Condition (MeSH)
Severe Combined Immunodeficiency
ClinicalTrials.gov lists 77 trials for Severe Combined Immunodeficiency or a narrower condition beneath it. National Institute of Allergy and Infectious Diseases (NIAID) has the most among industry and government lead sponsors, with 10.
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Trials per phase (log scale)
1 more section with rows
56 of the 77 trials are tagged with Severe Combined Immunodeficiency itself; the rest are tagged with a narrower condition beneath it.
When did Severe Combined Immunodeficiency trials start? (28)
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. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 6
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) | 3 |
| 2025 | 2 |
| 2024 | 1 |
| 2023 | 1 |
| 2022 | 3 |
| 2021 | 2 |
| 2020 | 4 |
| 2019 | 2 |
| 2018 | 6 |
| 2016 | 1 |
Showing 10 of 28 earlier years.
No forecast is made for this condition: it averages 1.8 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 5 trials still running at the 2026-10-09 snapshot; registered median of 54 months across 36 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Severe Combined Immunodeficiency trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 9 |
| Phase 1/2 | 20 |
| Phase 2 | 8 |
| Phase 2/3 | 1 |
| Phase 3 | 2 |
| Phase 4 | 1 |
| Not applicable | 7 |
| Observational | 27 |
| Expanded access | 2 |
How many Severe Combined Immunodeficiency trials are recruiting? (11)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 12 |
| Enrolling by invitation | 5 |
| Active, not recruiting | 4 |
| Available | 1 |
Showing 5 of 11 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Severe Combined Immunodeficiency trials? (4)
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 Institute of Allergy and Infectious Diseases (NIAID) | 10 |
| National Human Genome Research Institute (NHGRI) | 6 |
| National Heart, Lung, and Blood Institute (NHLBI) | 3 |
| Phenox GmbH | 2 |
Which drugs are being tested for Severe Combined Immunodeficiency? (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 |
|---|---|
| Busulfan | 11 |
| Cytoxan | 3 |
| Melphalan | 3 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| SQ Fludarabine | 6 |
| Alemtuzumab s.c. | 4 |
| Palifermin before only | 3 |
| Total Body Irradiation | 3 |
| data collection | 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 Severe Combined Immunodeficiency 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 |
|---|---|---|
| Infection | 13 | 2 |
| Pyrexia | 13 | 2 |
| Fever | 5 | 1 |
| Infection requiring hospitalization | 5 | 1 |
| Device related infection | 4 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Severe Combined Immunodeficiency? (138)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency. | The New England journal of medicine | 2025 | Yes |
| Clinical and functional spectrum of RAC2-related immunodeficiency. | Blood | 2024 | Yes |
| Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency. | Nature medicine | 2024 | Yes |
| Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients. | The Journal of allergy and clinical immunology | 2023 | Yes |
| Use of Monoclonal Antibodies in a Pediatric Patient With Severe Combined Immunodeficiency and Persistent SARS-CoV-2 Infection. | The Pediatric infectious disease journal | 2023 | Yes |
Showing 5 of 138 publications.
Prevalence (27)
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 |
|---|---|---|---|---|---|
| Severe combined immunodeficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Severe combined immunodeficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Unspecified |
| Severe combined immunodeficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Severe combined immunodeficiency | Prevalence at birth | 1-9 / 100 000 | France | Not stated | Exact |
| Severe combined immunodeficiency | Prevalence at birth | 1-9 / 100 000 | Costa rica | Not stated | Exact |
Showing 5 of 27 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Severe combined immunodeficiency | 183660 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Severe combined immunodeficiency | 183660 | Exact | Orphadata Science knowledge files, Product 1 |
Sub-conditions in MeSH (1)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.