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Condition (MeSH)

Hemoglobinopathies

ClinicalTrials.gov lists 1,513 trials for Hemoglobinopathies 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 D006453 Hemic and Lymphatic Diseases C15 C16 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 148 Phase 1/2 Phase 1/2 94 Phase 2 Phase 2 267 Phase 2/3 Phase 2/3 34 Phase 3 Phase 3 114 Phase 4 Phase 4 73 Not applicable Not applicable 349 Observational Observational 426 Phase 1 Phase 1 148 Phase 1/2 Phase 1/2 94 Phase 2 Phase 2 267 Phase 2/3 Phase 2/3 34 Phase 3 Phase 3 114 Phase 4 Phase 4 73 Not applicable Not applicable 349 Observational Observational 426

Trials per phase

1 more section with rows

98 of the 1,513 trials are tagged with Hemoglobinopathies 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 Hemoglobinopathies 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: 96

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: 7 trials (Not applicable 1, Phase 2 4, 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: 9 trials (Observational 2, Phase 2 5, Phase 1/2 1, 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: 12 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 2/3 1, Phase 2 5, Phase 1/2 1, Phase 1 2) 2004: 26 trials (Observational 6, Not applicable 1, Phase 4 3, Phase 3 2, Phase 2 10, Phase 1/2 1, Phase 1 3) 2005: 30 trials (Observational 10, Not applicable 2, Phase 4 2, Phase 3 4, Phase 2 6, Phase 1/2 3, 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: 31 trials (Observational 11, Not applicable 4, Phase 4 3, Phase 3 2, Phase 2 8, Phase 1/2 2, Phase 1 1) 2008: 39 trials (Observational 14, Not applicable 6, Phase 4 1, Phase 3 1, Phase 2/3 2, Phase 2 7, Phase 1/2 5, Phase 1 3) 2009: 47 trials (Observational 16, Not applicable 6, Phase 4 3, Phase 3 1, Phase 2/3 3, Phase 2 9, Phase 1/2 7, Phase 1 2) 2010: 42 trials (Observational 12, Not applicable 10, Phase 4 1, Phase 3 4, Phase 2/3 1, Phase 2 8, Phase 1 6) 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 20, Not applicable 15, Phase 4 1, Phase 3 3, Phase 2/3 4, Phase 2 10, Phase 1/2 4, Phase 1 6) 2013: 56 trials (Observational 15, Not applicable 12, Phase 4 4, Phase 3 3, Phase 2 11, Phase 1/2 5, Phase 1 6) 2014: 70 trials (Observational 16, Not applicable 15, Phase 4 3, Phase 3 2, Phase 2/3 3, Phase 2 17, Phase 1/2 7, Phase 1 7) 2015: 70 trials (Observational 14, Not applicable 21, Phase 4 7, Phase 3 5, Phase 2 8, Phase 1/2 3, Phase 1 12) 2016: 64 trials (Observational 15, Not applicable 19, Phase 4 4, Phase 3 8, Phase 2 11, Phase 1/2 3, Phase 1 4) 2017: 75 trials (Observational 22, Not applicable 12, Phase 4 5, Phase 3 4, Phase 2/3 1, Phase 2 19, Phase 1/2 4, Phase 1 8) 2018: 94 trials (Observational 24, Not applicable 28, Phase 4 4, Phase 3 6, Phase 2/3 5, Phase 2 13, Phase 1/2 5, Phase 1 9) 2019: 69 trials (Observational 18, Not applicable 14, Phase 4 1, Phase 3 9, Phase 2/3 2, Phase 2 14, Phase 1/2 3, Phase 1 8) 2020: 86 trials (Observational 27, Not applicable 22, Phase 4 4, Phase 3 6, Phase 2/3 3, Phase 2 12, Phase 1/2 4, Phase 1 8) 2021: 85 trials (Observational 31, Not applicable 24, Phase 4 4, Phase 3 6, Phase 2 10, Phase 1/2 3, Phase 1 7) 2022: 92 trials (Observational 27, Not applicable 26, Phase 4 3, Phase 3 9, Phase 2/3 3, Phase 2 12, Phase 1/2 6, Phase 1 6) 2023: 81 trials (Observational 28, Not applicable 19, Phase 4 5, Phase 3 2, Phase 2/3 3, Phase 2 11, Phase 1/2 3, Phase 1 10) 2024: 96 trials (Observational 32, Not applicable 32, Phase 4 4, Phase 3 2, Phase 2 8, Phase 1/2 6, Phase 1 12) 2025: 68 trials (Observational 19, Not applicable 21, Phase 4 1, Phase 3 6, Phase 2/3 1, Phase 2 7, Phase 1/2 4, Phase 1 9)

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 68
2024 96
2023 81
2022 92
2021 85
2020 86
2019 69
2018 94
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 92 trials starting in 2026 and about 80 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 144 trials still running at the 2026-10-09 snapshot; registered median of 28 months across 943 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Hemoglobinopathies trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 148
Phase 1/2 94
Phase 2 267
Phase 2/3 34
Phase 3 114
Phase 4 73
Not applicable 349
Observational 426
Expanded access 8

How many Hemoglobinopathies 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 194
Enrolling by invitation 14
Active, not recruiting 77
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 Hemoglobinopathies 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 Hemoglobinopathies? (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 21
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 37
Deferasirox FCT 33
Alemtuzumab s.c. 31
Total Body Irradiation 20

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 Hemoglobinopathies 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
Adverse side effects (GI distress, constipation, vomiting, diarrhea) 428 1
infections and infestations 346 1
Acute chest syndrome 299 33
Pneumonia 265 50

Showing 5 of 10 adverse events.

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Which publications cover Hemoglobinopathies? (56)

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

Publications
Publication Journal Year Major topic
A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis. Blood 2023 Yes
Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease. European journal of pediatrics 2023 Yes
Genome editing approaches to β-hemoglobinopathies. Progress in molecular biology and translational science 2021 Yes
A comprehensive review of hydroxyurea for β-haemoglobinopathies: the role revisited during COVID-19 pandemic. Orphanet journal of rare diseases 2021 Yes
Pregnancy outcomes in women with a hemoglobinopathy trait: a multicenter, retrospective study. Archives of gynecology and obstetrics 2021 Yes

Showing 5 of 56 publications.

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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
Hemoglobinopathy 68364 Exact Orphanet Rare Disease Ontology (ORDO)
Hemoglobinopathy 68364 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (3)

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.

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