Condition (MeSH)
GATA2 Deficiency
ClinicalTrials.gov lists 18 trials for GATA2 Deficiency or a narrower condition beneath it. National Cancer Institute (NCI) has the most among industry and government lead sponsors, with 4.
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Trials per phase
18 of the 18 trials are tagged with GATA2 Deficiency itself; the rest are tagged with a narrower condition beneath it.
When did GATA2 Deficiency trials start? (14)
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
- Observational
Top of scale: 2
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 |
| 2025 | 2 |
| 2024 | 2 |
| 2023 | 1 |
| 2022 | 1 |
| 2021 | 2 |
| 2020 | 1 |
| 2019 | 2 |
| 2018 | 1 |
| 2017 | 1 |
Showing 10 of 14 earlier years.
No forecast is made for this condition: it averages 1.4 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 6 trials still running at the 2026-10-09 snapshot; registered median of 57 months across 5 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are GATA2 Deficiency trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 8 |
| Phase 1/2 | 3 |
| Phase 2 | 4 |
| Phase 2/3 | 1 |
| Observational | 2 |
How many GATA2 Deficiency trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 6 |
| Active, not recruiting | 2 |
| Completed | 2 |
| Suspended | 1 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most GATA2 Deficiency trials? (8)
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 Cancer Institute (NCI) | 4 |
| AB Science | 1 |
| Apollo Therapeutics Ltd | 1 |
| Celyad Oncology SA | 1 |
| National Institute of Allergy and Infectious Diseases (NIAID) | 1 |
Showing 5 of 8 sponsors.
Which drugs are being tested for GATA2 Deficiency? (2)
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 |
|---|---|
| NIVOLUMAB | 3 |
| Cytoxan | 2 |
Interventions as named in the registry (13)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Azacitidine MTD | 4 |
| Bone marrow aspiration | 3 |
| Venetoclax 600 | 3 |
| Total Body Irradiation | 2 |
| SQ Fludarabine | 2 |
Showing 5 of 13 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 GATA2 Deficiency 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 |
|---|---|---|
| Febrile neutropenia | 29 | 3 |
| Platelet count decreased | 23 | 2 |
| Anemia | 21 | 2 |
| Sepsis | 21 | 2 |
| Lung infection | 19 | 2 |
Showing 5 of 10 adverse events.
Which publications cover GATA2 Deficiency? (4)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| A novel GATA2 distal enhancer mutation results in MonoMAC syndrome in 2 second cousins. | Blood advances | 2023 | Yes |
| A Nationwide Study of GATA2 Deficiency in Norway-the Majority of Patients Have Undergone Allo-HSCT. | Journal of clinical immunology | 2022 | Yes |
| Sequencing of RNA in single cells reveals a distinct transcriptome signature of hematopoiesis in GATA2 deficiency. | Blood advances | 2020 | Yes |
| Life-Threatening Primary Varicella Zoster Virus Infection With Hemophagocytic Lymphohistiocytosis-Like Disease in GATA2 Haploinsufficiency Accompanied by Expansion of Double Negative T-Lymphocytes. | Frontiers in immunology | 2018 | Yes |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence, rare-disease identity, and sub-conditions in mesh.