Condition (MeSH)
Wiskott-Aldrich Syndrome
ClinicalTrials.gov lists 41 trials for Wiskott-Aldrich Syndrome or a narrower condition beneath it. National Human Genome Research Institute (NHGRI) has the most among industry and government lead sponsors, with 3.
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Trials per phase
41 of the 41 trials are tagged with Wiskott-Aldrich Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Wiskott-Aldrich Syndrome trials start? (22)
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 3
- Phase 4
- 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 | 2 |
| 2019 | 2 |
| 2018 | 2 |
| 2017 | 1 |
| 2016 | 1 |
| 2015 | 1 |
| 2014 | 4 |
| 2013 | 3 |
Showing 10 of 22 earlier years.
No forecast is made for this condition: it averages 0.4 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 41 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Wiskott-Aldrich Syndrome trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 9 |
| Phase 2 | 9 |
| Phase 3 | 3 |
| Phase 4 | 2 |
| Not applicable | 4 |
| Observational | 11 |
How many Wiskott-Aldrich Syndrome trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 2 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 4 |
| Completed | 23 |
| Terminated | 5 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Wiskott-Aldrich 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.
| Sponsor | Trials |
|---|---|
| National Human Genome Research Institute (NHGRI) | 3 |
| Capital Research Institute of Pediatrics | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
Which drugs are being tested for Wiskott-Aldrich 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 | 4 |
| Melphalan | 3 |
| Busulfan | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| SQ Fludarabine | 5 |
| HD- Methotrexate (MTX) | 3 |
| Peripheral blood stem cell transplantation | 3 |
| Total Body Irradiation | 3 |
| Globulin, Immune | 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 Wiskott-Aldrich Syndrome trials report? (10)
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 |
|---|---|---|
| Pyrexia | 5 | 2 |
| Hypoxia: Decreased O2 sat requiring support | 4 | 1 |
| Graft versus host disease | 3 | 1 |
| Acute graft versus host disease in intestine | 2 | 1 |
| Anaphylaxis reaction (rATG) | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Wiskott-Aldrich Syndrome? (31)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy. | Nature communications | 2023 | Yes |
| Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. | Blood | 2023 | Yes |
| Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. | Nature medicine | 2022 | Yes |
| Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. | Blood | 2020 | Yes |
| Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report. | Frontiers in immunology | 2020 | Yes |
Showing 5 of 31 publications.
Prevalence (24)
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 |
|---|---|---|---|---|---|
| Wiskott-Aldrich syndrome | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Unspecified |
| Wiskott-Aldrich syndrome | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Wiskott-Aldrich syndrome | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Wiskott-Aldrich syndrome | Prevalence at birth | 1-9 / 1 000 000 | Switzerland | Not stated | Exact |
| Wiskott-Aldrich syndrome | Point prevalence | <1 / 1 000 000 | Australia | Not stated | Unspecified |
Showing 5 of 24 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Wiskott-Aldrich syndrome | 906 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Wiskott-Aldrich syndrome | 906 | 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.