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ORPHA:70567

Cholangiocarcinoma

low confidenceDisorder

Also known as: Bile duct cancer · CCA

Publications

74,568

Trials

748

Interventional, condition-specific

Researchers

1,482

Distinct authors in sample

Gene link

—

Readiness

5/6

Stages with a signal

Clinical definition (Orphanet)

Cholangiocarcinoma (CCA) is a biliary tract cancer (BTC) originating in the epithelium of the biliary tree, either intra or extra hepatic.

How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (6)

CC · Cholangiocar.- intra/extrahepatic · Cholangiocellular carcinoma · cholangiocarcinoma · cholangiocarcinoma, intrahepatic and extrahepatic bile ducts (adenocarcinoma) · cholangiocarcinoma, malignant

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

5/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    74,568 matched papers (54,371 in last 10 years) Source

  3. Phenotype characterisedPresent

    18 HPO annotations (e.g. Pruritus; Fever; Abdominal pain) Source

  4. Animal modelPresent

    4 genotype models (Mus musculus) Source

  5. Orphan designationPresent

    11 FDA · 3 EMA designations (10 FDA orphan-indication approvals) — e.g. Gemcitabine Source

  6. Interventional trialPresent

    748 matched on ClinicalTrials.gov (193 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

18

Associated phenotypes · MONDO:0019087

  • Pruritus
  • Fever
  • Abdominal pain
  • Anorexia
  • Acholic stools

Showing 5 of 18 — open Monarch for the full list.

Animal models (Monarch / Alliance)

4

Model associations linked to this Mondo ID

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

14

Designations · 10 with FDA orphan-indication approval

  • FDA GemcitabineCholangiocarcinoma · 2020-05-18 · Not FDA Approved for Orphan Indication
  • FDA InfigratinibCholangiocarcinoma · 2019-09-11 · Not FDA Approved for Orphan Indication
  • FDA fimaporfinCholangiocarcinoma · 2017-09-27 · Not FDA Approved for Orphan Indication
  • FDA ivosidenibCholangiocarcinoma · 2017-04-26 · Not FDA Approved for Orphan Indication
  • FDA RedaporfinCholangiocarcinoma · 2017-04-12 · Not FDA Approved for Orphan Indication
  • FDA SilmitasertibCholangiocarcinoma · 2016-12-22 · Not FDA Approved for Orphan Indication
  • FDA gemcitabineCholangiocarcinoma · 2016-01-11 · Not FDA Approved for Orphan Indication
  • FDA (R)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochlorideCholangiocarcinoma · 2015-11-19 · Not FDA Approved for Orphan Indication

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

145

Drugs / clinical candidates · MONDO_0019087

CTD chemicals (MyDisease.info)

29 associated chemicals · 528 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • 5-(2,4-dihydroxy-5-isopropylphenyl)-4-(4-morpholin-4-ylmethylphenyl)isoxazole-3-carboxylic acid ethylamide · therapeutic
  • Carmustine · therapeutic
  • futibatinib · therapeutic
  • Melatonin · therapeutic
  • Thalidomide · therapeutic
  • 1,2,3-trichloropropane · marker/mechanism
  • 2-aminofluorene · marker/mechanism
  • 2-nitrotoluene · marker/mechanism
  • 2,2-bis(bromomethyl)-1,3-propanediol · marker/mechanism
  • 2,3',4,4',5-pentachlorobiphenyl · marker/mechanism
  • 3,4,5,3',4'-pentachlorobiphenyl · marker/mechanism
  • Anabolic Agents · marker/mechanism

Pathways: Citrate cycle (TCA cycle); Arginine biosynthesis; Lysine degradation; Arginine and proline metabolism; Glutathione metabolism; Inositol phosphate metabolism; Arachidonic acid metabolism; Metabolic pathways

MyDisease.info · MONDO:0019087

Literature

Is anyone studying this?

74,568

74,568 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

74,568 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

54,371 in the last 10 years · low confidence

Phrase hits: 74,568 · MeSH hits: 1,865

Open Europe PMC search

Who's working on it?

1,482

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Zhang Y7 papers · 2026

    School of Pharmacy, Lanzhou University, Lanzhou, 730000, People's Republic of China.

    Papers in Europe PMC
  2. 02
    Li X6 papers · 2026

    Department of Scientific Research, Nanyang City Center Hospital, Nanyang, China.

    Papers in Europe PMC
  3. 03
    Wang Z5 papers · 2026

    Department of Gastroenterology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China.

    Papers in Europe PMC
  4. 04
    Zhang J5 papers · 2026

    Department of Clinical Oncology, Affiliated Jinling Hospital, Medical School of Nanjing University, Nanjing, 210000, China.

    Papers in Europe PMC
  5. 05
    Bessho K4 papers · 2026

    Pancreatobiliary Advanced Medical Center, Osaka Medical and Pharmaceutical University Hospital, Osaka, Japan.

    Papers in Europe PMC
  6. 06
    Kim JS4 papers · 2026

    Division of Medical Oncology, Department of Internal Medicine, CHA Bundang Medical Center, CHA University, 59 Yatap-ro, Bundang-gu, Seongnam 13496, Republic of Korea.

    Papers in Europe PMC
  7. 07
    Li H4 papers · 2026

    Department of Clinical Oncology, Affiliated Nanjing Tianyinshan Hospital, Pharmaceutical University, Nanjing, 210000, China.

    Papers in Europe PMC
  8. 08
    Nishikawa H4 papers · 2026

    Second Department of Internal Medicine, Osaka Medical and Pharmaceutical University, Osaka, Japan.

    Papers in Europe PMC
  9. 09
    Ogura T4 papers · 2026

    Pancreatobiliary Advanced Medical Center, Osaka Medical and Pharmaceutical University Hospital, 2-7 Daigakuchou, Takatsukishi, Osaka 569-8686, Japan.

    Papers in Europe PMC
  10. 10
    Wang J4 papers · 2026

    Department of General Surgery, Renmin Hospital of Wuhan University, Wuhan, China.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

748

interventional trials for this specific condition

748 interventional trials matched this specific condition name; 193 currently recruiting in our sample.

Data as of 11 September 2026

748 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 99.8th percentile).

low confidence · 99.8th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

748 interventional trials matched after quoted-phrase search and title/condition post-filter.

Observational and natural-history studies

195 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 106 · after dedupe 104 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 104 · dropped 0 · fetched 2026-07-30

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (104)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Cholangiocarcinoma — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Cholangiocarcinoma" OR "Bile duct cancer" OR "Cholangiocar.- intra/extrahepatic" OR "Cholangiocellular carcinoma" OR "cholangiocarcinoma, intrahepatic and extrahepatic bile ducts (adenocarcinoma)" OR "cholangiocarcinoma, malignant"

Run this search on Europe PMC

MeSH descriptor terms unioned into the query: Cholangiocarcinoma

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Cholangiocarcinoma" OR "Bile duct cancer" OR "Cholangiocar.- intra/extrahepatic" OR "Cholangiocellular carcinoma" OR "cholangiocarcinoma, intrahepatic and extrahepatic bile ducts (adenocarcinoma)" OR "cholangiocarcinoma, malignant"

Interventional trials matched via: both, phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 748 interventional · 195 observational · 6 expanded access. Only interventional studies enter the trial headline.

Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: CCA; CC

Confidence reasoning

  • Preferred label is short or not clearly distinctive
  • 2 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-27T01:31:39.776Z