Very rare (<1-4%)
HP:0002893	Pituitary adenoma	Very rare (<1-4%)
HP:0005230	Biliary tract obstruction	Very rare (<1-4%)
HP:0006725	Pancreatic adenocarcinoma	Very rare (<1-4%)
HP:0009592	Astrocytoma	Very rare (<1-4%)
HP:0010614	Fibroma	Very rare (<1-4%)
HP:0011355	Localized skin lesion	Very rare (<1-4%)
HP:0012032	Lipoma	Very rare (<1-4%)
HP:0012126	Stomach cancer	Very rare (<1-4%)
HP:0030153	Cholangiocarcinoma	Very rare (<1-4%)

Identifiers

Disease identifiers

Field	Value
Canonical name	classic familial adenomatous polyposis
Mondo ID	MONDO:0021055
OMIM	175100
Orphanet	733
DOID	DOID:0050424
NCIT	C3339
SNOMED CT	72900001
UMLS	C0032580
MedGen	46010
GARD	0006408
MedDRA	10056981
NORD	1121
Is cancer (heuristic)	no

Also known as: adenomatous polyposis coli · classic familial adenomatous polyposis · classic FAP · colorectal adenomatous polyposis · Familial Adenomatous Polyposis · familial adenomatous polyposis · familial adenomatous polyposis coli · familial adenomatous polyposis of the colon · familial adenomatous polyposis syndrome · familial multiple polyposis · familial polyposis · familial polyposis coli · FAP · FPC · hereditary adenomatous polyposis coli · hereditary polyposis coli · polyposis coli

Data availability: 213 ClinVar variants · 1 ClinGen variant curation · 95 cell lines.

Disease family

An umbrella term covering 3 Mondo subtypes.

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary disease › hereditary neoplastic syndrome › intestinal polyposis syndrome › classic or attenuated familial adenomatous polyposis › classic familial adenomatous polyposis

Related subtypes (7): familial adenomatous polyposis 2, familial adenomatous polyposis 3, attenuated familial adenomatous polyposis, AXIN2-related attenuated familial adenomatous polyposis, Polymerase proofreading-related adenomatous polyposis, familial adenomatous polyposis 1, familial adenomatous polyposis 4

Subtypes (3): familial adenomatous polyposis due to 5q22.2 microdeletion, Gardner syndrome, Turcot syndrome with polyposis

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

213 retrieved; paginated sample, class counts are floors:

122 pathogenic, 35 pathogenic/likely pathogenic, 20 likely pathogenic, 16 conflicting classifications of pathogenicity, 10 benign/likely benign, 5 uncertain significance, 4 benign, 1 conflicting classifications of pathogenicity; association; risk factor

ClinVar	Variant (HGVS)	Gene	Classification	Review
1070046	NM_000038.6(APC):c.1958+2T>G	APC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1073668	NM_000038.6(APC):c.1500del (p.Arg499_Tyr500insTer)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1192196	NM_000038.6(APC):c.3581C>A (p.Ser1194Ter)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
127275	NM_000038.6(APC):c.1213C>T (p.Arg405Ter)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
127281	NM_000038.6(APC):c.2413C>T (p.Arg805Ter)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
127299	NM_000038.6(APC):c.4875del (p.Gln1625fs)	APC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
127312	NM_000038.6(APC):c.646C>T (p.Arg216Ter)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1330021	NM_000038.6(APC):c.1775T>G (p.Leu592Ter)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1371144	NM_000038.6(APC):c.5214_5215del (p.His1738fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
140863	NM_000038.6(APC):c.5936_5939del (p.Asn1979fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
140952	NM_000038.6(APC):c.637C>T (p.Arg213Ter)	APC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
141368	NM_000038.6(APC):c.147_150del (p.Lys49fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
141515	NM_000038.6(APC):c.220+2T>A	APC	Pathogenic	reviewed by expert panel
142199	NM_000038.6(APC):c.2004del (p.His668_Leu669insTer)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
142574	NM_000038.6(APC):c.426_427del (p.Leu143fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1451499	NM_000038.6(APC):c.2377C>T (p.Gln793Ter)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1457212	NM_000038.6(APC):c.2053_2054del (p.Trp685fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
156482	NM_000038.6(APC):c.1744-2A>G	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1697514	NM_000038.6(APC):c.289G>A (p.Gly97Arg)	APC	Pathogenic	criteria provided, single submitter
1704635	NC_000005.9:g.(112103088_112111325)_(112111435_112116486)del	APC	Pathogenic	criteria provided, single submitter
1723265	NM_000038.6(APC):c.3258_3259del (p.Leu1087fs)	APC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1730011	NM_000038.6(APC):c.3304del (p.Tyr1102fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1735824	NM_000038.6(APC):c.3883_3886del (p.Glu1295fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
1736223	NM_000038.6(APC):c.3925_3926del (p.Glu1309fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
177906	NM_000038.4(APC):c.(?1)(8477_?)del	APC	Pathogenic	criteria provided, single submitter
1779228	NM_000038.6(APC):c.1743+1G>C	APC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1804741	NM_000038.6(APC):c.778C>T (p.Gln260Ter)	APC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
181831	NM_000038.6(APC):c.288T>A (p.Tyr96Ter)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
181835	NM_000038.6(APC):c.5490_5493del (p.Asn1830fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts
183078	NM_000038.6(APC):c.3149del (p.Ala1050fs)	APC	Pathogenic	criteria provided, multiple submitters, no conflicts

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 7 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
APC	Orphanet:220460	Attenuated familial adenomatous polyposis
APC	Orphanet:261584	5q22 microdeletion syndrome
APC	Orphanet:314022	Gastric adenocarcinoma and proximal polyposis of the stomach
APC	Orphanet:3258	Cenani-Lenz syndrome
APC	Orphanet:873	Desmoid tumor
MUTYH	Orphanet:247798	MUTYH-related polyposis
MUTYH	Orphanet:440437	Familial colorectal cancer Type X

Cohort genes → proteins

2 cohort genes, 2 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	2

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
APC	HGNC:583	ENSG00000134982	P25054	Adenomatous polyposis coli protein	clinvar
MUTYH	HGNC:7527	ENSG00000132781	Q9UIF7	Adenine DNA glycosylase	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
APC	Adenomatous polyposis coli protein	Tumor suppressor.
MUTYH	Adenine DNA glycosylase	Involved in oxidative DNA damage repair.

Protein-family classification

Druggable: 0 · Difficult: 0 · Unknown: 2 · Druggable fraction: 0.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Other/Unknown	2	1.8×	0.312

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
APC	Other/Unknown	no		Armadillo, APC_rpt, SAMP
MUTYH	Other/Unknown	no		NUDIX_hydrolase_dom, HhH_motif, HhH-GPD_domain

Expression context

Cohort genes with no expression data: 0.

2 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	2
unknown	0

Top tissues across cohort

Tissue	Cohort genes
medial globus pallidus	1
substantia nigra pars compacta	1
substantia nigra pars reticulata	1
cerebellar cortex	1
cerebellar hemisphere	1
right hemisphere of cerebellum	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
APC	297	ubiquitous	marker	substantia nigra pars compacta, substantia nigra pars reticulata, medial globus pallidus
MUTYH	134	ubiquitous	marker	cerebellar hemisphere, cerebellar cortex, right hemisphere of cerebellum

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
APC	2,903
MUTYH	1,815

Structural data

PDB: 2 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
APC	P25054	31
MUTYH	Q9UIF7	3

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 36. Enrichment computed across 2 evidence-associated genes (2 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
APC truncation mutants are not K63 polyubiquitinated	1	5710.0×	0.002	APC
Defective MUTYH substrate binding	1	5710.0×	0.002	MUTYH
Defective MUTYH substrate processing	1	5710.0×	0.002	MUTYH
Displacement of DNA glycosylase by APEX1	1	519.1×	0.006	MUTYH
Signaling by AXIN mutants	1	519.1×	0.006	APC
Signaling by CTNNB1 phospho-site mutants	1	519.1×	0.006	APC
Signaling by APC mutants	1	519.1×	0.006	APC
Signaling by AMER1 mutants	1	519.1×	0.006	APC
APC truncation mutants have impaired AXIN binding	1	407.9×	0.006	APC
AXIN missense mutants destabilize the destruction complex	1	407.9×	0.006	APC
Truncations of AMER1 destabilize the destruction complex	1	407.9×	0.006	APC
Signaling by GSK3beta mutants	1	380.7×	0.006	APC
CTNNB1 S33 mutants aren’t phosphorylated	1	380.7×	0.006	APC
CTNNB1 S37 mutants aren’t phosphorylated	1	380.7×	0.006	APC
CTNNB1 S45 mutants aren’t phosphorylated	1	380.7×	0.006	APC
CTNNB1 T41 mutants aren’t phosphorylated	1	380.7×	0.006	APC
Beta-catenin phosphorylation cascade	1	335.9×	0.006	APC
Signaling by WNT in cancer	1	300.5×	0.007	APC
Apoptotic cleavage of cellular proteins	1	237.9×	0.008	APC
Apoptotic execution phase	1	237.9×	0.008	APC
Disassembly of the destruction complex and recruitment of AXIN to the membrane	1	178.4×	0.010	APC
Ovarian tumor domain proteases	1	139.3×	0.012	APC
Deactivation of the beta-catenin transactivating complex	1	116.5×	0.013	APC
Recognition and association of DNA glycosylase with site containing an affected purine	1	102.0×	0.014	MUTYH
Cleavage of the damaged purine	1	102.0×	0.014	MUTYH
Degradation of beta-catenin by the destruction complex	1	86.5×	0.016	APC
Apoptosis	1	84.0×	0.016	APC
Programmed Cell Death	1	73.2×	0.018	APC
Deubiquitination	1	62.1×	0.020	APC
TCF dependent signaling in response to WNT	1	58.9×	0.020	APC

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
depurination	1	2106.5×	0.006	MUTYH
regulation of microtubule-based movement	1	1404.3×	0.006	APC
negative regulation of cell cycle G1/S phase transition	1	1203.7×	0.006	APC
positive regulation of protein localization to centrosome	1	1203.7×	0.006	APC
negative regulation of cyclin-dependent protein serine/threonine kinase activity	1	1053.2×	0.006	APC
regulation of microtubule-based process	1	936.2×	0.006	APC
regulation of attachment of spindle microtubules to kinetochore	1	842.6×	0.006	APC
heart valve development	1	766.0×	0.006	APC
positive regulation of pseudopodium assembly	1	648.1×	0.006	APC
negative regulation of necroptotic process	1	495.6×	0.007	MUTYH
endocardial cushion morphogenesis	1	421.3×	0.008	APC
mismatch repair	1	324.1×	0.009	MUTYH
mitotic spindle assembly checkpoint signaling	1	280.9×	0.009	APC
cell fate specification	1	263.3×	0.009	APC
negative regulation of microtubule depolymerization	1	247.8×	0.009	APC
base-excision repair	1	234.1×	0.009	MUTYH
pattern specification process	1	234.1×	0.009	APC
negative regulation of G1/S transition of mitotic cell cycle	1	179.3×	0.011	APC
bicellular tight junction assembly	1	165.2×	0.011	APC
mitotic cytokinesis	1	129.6×	0.013	APC
insulin receptor signaling pathway	1	110.9×	0.015	APC
positive regulation of protein catabolic process	1	101.5×	0.016	APC
positive regulation of cold-induced thermogenesis	1	81.8×	0.019	APC
negative regulation of canonical Wnt signaling pathway	1	58.9×	0.024	APC
protein-containing complex assembly	1	56.9×	0.024	APC
Wnt signaling pathway	1	49.9×	0.027	APC
DNA repair	1	31.9×	0.039	MUTYH
positive regulation of cell migration	1	30.9×	0.039	APC
cell migration	1	30.8×	0.039	APC
positive regulation of apoptotic process	1	28.4×	0.041	APC

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 2

Druggability breadth: 2 of 2 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
APC	0	0
MUTYH	0	0

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
APC	24	Binding:24
MUTYH	1	Functional:1

Pharmacogenomics

Cohort genes with a PharmGKB record: 2; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	2	APC, MUTYH

Undrugged target profiles

2 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
APC	24	—
MUTYH	1	—

Clinical trials & evidence

Clinical trials

Clinical trials: 89.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	48
PHASE2	18
PHASE3	7
PHASE1	6
PHASE2/PHASE3	4
PHASE4	2
PHASE1/PHASE2	2
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00140894	PHASE4	TERMINATED	A Study of Rofecoxib in Familial Adenomatous Polyposis (FAP) (0966-205)(TERMINATED)
NCT04454151	PHASE4	UNKNOWN	Azithromycin Treatment for Readthrough of APC Gene Stop Codon Mutations in Familial Adenomatous Polyposis
NCT06545526	PHASE3	RECRUITING	Chemopreventive Effect of Combination of Celecoxib and Metformin in Patients With Familial Adenomatous Polyposis
NCT06782412	PHASE2/PHASE3	RECRUITING	Multicenter Validation Trial of [18F]AlF-FAPI-74 for PET Imaging of Cancer-associated Fibroblasts Through Fibroblast Activation Protein Inhibitors (FAPI) in Different Tumor Types
NCT06950385	PHASE3	RECRUITING	Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis
NCT00510692	PHASE2/PHASE3	COMPLETED	Chemoprevention Trial in Familial Adenomatous Polyposis (FAP) Coli Using EPA
NCT00585312	PHASE3	TERMINATED	Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP)
NCT00808743	PHASE2/PHASE3	COMPLETED	Prevention of Progression of Duodenal Adenomas in Patients With Familial Adenomatous Polyposis
NCT01245816	PHASE3	WITHDRAWN	A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
NCT01483144	PHASE3	COMPLETED	Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)
NCT01737398	PHASE2/PHASE3	COMPLETED	Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
NCT02175004	PHASE3	COMPLETED	Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
NCT03806426	PHASE3	UNKNOWN	Effect of EPA-FFA on Polypectomy in Familial Adenomatous Polyposis
NCT04230499	PHASE2	ACTIVE_NOT_RECRUITING	Trial of ERapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Surveillance
NCT05552755	PHASE1/PHASE2	RECRUITING	Evaluate REC-4881 in Participants With Familial Adenomatous Polyposis (FAP)
NCT05919264	PHASE1/PHASE2	RECRUITING	FOG-001 in Locally Advanced or Metastatic Solid Tumors
NCT06189820	PHASE2	RECRUITING	Role of the Fibroblast Activation Protein (FAP) as Biomarker of Fibrotic Lung Diseases
NCT06308445	PHASE2	NOT_YET_RECRUITING	Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis
NCT06557733	PHASE2	RECRUITING	An Investigational Drug (TPST-1495) in Patients With Familial Adenomatous Polyposis
NCT00033371	PHASE2	COMPLETED	Celecoxib With or Without Eflornithine in Preventing Colorectal Cancer in Patients With Familial Adenomatous Polyposis
NCT00248053	PHASE2	WITHDRAWN	Use of Curcumin in the Lower Gastrointestinal Tract in Familial Adenomatous Polyposis Patients
NCT00319007	PHASE2	UNKNOWN	Influence of Sulindac and Probiotics on the Development of Pouch Adenomas in Patients With Familial Adenomatous Polyposis
NCT00503035	PHASE2	COMPLETED	Molecular Targeting of 15-Lipoxygenase-1 (15-LOX-1) for Apoptosis Induction in Human Colorectal Cancers
NCT00641147	PHASE2	COMPLETED	Curcumin in Treating Patients With Familial Adenomatous Polyposis
NCT01187901	PHASE2	COMPLETED	A Clinical Trial of COX and EGFR Inhibition in Familial Polyposis Patients
NCT01725490	PHASE2	COMPLETED	The Chemopreventive Effect of Metformin in Patients With Familial Adenomatous Polyposis: Double Blinded Randomized Controlled Study
NCT02961374	PHASE2	COMPLETED	Erlotinib Hydrochloride in Reducing Duodenal Polyp Burden in Patients With Familial Adenomatous Polyposis at Risk of Developing Colon Cancer
NCT03061591	PHASE2	UNKNOWN	Turmeric Supplementation on Polyp Number and Size in Patients With Familial Adenomatous Polyposis.
NCT03095703	PHASE2	COMPLETED	Sirolimus and Familial Adenomatous Polyposis (FAP)
NCT04296851	PHASE2	UNKNOWN	Niclosamide for Familial Adenomatous Polyposis
NCT05223036	PHASE2	TERMINATED	Testing Obeticholic Acid for Familial Adenomatous Polyposis
NCT05262855	PHASE2	COMPLETED	Study of [68Ga]FAPI-46 PET in Patients With Pancreatic Ductal Carcinoma
NCT05402891	PHASE2	COMPLETED	The CHAMP-study: The CHemopreventive Effect of Lithium in Familial AdenoMatous Polyposis
NCT05630794	PHASE1	RECRUITING	Testing for Safety and Colorectal Cancer Preventive Effects of ONC201
NCT06640413	PHASE1	RECRUITING	A Study to Evaluate the Safety and Preliminary Signs of Efficacy of [177Lu]Lu-OncoFAP-23 Alone or in Combination With L19-IL2 as a Treatment of Metastatic FAP-positive Solid Tumors
NCT00770991	PHASE1	COMPLETED	Lyophilized Black Raspberries in Adults With Familial Adenomatous Polyposis (FAP)
NCT02113202	PHASE1	COMPLETED	Molecular Fluorescence Endoscopy in Patients With Familial Adenomatous Polyposis, Using Bevacizumab-IRDye800CW
NCT03649971	PHASE1	COMPLETED	A Study of Guselkumab in Participants With Familial Adenomatous Polyposis
NCT05014360	PHASE1	COMPLETED	A Study of JNJ-64251330 in Participants With Familial Adenomatous Polyposis
NCT06387381	EARLY_PHASE1	RECRUITING	68Ga-PSFA PET Imaging in Patients With PSMA/FAP Positive Disease

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
SULINDAC	4	3
EFLORNITHINE	4	2
GUSELKUMAB	4	2
INOTERSEN	4	2
THIAMINE ION	4	2
URSODIOL	4	2
AZITHROMYCIN	4	1
FLUOROESTRADIOL F-18	4	1
INULIN	4	1
LITHIUM CARBONATE	4	1
NICLOSAMIDE	4	1
OBETICHOLIC ACID	4	1
TIPIRACIL	4	1
TRIFLURIDINE	4	1
CURCUMIN	3	2
GOZETOTIDE ALF-18	3	1
L19IL2	3	1
EFLORNITHINE, (S)-	2	2
DARLEUKIN	2	1
TAK-733	2	1
DORDAVIPRONE HYDROCHLORIDE	1	1
CHEMBL4446459	0	2
CHEMBL4299381	0	1
CHEMBL5409583	0	1
CHEMBL4303680	0	1
CHEMBL5197244	0	1
CHEMBL5412701	0	1