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Condition (MeSH)

beta-Thalassemia

ClinicalTrials.gov lists 263 trials for beta-Thalassemia or a narrower condition beneath it.

MeSH D017086 Hemic and Lymphatic Diseases C15 C16 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 22 Phase 1/2 Phase 1/2 18 Phase 2 Phase 2 54 Phase 2/3 Phase 2/3 15 Phase 3 Phase 3 16 Phase 4 Phase 4 17 Not applicable Not applicable 51 Observational Observational 69 Phase 1 Phase 1 22 Phase 1/2 Phase 1/2 18 Phase 2 Phase 2 54 Phase 2/3 Phase 2/3 15 Phase 3 Phase 3 16 Phase 4 Phase 4 17 Not applicable Not applicable 51 Observational Observational 69

Trials per phase

1 more section with rows

263 of the 263 trials are tagged with beta-Thalassemia 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 beta-Thalassemia trials start? (31)

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. 1 trial is 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: 25

1995: 1 trials (Not applicable 1) 1997: 1 trials (Phase 2 1) 1998: 1 trials (Observational 1) 1999: 3 trials (Phase 2 3) 2000: 5 trials (Observational 1, Not applicable 1, Phase 4 1, Phase 2 1, Phase 1 1) 2001: 1 trials (Phase 2 1) 2002: 1 trials (Phase 4 1) 2003: 4 trials (Phase 3 1, Phase 2/3 1, Phase 2 1, Phase 1/2 1) 2004: 7 trials (Observational 2, Phase 4 1, Phase 3 1, Phase 2 2, Phase 1/2 1) 2005: 3 trials (Observational 1, Phase 4 1, Phase 2 1) 2006: 2 trials (Not applicable 1, Phase 2 1) 2007: 4 trials (Observational 1, Not applicable 1, Phase 4 1, Phase 1 1) 2008: 6 trials (Not applicable 1, Phase 4 1, Phase 2/3 1, Phase 2 2, Phase 1/2 1) 2009: 7 trials (Observational 1, Phase 2/3 2, Phase 2 1, Phase 1/2 2, Phase 1 1) 2010: 5 trials (Observational 1, Not applicable 2, Phase 2 1, Phase 1 1) 2011: 5 trials (Observational 1, Phase 4 1, Phase 2 2, Phase 1/2 1) 2012: 13 trials (Observational 1, Not applicable 3, Phase 3 1, Phase 2/3 3, Phase 2 4, Phase 1 1) 2013: 9 trials (Observational 4, Not applicable 2, Phase 4 1, Phase 2 1, Phase 1/2 1) 2014: 14 trials (Observational 1, Not applicable 4, Phase 3 1, Phase 2/3 2, Phase 2 4, Phase 1 2) 2015: 9 trials (Not applicable 7, Phase 4 1, Phase 1/2 1) 2016: 6 trials (Observational 1, Not applicable 2, Phase 4 1, Phase 3 2) 2017: 13 trials (Observational 1, Not applicable 2, Phase 4 2, Phase 3 2, Phase 2 3, Phase 1/2 1, Phase 1 2) 2018: 12 trials (Observational 3, Phase 4 1, Phase 3 2, Phase 2/3 2, Phase 2 1, Phase 1/2 2, Phase 1 1) 2019: 16 trials (Observational 5, Not applicable 2, Phase 4 1, Phase 3 2, Phase 2/3 1, Phase 2 4, Phase 1 1) 2020: 17 trials (Observational 7, Not applicable 2, Phase 2/3 3, Phase 2 3, Phase 1/2 1, Phase 1 1) 2021: 15 trials (Observational 7, Not applicable 2, Phase 3 1, Phase 2 4, Phase 1 1) 2022: 16 trials (Observational 5, Not applicable 1, Phase 4 1, Phase 3 2, Phase 2 4, Phase 1/2 3) 2023: 19 trials (Observational 8, Not applicable 5, Phase 4 1, Phase 2 2, Phase 1 3) 2024: 25 trials (Observational 10, Not applicable 7, Phase 4 1, Phase 2 2, Phase 1/2 1, Phase 1 4) 2025: 10 trials (Observational 4, Not applicable 2, Phase 3 1, Phase 1/2 1, Phase 1 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.

Trials started per year
Year Trials started
2026 (partial year) 8
2025 10
2024 25
2023 19
2022 16
2021 15
2020 17
2019 16
2018 12
2017 13

Showing 10 of 31 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 9 trials starting in 2026 and about 11 in 2027, the average of the 24 months through September, counted as registered and carried forward. 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 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. 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 30 trials still running at the 2026-10-09 snapshot; registered median of 26 months across 135 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are beta-Thalassemia trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 22
Phase 1/2 18
Phase 2 54
Phase 2/3 15
Phase 3 16
Phase 4 17
Not applicable 51
Observational 69
Expanded access 1

How many beta-Thalassemia trials are recruiting? (10)

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 11
Recruiting 30
Enrolling by invitation 7
Active, not recruiting 18
Completed 124

Showing 5 of 10 statuses.

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

Which lead sponsors run the most beta-Thalassemia 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.

Lead sponsors
Sponsor Trials
Celgene 8
Novartis Pharmaceuticals 8
CorrectSequence Therapeutics Co., Ltd 5
National Heart, Lung, and Blood Institute (NHLBI) 5
Bioray Laboratories 4

Showing 5 of 8 sponsors.

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Which drugs are being tested for beta-Thalassemia? (4)

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
Busulfan 8
Cytoxan 7
SPD602 4
RITUXIMAB 4

Interventions as named in the registry (11)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Deferasirox FCT 15
Luspatercept Injection [Reblozyl] 12
SQ Fludarabine 10
HU - Hydroxyurea 9
Deferoxamine mesylate only 9

Showing 5 of 11 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 beta-Thalassemia 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
Pyrexia 21 8
Abdominal pain 16 6
Anaemia 13 4
Gastroenteritis 13 4
Pneumonia 12 9

Showing 5 of 10 adverse events.

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Which publications cover beta-Thalassemia? (278)

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

Publications
Publication Journal Year Major topic
Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel. Blood advances 2025 Yes
Impaired physical ability in patients with transfusion-dependent β-thalassaemia: Can regular physical activity be a countermeasure? British journal of haematology 2025 Yes
Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trial. The Lancet. Haematology 2025 Yes
Oxidative stress markers and tissue iron overload after 12-months vitamin E supplementation for children with transfusion-dependent β-thalassemia on different iron chelators: A randomized placebo-controlled trial. Clinical nutrition (Edinburgh, Scotland) 2025 Yes
Thalidomide confers therapeutic benefit in beta thalassemia patients by enhancing hemoglobin and hematopoietic gene expression: A non-randomized clinical trial. Blood cells, molecules & diseases 2025 Yes

Showing 5 of 278 publications.

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Prevalence (12)

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
Beta-thalassemia Annual incidence 1-5 / 10 000 Europe Not stated Unspecified
Beta-thalassemia Annual incidence 1-9 / 100 000 Worldwide Not stated Exact
Beta-thalassemia Annual incidence 1-5 / 10 000 Europe Not stated Exact
Beta-thalassemia Annual incidence 1-5 / 10 000 Europe Not stated Exact
Beta-thalassemia Annual incidence 1-9 / 100 000 Worldwide Not stated Unspecified

Showing 5 of 12 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
Beta-thalassemia 848 Exact Orphadata Science knowledge files, Product 1
Beta-thalassemia 848 Exact Orphanet Rare Disease Ontology (ORDO)

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: sub-conditions in mesh.

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