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Condition (MeSH)

Anemia, Sickle Cell

ClinicalTrials.gov lists 1,156 trials for Anemia, Sickle Cell or a narrower condition beneath it. National Heart, Lung, and Blood Institute (NHLBI) has the most among industry and government lead sponsors, with 62.

MeSH D000755 Hemic and Lymphatic Diseases C15 C16 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 120 Phase 1/2 Phase 1/2 78 Phase 2 Phase 2 201 Phase 2/3 Phase 2/3 17 Phase 3 Phase 3 96 Phase 4 Phase 4 42 Not applicable Not applicable 275 Observational Observational 321 Phase 1 Phase 1 120 Phase 1/2 Phase 1/2 78 Phase 2 Phase 2 201 Phase 2/3 Phase 2/3 17 Phase 3 Phase 3 96 Phase 4 Phase 4 42 Not applicable Not applicable 275 Observational Observational 321

Trials per phase

1 more section with rows

1,115 of the 1,156 trials are tagged with Anemia, Sickle Cell 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, Sickle Cell 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. 3 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: 75

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: 2 trials (Observational 1, Not applicable 1) 1997: 3 trials (Not applicable 1, Phase 3 1, Phase 2 1) 1998: 1 trials (Phase 2 1) 1999: 5 trials (Not applicable 1, Phase 2 2, Phase 1/2 2) 2000: 8 trials (Not applicable 1, Phase 3 3, Phase 2 3, Phase 1 1) 2001: 8 trials (Observational 2, Phase 2 5, Phase 1 1) 2002: 7 trials (Observational 2, Not applicable 2, Phase 2/3 1, Phase 2 2) 2003: 5 trials (Not applicable 1, Phase 2 2, Phase 1 2) 2004: 18 trials (Observational 3, Phase 4 1, Phase 3 1, Phase 2 9, Phase 1/2 1, Phase 1 3) 2005: 22 trials (Observational 7, Not applicable 2, Phase 4 1, Phase 3 3, Phase 2 4, Phase 1/2 2, Phase 1 3) 2006: 28 trials (Observational 7, Phase 4 1, Phase 3 10, Phase 2 5, Phase 1/2 2, Phase 1 3) 2007: 20 trials (Observational 8, Not applicable 2, Phase 3 2, Phase 2 5, Phase 1/2 2, Phase 1 1) 2008: 30 trials (Observational 13, Not applicable 5, Phase 4 1, Phase 3 1, Phase 2/3 1, Phase 2 4, Phase 1/2 4, Phase 1 1) 2009: 34 trials (Observational 13, Not applicable 5, Phase 4 1, Phase 3 1, Phase 2 7, Phase 1/2 5, Phase 1 2) 2010: 36 trials (Observational 10, Not applicable 7, Phase 4 1, Phase 3 4, Phase 2/3 1, Phase 2 7, Phase 1 6) 2011: 28 trials (Observational 7, Not applicable 2, Phase 4 2, Phase 3 3, Phase 2 7, Phase 1/2 4, Phase 1 3) 2012: 48 trials (Observational 19, Not applicable 11, Phase 4 1, Phase 3 1, Phase 2/3 2, Phase 2 6, Phase 1/2 3, Phase 1 5) 2013: 43 trials (Observational 10, Not applicable 9, Phase 3 3, Phase 2 10, Phase 1/2 5, Phase 1 6) 2014: 55 trials (Observational 15, Not applicable 12, Phase 4 2, Phase 3 1, Phase 2 12, Phase 1/2 7, Phase 1 6) 2015: 53 trials (Observational 12, Not applicable 12, Phase 4 5, Phase 3 5, Phase 2 7, Phase 1/2 2, Phase 1 10) 2016: 53 trials (Observational 13, Not applicable 16, Phase 4 2, Phase 3 5, Phase 2 10, Phase 1/2 3, Phase 1 4) 2017: 53 trials (Observational 16, Not applicable 11, Phase 4 1, Phase 3 2, Phase 2/3 1, Phase 2 14, Phase 1/2 3, Phase 1 5) 2018: 75 trials (Observational 20, Not applicable 25, Phase 4 1, Phase 3 4, Phase 2/3 2, Phase 2 12, Phase 1/2 4, Phase 1 7) 2019: 51 trials (Observational 13, Not applicable 12, Phase 3 7, Phase 2/3 1, Phase 2 9, Phase 1/2 3, Phase 1 6) 2020: 69 trials (Observational 19, Not applicable 21, Phase 4 4, Phase 3 6, Phase 2/3 1, Phase 2 6, Phase 1/2 4, Phase 1 8) 2021: 71 trials (Observational 26, Not applicable 19, Phase 4 4, Phase 3 6, Phase 2 7, Phase 1/2 3, Phase 1 6) 2022: 72 trials (Observational 21, Not applicable 21, Phase 4 2, Phase 3 8, Phase 2/3 3, Phase 2 8, Phase 1/2 3, Phase 1 6) 2023: 55 trials (Observational 17, Not applicable 12, Phase 4 4, Phase 3 2, Phase 2/3 2, Phase 2 10, Phase 1/2 3, Phase 1 5) 2024: 63 trials (Observational 19, Not applicable 22, Phase 4 3, Phase 3 2, Phase 2 6, Phase 1/2 5, Phase 1 6) 2025: 56 trials (Observational 14, Not applicable 19, Phase 4 1, Phase 3 5, Phase 2/3 1, Phase 2 7, Phase 1/2 2, Phase 1 7)

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) 60
2025 56
2024 63
2023 55
2022 72
2021 71
2020 69
2019 51
2018 75
2017 53

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 73 trials starting in 2026 and about 62 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 116 trials still running at the 2026-10-09 snapshot; registered median of 30 months across 749 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Anemia, Sickle Cell trials in? (9)

Shown in this order: phase order.

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Trials by phase
Phase Trials
Phase 1 120
Phase 1/2 78
Phase 2 201
Phase 2/3 17
Phase 3 96
Phase 4 42
Not applicable 275
Observational 321
Expanded access 6

How many Anemia, Sickle Cell trials are recruiting? (12)

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 47
Recruiting 157
Enrolling by invitation 10
Active, not recruiting 57
Available 3

Showing 5 of 12 statuses.

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

Which lead sponsors run the most Anemia, Sickle Cell 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) 62
Pfizer 27
Novartis Pharmaceuticals 22

Which drugs are being tested for Anemia, Sickle Cell? (7)

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 24
Sirolimus 22
Voxelotor 21
Busulfan 14
PMI-150 (intranasal ketamine) 13

Showing 5 of 7 drugs.

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Interventions as named in the registry (8)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
HU - Hydroxyurea 67
SQ Fludarabine 31
Alemtuzumab s.c. 30
Total Body Irradiation 19
Mofetil Mycophenolate 14

Showing 5 of 8 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, Sickle Cell 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
Pneumonia 248 39
Pyrexia 232 33

Showing 5 of 10 adverse events.

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Which publications cover Anemia, Sickle Cell? (1,359)

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

Publications
Publication Journal Year Major topic
Hydroxyurea pharmacokinetics in children with sickle cell anemia across different global populations. Blood advances 2026 Yes
Safety, Pharmacokinetics, and Pharmacodynamics of Osivelotor for Sickle Cell Disease: First-in-Human Studies in Healthy Participants and Patients. Clinical pharmacology and therapeutics 2026 Yes
Hospital Variations in Time-To-Crisis-Resolution Among Children and Adolescents With Sickle Cell Disease. American journal of hematology 2026 Yes
Validation and Application of a Predictive Score of Acute Chest Syndrome. NEJM evidence 2026 Yes
Management of Kidney Disease with Sickle Cell Disease. Journal of the American Society of Nephrology : JASN 2025 Yes

Showing 5 of 1,359 publications.

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Prevalence (36)

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.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Sickle cell anemia Prevalence at birth 1-5 / 10 000 United Kingdom GB Exact
Sickle cell anemia Prevalence at birth 1-5 / 10 000 United Kingdom GB Exact
Sickle cell anemia Prevalence at birth 1-5 / 10 000 United Kingdom GB Unspecified
Sickle cell anemia Point prevalence 1-5 / 10 000 United States US Exact
Sickle cell anemia Prevalence at birth 1-5 / 10 000 United States US Exact

Showing 5 of 36 prevalence observations.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Sickle cell anemia 232 Exact Orphanet Rare Disease Ontology (ORDO)
Sickle cell anemia 232 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (4)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

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