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Condition (MeSH)

Anemia, Hemolytic, Congenital

ClinicalTrials.gov lists 1,530 trials for Anemia, Hemolytic, Congenital or a narrower condition beneath it. National Heart, Lung, and Blood Institute (NHLBI) has the most among industry and government lead sponsors, with 64.

MeSH D000745 Hemic and Lymphatic Diseases C15 C16
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Phase 1 Phase 1 152 Phase 1/2 Phase 1/2 92 Phase 2 Phase 2 266 Phase 2/3 Phase 2/3 36 Phase 3 Phase 3 117 Phase 4 Phase 4 77 Not applicable Not applicable 350 Observational Observational 433 Phase 1 Phase 1 152 Phase 1/2 Phase 1/2 92 Phase 2 Phase 2 266 Phase 2/3 Phase 2/3 36 Phase 3 Phase 3 117 Phase 4 Phase 4 77 Not applicable Not applicable 350 Observational Observational 433

Trials per phase

1 more section with rows

11 of the 1,530 trials are tagged with Anemia, Hemolytic, Congenital 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, Hemolytic, Congenital trials start? (35)

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. 4 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
  • Other (Expanded access)

Top of scale: 97

1990: 1 trials (Observational 1) 1992: 2 trials (Phase 3 1, Phase 2 1) 1993: 2 trials (Observational 2) 1994: 3 trials (Phase 3 1, Phase 2 2) 1995: 3 trials (Observational 1, Not applicable 2) 1997: 3 trials (Not applicable 1, Phase 3 1, Phase 2 1) 1998: 3 trials (Observational 1, Phase 2 1, Phase 1 1) 1999: 8 trials (Not applicable 1, Phase 2 5, Phase 1/2 2) 2000: 11 trials (Observational 1, Not applicable 1, Phase 4 1, Phase 3 3, Phase 2 4, Phase 1 1) 2001: 8 trials (Observational 2, Phase 2 5, Phase 1 1) 2002: 9 trials (Observational 2, Not applicable 2, Phase 4 1, Phase 3 1, Phase 2/3 1, Phase 2 2) 2003: 14 trials (Observational 1, Not applicable 2, Phase 3 2, Phase 2/3 1, Phase 2 5, Phase 1/2 1, Phase 1 2) 2004: 28 trials (Observational 6, Not applicable 1, Phase 4 3, Phase 3 4, Phase 2 10, Phase 1/2 1, Phase 1 3) 2005: 29 trials (Observational 9, Not applicable 2, Phase 4 3, Phase 3 4, Phase 2 6, Phase 1/2 2, Phase 1 3) 2006: 35 trials (Other 1, Observational 10, Not applicable 2, Phase 4 1, Phase 3 10, Phase 2 6, Phase 1/2 2, Phase 1 3) 2007: 30 trials (Observational 11, Not applicable 4, Phase 4 2, Phase 3 2, Phase 2 8, Phase 1/2 2, Phase 1 1) 2008: 40 trials (Observational 16, Not applicable 6, Phase 4 1, Phase 3 1, Phase 2/3 2, Phase 2 6, Phase 1/2 5, Phase 1 3) 2009: 46 trials (Observational 16, Not applicable 7, Phase 4 2, Phase 3 1, Phase 2/3 3, Phase 2 8, Phase 1/2 7, Phase 1 2) 2010: 44 trials (Observational 13, Not applicable 10, Phase 4 1, Phase 3 4, Phase 2/3 1, Phase 2 8, Phase 1 7) 2011: 33 trials (Observational 10, Not applicable 2, Phase 4 3, Phase 3 3, Phase 2 8, Phase 1/2 4, Phase 1 3) 2012: 63 trials (Observational 21, Not applicable 14, Phase 4 1, Phase 3 3, Phase 2/3 4, Phase 2 10, Phase 1/2 4, Phase 1 6) 2013: 57 trials (Observational 15, Not applicable 12, Phase 4 4, Phase 3 3, Phase 2 12, Phase 1/2 5, Phase 1 6) 2014: 74 trials (Observational 17, Not applicable 16, Phase 4 4, Phase 3 2, Phase 2/3 4, Phase 2 17, Phase 1/2 7, Phase 1 7) 2015: 68 trials (Observational 13, Not applicable 19, Phase 4 8, Phase 3 5, Phase 2/3 1, Phase 2 7, Phase 1/2 3, Phase 1 12) 2016: 65 trials (Observational 16, Not applicable 19, Phase 4 4, Phase 3 8, Phase 2 11, Phase 1/2 3, Phase 1 4) 2017: 75 trials (Observational 21, Not applicable 12, Phase 4 5, Phase 3 4, Phase 2/3 1, Phase 2 20, Phase 1/2 4, Phase 1 8) 2018: 97 trials (Observational 25, Not applicable 27, Phase 4 5, Phase 3 6, Phase 2/3 5, Phase 2 14, Phase 1/2 5, Phase 1 10) 2019: 73 trials (Observational 19, Not applicable 17, Phase 4 1, Phase 3 9, Phase 2/3 2, Phase 2 14, Phase 1/2 3, Phase 1 8) 2020: 85 trials (Observational 25, Not applicable 23, Phase 4 5, Phase 3 6, Phase 2/3 3, Phase 2 10, Phase 1/2 4, Phase 1 9) 2021: 86 trials (Observational 35, Not applicable 21, Phase 4 4, Phase 3 6, Phase 2 10, Phase 1/2 3, Phase 1 7) 2022: 92 trials (Observational 28, Not applicable 25, Phase 4 3, Phase 3 9, Phase 2/3 3, Phase 2 12, Phase 1/2 6, Phase 1 6) 2023: 85 trials (Observational 28, Not applicable 21, Phase 4 5, Phase 3 2, Phase 2/3 3, Phase 2 12, Phase 1/2 3, Phase 1 11) 2024: 97 trials (Observational 32, Not applicable 32, Phase 4 5, Phase 3 2, Phase 2 8, Phase 1/2 6, Phase 1 12) 2025: 66 trials (Observational 18, Not applicable 22, Phase 4 1, Phase 3 6, Phase 2/3 1, Phase 2 7, Phase 1/2 3, Phase 1 8)

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) 75
2025 66
2024 97
2023 85
2022 92
2021 86
2020 85
2019 73
2018 97
2017 75

Showing 10 of 35 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 91 trials starting in 2026 and about 79 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 143 trials still running at the 2026-10-09 snapshot; registered median of 28 months across 962 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Anemia, Hemolytic, Congenital trials in? (9)

Shown in this order: phase order.

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Trials by phase
Phase Trials
Phase 1 152
Phase 1/2 92
Phase 2 266
Phase 2/3 36
Phase 3 117
Phase 4 77
Not applicable 350
Observational 433
Expanded access 7

How many Anemia, Hemolytic, Congenital trials are recruiting? (13)

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 65
Recruiting 186
Enrolling by invitation 13
Active, not recruiting 76
Available 3

Showing 5 of 13 statuses.

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

Which lead sponsors run the most Anemia, Hemolytic, Congenital 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.

Lead sponsors
Sponsor Trials
National Heart, Lung, and Blood Institute (NHLBI) 64
Novartis Pharmaceuticals 43
Pfizer 27

Which drugs are being tested for Anemia, Hemolytic, Congenital? (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 28
Sirolimus 25
Voxelotor 21
Busulfan 18
PMI-150 (intranasal ketamine) 13

Interventions as named in the registry (10)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
HU - Hydroxyurea 74
SQ Fludarabine 35
Deferasirox FCT 32
Alemtuzumab s.c. 31
Total Body Irradiation 19

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, Hemolytic, Congenital 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
Sickle cell anaemia with crisis 1,374 43
infections and infestations 346 1
Acute chest syndrome 299 33
Pyrexia 262 45
Pneumonia 261 49

Showing 5 of 10 adverse events.

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Which publications cover Anemia, Hemolytic, Congenital? (10)

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

Publications
Publication Journal Year Major topic
Breath-holding spells occur disproportionately more often in children with transient erythroblastopenia. Acta paediatrica (Oslo, Norway : 1992) 2016 Yes
Novel Gardos channel mutations linked to dehydrated hereditary stomatocytosis (xerocytosis). American journal of hematology 2015 Yes
Structural and functional effects of hereditary hemolytic anemia-associated point mutations in the alpha spectrin tetramer site. Blood 2008 Yes
Disorders of red cell membrane. British journal of haematology 2008 Yes
Using the eosin-5-maleimide binding test in the differential diagnosis of hereditary spherocytosis and hereditary pyropoikilocytosis. Cytometry. Part B, Clinical cytometry 2008 Yes

Showing 5 of 10 publications.

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Sub-conditions in MeSH (8)

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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