Condition (MeSH)
Chediak-Higashi Syndrome
ClinicalTrials.gov lists 16 trials for Chediak-Higashi Syndrome or a narrower condition beneath it. National Human Genome Research Institute (NHGRI) has the most among industry and government lead sponsors, with 2.
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Trials per phase
16 of the 16 trials are tagged with Chediak-Higashi Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Chediak-Higashi Syndrome trials start? (11)
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
- Phase 2/3
- Not applicable
- Observational
Top of scale: 4
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.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 1 |
| 2022 | 1 |
| 2020 | 1 |
| 2014 | 2 |
| 2013 | 1 |
| 2012 | 1 |
| 2010 | 1 |
| 2008 | 1 |
| 2004 | 1 |
| 2002 | 2 |
Showing 10 of 11 earlier years.
No forecast is made for this condition: it averages 0.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 16 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Chediak-Higashi Syndrome trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 1 |
| Phase 2 | 3 |
| Phase 2/3 | 1 |
| Not applicable | 4 |
| Observational | 5 |
How many Chediak-Higashi Syndrome trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 3 |
| Active, not recruiting | 2 |
| Completed | 6 |
| Terminated | 3 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Chediak-Higashi Syndrome trials? (2)
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.
| Sponsor | Trials |
|---|---|
| National Human Genome Research Institute (NHGRI) | 2 |
| National Institute of Allergy and Infectious Diseases (NIAID) | 1 |
Which drugs are being tested for Chediak-Higashi Syndrome? (3)
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.
| Drug | Trials |
|---|---|
| Cytoxan | 3 |
| Busulfan | 3 |
| Melphalan | 3 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 3 |
| Thymoglobulin [Anti-Thymocyte Globulin (Rabbit)] | 3 |
| Cyclosporine 0.05% | 3 |
| SQ Fludarabine | 3 |
| Stem Cell Transplant | 2 |
Showing 5 of 12 interventions.
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 Chediak-Higashi Syndrome trials report? (5)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Chronic graft versus host disease | 2 | 1 |
| Stomatitis | 2 | 1 |
| Death | 1 | 1 |
| Multi-organ failure | 1 | 1 |
| Secondary Malignancy | 1 | 1 |
Which publications cover Chediak-Higashi Syndrome? (2)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Defective lysosomal enzyme secretion in kidneys of Chediak-Higashi (beige) mice. | The Journal of cell biology | 1975 | Yes |
| [Clinical and genetic aspects of albinism]. | Presse medicale (Paris, France : 1983) | 2017 | 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.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Chédiak-Higashi syndrome | Cases families | Worldwide | Not stated | Exact | |
| Chédiak-Higashi syndrome | Point prevalence | <1 / 1 000 000 | Worldwide | Not stated | Exact |
| Chédiak-Higashi syndrome | Point prevalence | <1 / 1 000 000 | Worldwide | Not stated | Unspecified |
| Chédiak-Higashi syndrome | Cases families | Worldwide | Not stated | Exact | |
| Chédiak-Higashi syndrome | Cases families | Worldwide | Not stated | Unspecified |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Chédiak-Higashi syndrome | 167 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Chédiak-Higashi syndrome | 167 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.