Condition (MeSH)
alpha-Thalassemia
ClinicalTrials.gov lists 24 trials for alpha-Thalassemia or a narrower condition beneath it.
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Trials per phase
16 of the 24 trials are tagged with alpha-Thalassemia itself; the rest are tagged with a narrower condition beneath it.
When did alpha-Thalassemia trials start? (13)
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.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Not applicable
- Observational
Top of scale: 5
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.
| Year | Trials started |
|---|---|
| 2024 | 2 |
| 2023 | 1 |
| 2022 | 5 |
| 2021 | 3 |
| 2020 | 1 |
| 2018 | 2 |
| 2017 | 4 |
| 2016 | 1 |
| 2011 | 1 |
| 2009 | 1 |
Showing 10 of 13 earlier years.
No forecast is made for this condition: it averages 2.0 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 2 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are alpha-Thalassemia trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 1 |
| Phase 2 | 3 |
| Not applicable | 5 |
| Observational | 13 |
How many alpha-Thalassemia trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 8 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 3 |
| Completed | 8 |
| Terminated | 2 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most alpha-Thalassemia trials? (5)
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.
| Sponsor | Trials |
|---|---|
| Bristol-Myers Squibb | 1 |
| Forma Therapeutics, Inc. | 1 |
| National Institute of Allergy and Infectious Diseases (NIAID) | 1 |
| Novartis Pharmaceuticals | 1 |
| Obstetrix Medical Group | 1 |
Which drugs are being tested for alpha-Thalassemia? (1)
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.
| Drug | Trials |
|---|---|
| Etavopivat | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Luspatercept Injection [Reblozyl] | 1 |
| in utero hematopoietic stem cell transplantation | 1 |
| Enriched Hematopoetic Stem Cell Infusion | 1 |
| Deferasirox FCT | 1 |
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 alpha-Thalassemia trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Gastroenteritis | 9 | 1 |
| Anaemia | 6 | 1 |
| Pyrexia | 5 | 1 |
| Abdominal pain | 2 | 1 |
| Cholangitis | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover alpha-Thalassemia? (41)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial. | Lancet (London, England) | 2025 | Yes |
| The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry. | Blood advances | 2023 | Yes |
| Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study. | Lancet (London, England) | 2022 | Yes |
| Hemoglobins F, A<sub>2</sub> , and E levels in Laotian children aged 6-23 months with Hb E disorders: Effect of age, sex, and thalassemia types. | International journal of laboratory hematology | 2020 | Yes |
| Sickle Cell and α+-Thalassemia Traits Influence the Association between Ferritin and Hepcidin in Rural Kenyan Children Aged 14-26 Months. | The Journal of nutrition | 2018 | Yes |
Showing 5 of 41 publications.
Prevalence (9)
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.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Alpha-thalassemia | Prevalence at birth | 1-5 / 10 000 | United States | US | Exact |
| Alpha-thalassemia | Prevalence at birth | 1-5 / 10 000 | United States | US | Exact |
| Alpha-thalassemia | Prevalence at birth | 1-5 / 10 000 | United States | US | Unspecified |
| Alpha-thalassemia | Point prevalence | Unknown | Europe | Not stated | Exact |
| Alpha-thalassemia | Prevalence at birth | Unknown | Europe | Not stated | Unspecified |
Showing 5 of 9 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Alpha-thalassemia | 846 | Exact | Orphadata Science knowledge files, Product 1 |
| Alpha-thalassemia | 846 | Exact | Orphanet Rare Disease Ontology (ORDO) |
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.