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Condition (MeSH)

Xanthomatosis

ClinicalTrials.gov lists 29 trials for Xanthomatosis or a narrower condition beneath it. TRPHARM has the most among industry and government lead sponsors, with 3.

MeSH D014973 Nutritional and Metabolic Diseases C18
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Phase 1 Phase 1 2 Phase 2 Phase 2 2 Phase 3 Phase 3 3 Phase 4 Phase 4 1 Not applicable Not applicable 8 Observational Observational 13 Phase 1 Phase 1 2 Phase 2 Phase 2 2 Phase 3 Phase 3 3 Phase 4 Phase 4 1 Not applicable Not applicable 8 Observational Observational 13

Trials per phase

6 of the 29 trials are tagged with Xanthomatosis itself; the rest are tagged with a narrower condition beneath it.

Get the Xanthomatosis report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Xanthomatosis trials start? (17)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.

Trials over time, by phase

  • Phase 1
  • Phase 2
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 5

1996: 1 trials (Phase 2 1) 1999: 1 trials (Not applicable 1) 2006: 1 trials (Phase 3 1) 2010: 1 trials (Observational 1) 2013: 1 trials (Not applicable 1) 2014: 2 trials (Observational 2) 2015: 1 trials (Observational 1) 2016: 1 trials (Observational 1) 2017: 1 trials (Observational 1) 2018: 5 trials (Observational 2, Not applicable 2, Phase 1 1) 2019: 2 trials (Observational 2) 2020: 1 trials (Phase 3 1) 2021: 3 trials (Observational 2, Not applicable 1) 2022: 3 trials (Observational 1, Phase 4 1, Phase 1 1) 2024: 3 trials (Not applicable 2, Phase 3 1) 2025: 1 trials (Not applicable 1)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 1
2025 1
2024 3
2022 3
2021 3
2020 1
2019 2
2018 5
2017 1
2016 1

No forecast is made for this condition: it averages 1.6 trial starts a year over the five years to September, under the 5 a year the method needs.

Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Xanthomatosis trials in? (6)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 2
Phase 2 2
Phase 3 3
Phase 4 1
Not applicable 8
Observational 13

How many Xanthomatosis trials are recruiting? (8)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 1
Recruiting 4
Enrolling by invitation 2
Active, not recruiting 2
Completed 9

Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Xanthomatosis trials? (6)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
TRPHARM 3
Humanis Saglık Anonim Sirketi 1
Leadiant Biosciences Ltd. 1
Leadiant Biosciences, Inc. 1
Mirum Pharmaceuticals, Inc. 1

Interventions as named in the registry (15)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Chenodeoxycholic Acid 5
Rescue Medication CDCA 250 mg TID 1
Blinded CDCA 250 mg TID 1
Blood tests 1
acid Zoledronic 1

These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Xanthomatosis trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Angina pectoris 2 1
Cerebrovascular accident 2 1
Cystocele 2 1
Haematoma 2 1
Hypokalaemia 2 1

Which publications cover Xanthomatosis? (28)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Ophthalmologic Findings in Pediatric Erdheim-Chester Disease: A Literature Review With a Novel Case Report. Ophthalmic plastic and reconstructive surgery 2023 Yes
Topical Simvastatin Improves Lesions of Diffuse Normolipemic Plane Xanthoma by Inhibiting Foam Cell Pyroptosis. Frontiers in immunology 2022 Yes
Alcohol consumption, smoking and development of visible age-related signs: a prospective cohort study. Journal of epidemiology and community health 2017 Yes
Successful Treatment of a Progressive BRAF V600E-Mutated Anaplastic Pleomorphic Xanthoastrocytoma With Vemurafenib Monotherapy. Journal of clinical oncology : official journal of the American Society of Clinical Oncology 2016 Yes
Management of cholestatic pruritus in children with Alagille syndrome: Case report and literature review. Archives de pediatrie : organe officiel de la Societe francaise de pediatrie 2016 Yes

Sub-conditions in MeSH (2)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: prevalence and rare-disease identity.

Browse all condition evidence pages