Skip to content
Pharma Observatory a Scientist.com platform

Condition (MeSH)

Crigler-Najjar Syndrome

ClinicalTrials.gov lists 13 trials for Crigler-Najjar Syndrome or a narrower condition beneath it. Cellaion SA has the most among industry and government lead sponsors, with 3.

MeSH D003414 Congenital / Hereditary / Neonatal C16 C18 Listed by Orphanet as a rare disease
Email me this page as a PDF

Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.

Phase 1 Phase 1 2 Phase 1/2 Phase 1/2 2 Phase 2 Phase 2 1 Not applicable Not applicable 3 Observational Observational 5 Phase 1 Phase 1 2 Phase 1/2 Phase 1/2 2 Phase 2 Phase 2 1 Not applicable Not applicable 3 Observational Observational 5

Trials per phase

13 of the 13 trials are tagged with Crigler-Najjar Syndrome itself; the rest are tagged with a narrower condition beneath it.

Get the Crigler-Najjar Syndrome 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 Crigler-Najjar Syndrome trials start? (9)

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 1/2
  • Phase 2
  • Not applicable
  • Observational

Top of scale: 2

2003: 1 trials (Not applicable 1) 2011: 1 trials (Phase 1 1) 2012: 1 trials (Phase 1/2 1) 2013: 1 trials (Observational 1) 2014: 2 trials (Observational 1, Not applicable 1) 2017: 2 trials (Observational 1, Phase 1 1) 2018: 2 trials (Observational 1, Not applicable 1) 2022: 1 trials (Observational 1) 2024: 2 trials (Phase 2 1, Phase 1/2 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
2024 2
2022 1
2018 2
2017 2
2014 2
2013 1
2012 1
2011 1
2003 1

No forecast is made for this condition: it averages 0.6 trial starts a year over the five years to September, under the 5 a year the method needs.

Recruitment duration, a model prediction: 2 trials 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 Crigler-Najjar Syndrome trials in? (5)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 2
Phase 1/2 2
Phase 2 1
Not applicable 3
Observational 5

How many Crigler-Najjar Syndrome trials are recruiting? (4)

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

Trials by recruitment status
Status Trials
Recruiting 3
Completed 6
Terminated 3
Unknown status 1

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

Which lead sponsors run the most Crigler-Najjar Syndrome trials? (3)

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
Cellaion SA 3
Audentes Therapeutics 2
Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare 1

Which drugs are being tested for Crigler-Najjar Syndrome? (1)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Imlifidase 1

Interventions as named in the registry (6)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
GNT0003 2
Orlistat 60 1
human hepatocyte transplantation 1
GT-UGT1A1-AAV8-02 1
Blood sample for immunology 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 publications cover Crigler-Najjar Syndrome? (4)

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

Publications
Publication Journal Year Major topic
Gene Therapy in Patients with the Crigler-Najjar Syndrome. The New England journal of medicine 2023 Yes
Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder. Pediatrics 2003 Yes
Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation. The New England journal of medicine 1998 Yes
Vigintiphobia revisited. Pediatrics 2005 No

Prevalence (6)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Crigler-Najjar syndrome Prevalence at birth 1-9 / 1 000 000 Europe Not stated Exact
Crigler-Najjar syndrome Prevalence at birth 1-9 / 1 000 000 Europe Not stated Unspecified
Crigler-Najjar syndrome Point prevalence 1-9 / 100 000 Europe Not stated Unspecified
Crigler-Najjar syndrome Point prevalence 1-9 / 100 000 Europe Not stated Exact
Crigler-Najjar syndrome Prevalence at birth 1-9 / 1 000 000 Europe Not stated Exact

Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Crigler-Najjar syndrome 205 Exact Orphanet Rare Disease Ontology (ORDO)
Crigler-Najjar syndrome 205 Exact Orphadata Science knowledge files, Product 1

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: serious adverse events in this condition's trials and sub-conditions in mesh.

Browse all condition evidence pages