RecruitingPhase 1 / Phase 2NCT07599176
Blood
Beta-Thalassemia
Also known as Cooley anemia, beta-thal, Mediterranean anemia
Beta-thalassemia is an inherited blood disorder caused by mutations in the HBB gene that reduce or eliminate production of the beta-globin chain of hemoglobin, leading to chronic hemolytic anemia. The severity ranges from thalassemia minor
ORPHA:848 ↗Gene HBBPrevalence 1 in 100,000 in Western countries; higher in Mediterranean, Middle East, and Southeast AsiaOnset Infancy to early childhoodAutosomal recessive
48
studies recruiting now
as of 7 Sept 2026
363
studies registered in total
as of 7 Sept 2026
1
countries with a recruiting site
as of 7 Sept 2026
20 May 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT04872179
International Registry of Patients With Alpha Thalassemia
RecruitingPhase 1NCT05904093
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
RecruitingNCT03937817
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
RecruitingPhase 3NCT07517133
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 48 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Registry: Thalassemia Patient Registry (TIF) · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Beta-Thalassemia
Beta-thalassemia is an inherited blood disorder caused by mutations in the HBB gene that reduce or eliminate production of the beta-globin chain of hemoglobin, leading to chronic hemolytic anemia. The severity ranges from thalassemia minor (trait, typically asymptomatic) to thalassemia intermedia and thalassemia major (Cooley anemia), which requires lifelong transfusions. Without treatment, thalassemia major causes progressive organ damage from iron overload and severe anemia.
Common clinical features
Severe chronic anemia requiring regular red blood cell transfusionsSplenomegaly and hepatomegaly from extramedullary hematopoiesisSkeletal deformities including frontal bossing and maxillary hyperplasiaGrowth retardation and delayed pubertyIron overload causing cardiac, liver, and endocrine complicationsJaundice and pallorFatigue and exercise intoleranceIncreased susceptibility to infections
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
5 approved treatments and 16 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Deferiprone (Deferiprone lipomed)Approved: Deferasirox (Deferasirox)Approved: Luspatercept (Reblozyl)Approved: Betibeglogene Autotemcel (Zynteglo)Approved: Exagamglogene Autotemcel (Casgevy)
Phase 3Mitapivat
Phase 3Sildenafil
Phase 3Thalidomide (Kevadon)
Phase 3Deferoxamine
Phase 2/3Metoprolol
Phase 2/3Hydroxyurea (Droxia)
Phase 2Arginine Butyrate
Phase 2Sodium 2,2-Dimethylbutyrate
+ 8 more in development
Before you apply
Things trial teams commonly ask about for Beta-Thalassemia. Not eligibility rules; those are set by each study.
- Transfusion-dependent patients (TDT) and non-transfusion-dependent patients (NTDT) are often enrolled in separate trial arms — clarify your transfusion history and frequency before screening.
- Many gene therapy and gene editing trials require patients to have no matched sibling donor and adequate organ function; obtain recent ferritin, liver iron concentration (LIC), and cardiac T2* MRI results.
- Prior splenectomy status and alloantibody burden from transfusions may affect eligibility — gather your full transfusion and surgical history.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).