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Condition (MeSH)

Anemia, Aplastic

ClinicalTrials.gov lists 458 trials for Anemia, Aplastic or a narrower condition beneath it. National Heart, Lung, and Blood Institute (NHLBI) has the most among industry and government lead sponsors, with 46.

MeSH D000741 Hemic and Lymphatic Diseases C15 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 36 Phase 1/2 Phase 1/2 66 Phase 2 Phase 2 151 Phase 2/3 Phase 2/3 14 Phase 3 Phase 3 17 Phase 4 Phase 4 20 Not applicable Not applicable 56 Observational Observational 93 Phase 1 Phase 1 36 Phase 1/2 Phase 1/2 66 Phase 2 Phase 2 151 Phase 2/3 Phase 2/3 14 Phase 3 Phase 3 17 Phase 4 Phase 4 20 Not applicable Not applicable 56 Observational Observational 93

Trials per phase

1 more section with rows

354 of the 458 trials are tagged with Anemia, Aplastic itself; the rest are tagged with a narrower condition beneath it.

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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 Anemia, Aplastic trials start? (34)

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. 2 trials are 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: 29

1993: 4 trials (Phase 1/2 1, Phase 1 3) 1994: 2 trials (Phase 2 2) 1995: 2 trials (Not applicable 1, Phase 2 1) 1996: 3 trials (Not applicable 1, Phase 2 2) 1997: 1 trials (Phase 2 1) 1998: 5 trials (Observational 1, Phase 3 1, Phase 2/3 1, Phase 2 1, Phase 1 1) 1999: 4 trials (Not applicable 1, Phase 2 3) 2000: 15 trials (Observational 1, Not applicable 5, Phase 2/3 1, Phase 2 2, Phase 1/2 3, Phase 1 3) 2001: 11 trials (Observational 2, Not applicable 3, Phase 3 1, Phase 2 3, Phase 1/2 1, Phase 1 1) 2002: 12 trials (Observational 1, Not applicable 1, Phase 2/3 2, Phase 2 5, Phase 1/2 2, Phase 1 1) 2003: 9 trials (Observational 4, Phase 2 5) 2004: 7 trials (Observational 1, Not applicable 2, Phase 1/2 2, Phase 1 2) 2005: 15 trials (Observational 1, Not applicable 2, Phase 3 2, Phase 2 7, Phase 1/2 1, Phase 1 2) 2006: 11 trials (Observational 2, Phase 2/3 1, Phase 2 5, Phase 1/2 2, Phase 1 1) 2007: 16 trials (Observational 4, Not applicable 2, Phase 4 1, Phase 2/3 1, Phase 2 4, Phase 1/2 3, Phase 1 1) 2008: 10 trials (Observational 1, Not applicable 1, Phase 2 5, Phase 1/2 2, Phase 1 1) 2009: 20 trials (Observational 5, Not applicable 3, Phase 2 9, Phase 1/2 3) 2010: 12 trials (Observational 3, Not applicable 1, Phase 4 1, Phase 3 1, Phase 2 4, Phase 1/2 2) 2011: 18 trials (Observational 3, Not applicable 2, Phase 4 1, Phase 3 1, Phase 2 6, Phase 1/2 4, Phase 1 1) 2012: 14 trials (Not applicable 1, Phase 4 1, Phase 2 8, Phase 1/2 2, Phase 1 2) 2013: 16 trials (Observational 4, Not applicable 1, Phase 4 1, Phase 2 5, Phase 1/2 4, Phase 1 1) 2014: 15 trials (Observational 1, Not applicable 1, Phase 4 1, Phase 2 7, Phase 1/2 4, Phase 1 1) 2015: 10 trials (Observational 2, Not applicable 1, Phase 3 1, Phase 2/3 1, Phase 2 3, Phase 1/2 1, Phase 1 1) 2016: 16 trials (Observational 3, Not applicable 1, Phase 4 2, Phase 3 1, Phase 2/3 1, Phase 2 5, Phase 1/2 3) 2017: 18 trials (Observational 3, Not applicable 3, Phase 4 2, Phase 2 4, Phase 1/2 4, Phase 1 2) 2018: 19 trials (Observational 4, Not applicable 3, Phase 2 8, Phase 1/2 3, Phase 1 1) 2019: 13 trials (Observational 2, Phase 4 1, Phase 3 1, Phase 2/3 3, Phase 2 3, Phase 1/2 2, Phase 1 1) 2020: 16 trials (Observational 6, Phase 3 1, Phase 2 6, Phase 1/2 1, Phase 1 2) 2021: 18 trials (Observational 5, Not applicable 3, Phase 2/3 2, Phase 2 6, Phase 1/2 1, Phase 1 1) 2022: 19 trials (Observational 4, Not applicable 7, Phase 3 1, Phase 2 5, Phase 1/2 1, Phase 1 1) 2023: 20 trials (Observational 5, Not applicable 2, Phase 4 4, Phase 3 1, Phase 2 5, Phase 1/2 2, Phase 1 1) 2024: 29 trials (Observational 7, Not applicable 2, Phase 4 5, Phase 3 1, Phase 2 7, Phase 1/2 4, Phase 1 3) 2025: 19 trials (Observational 6, Not applicable 2, Phase 3 1, Phase 2 6, Phase 1/2 3, Phase 1 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
2026 (partial year) 26
2025 19
2024 29
2023 20
2022 19
2021 18
2020 16
2019 13
2018 19
2017 18

Showing 10 of 34 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 31 trials starting in 2026 and about 26 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 37 trials still running at the 2026-10-09 snapshot; registered median of 54 months across 243 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Anemia, Aplastic trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 36
Phase 1/2 66
Phase 2 151
Phase 2/3 14
Phase 3 17
Phase 4 20
Not applicable 56
Observational 93
Expanded access 5

How many Anemia, Aplastic trials are recruiting? (11)

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

Trials by recruitment status
Status Trials
Not yet recruiting 24
Recruiting 63
Enrolling by invitation 4
Active, not recruiting 22
Available 4

Showing 5 of 11 statuses.

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

Which lead sponsors run the most Anemia, Aplastic 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.

Lead sponsors
Sponsor Trials
National Heart, Lung, and Blood Institute (NHLBI) 46
Novartis Pharmaceuticals 18

Which drugs are being tested for Anemia, Aplastic? (5)

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
Cytoxan 63
Busulfan 21
Melphalan 20
Eltrombopag 20
METHYLPREDNISOLONE 17

Interventions as named in the registry (10)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
SQ Fludarabine 69
Thymoglobulin [Anti-Thymocyte Globulin (Rabbit)] 49
Cyclosporine 0.05% 47
Total Body Irradiation 34
Mofetil Mycophenolate 25

Showing 5 of 10 interventions.

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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 Anemia, Aplastic trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Febrile neutropenia 95 23
1 yr TRM 93 1
Pyrexia 68 13
Sepsis 61 26
Death 40 10

Showing 5 of 10 adverse events.

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Which publications cover Anemia, Aplastic? (289)

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

Publications
Publication Journal Year Major topic
Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials. Annals of hematology 2025 Yes
Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial. Blood advances 2025 Yes
Targeting CD38 with daratumumab for platelet transfusion refractoriness in aplastic anemia. Blood 2025 Yes
Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes. NEJM evidence 2024 Yes
Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia. Immunotherapy 2024 Yes

Showing 5 of 289 publications.

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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.

Checked, with nothing recorded for this condition: prevalence and rare-disease identity.

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