beta-Thalassemia clinical trials

The registry lists 294 studies for beta-Thalassemia across more than 10 countries. 69 are recruiting or about to start. 217 of them are mainly about beta-Thalassemia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.

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Status

Completed: 130
Recruiting: 43
Status unknown: 43
Active, not recruiting: 25
Not yet recruiting: 20
Terminated: 19

Phase

Phase 2: 94
Phase 1: 38
Phase 3: 38
Phase 4: 19
Early Phase 1: 7

Where

United States: 91
Italy: 49
China: 44
Egypt: 34
Greece: 26
Thailand: 24

Who runs them

Assiut University (18) · Tanta University (11) · Ain Shams University (10) · Novartis Pharmaceuticals (9) · Celgene (8) · CorrectSequence Therapeutics Co., Ltd (6) · Institute of Hematology & Blood Diseases Hospital, China (6) · Bioray Laboratories (6)

What is being tested here

Drugs under test in beta-Thalassemia studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

Drug	Open	Phase 3	All
Fludarabine	4	0	8
Alemtuzumab	3	0	5
Hydroxyurea	2	4	11
Luspatercept	2	2	8
Exagamglogene autotemcel	2	4	4
Cyclophosphamide	2	0	4
Thymoglobulin	2	0	4
Thiotepa	2	0	3
Rituximab	2	0	3
Melphalan	2	0	2

Drug classes tested here Antimetabolites 4 open · Alkylating agents 4 open · Corticosteroids 2 open · Calcineurin inhibitors 1 open · HDAC inhibitors 1 open

Coming up

10phase 3 studies under way

23studies finish main data collection within a year3 of them phase 3

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

The 10 nearest, phase 3 first

Apr 2027Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia · Phase 3 · Khyber Medical University Peshawar
Jun 2027Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease · Phase 3 · Vertex Pharmaceuticals Incorporated
Nov 2027Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) · Phase 3 · Vertex Pharmaceuticals Incorporated
Nov 2026Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT · Paul Szabolcs
Nov 2026Early Check: Expanded Screening in Newborns · RTI International
Dec 2026Pumilio1 (PUM1) Expression, Sickle Cell Anemia, β-thalassemia Intermedia · Assiut University
Dec 2026A Long-term Follow-up Study in Participants Who Received CS-101 · Children's Hospital of Fudan University
Dec 2026A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body · Regeneron Pharmaceuticals
Dec 2026AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion · Mitchell Cairo
Dec 2026Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease · Abdullah Kutlar

Who these studies take

Of the 43 beta-Thalassemia studies recruiting now, 10 (23%) set no upper age limit and 31 (72%) stop at 75 or younger. 34 (79%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 6 (14%) take healthy volunteers.

Recruiting studies that take each age, of 43
Age	Women	Men
10	31 (72%)	31 (72%)
18	32 (74%)	32 (74%)
30	31 (72%)	31 (72%)
40	22 (51%)	22 (51%)
50	20 (47%)	20 (47%)
65	16 (37%)	16 (37%)
75	12 (28%)	12 (28%)
85	12 (28%)	12 (28%)

Across all 294 studies, recruiting or not: 61% stop at 75 or younger and 58% at 65; 70% take children; 0% are for women only and 1% for men only; 12% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long beta-Thalassemia studies take

Of 29 phase 2 interventional studies under beta-Thalassemia, half reached primary completion within 32 months of starting, counting those still running.

Start to primary completion	Every sponsor	Industry	Non-industry
Phase 2	32 mo21–57 · 29 studies	—	—
Phase 3	60 mo22–67 · 18 studies	—	—
Interventional, no phase	35 mo14–69 · 29 studies	—	—
Observational	72 mo24–156 · 29 studies	—	—

Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out

What changed recently

From the registry's own updates since Oct 13, 2026.

Stopped3

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem CellsTerminatedNov 4, 2026NCT06041620
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia PatientsTerminatedNov 4, 2026NCT06219239
Open-Label Extension Study to Pioneer Study 6058-SCD-101TerminatedOct 28, 2026NCT07401823

Newly registered1

Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-ThalassemiaOct 21, 2026NCT07884773

Studies

Recruiting first, then most recently updated. Showing 50 of 294. Browse all 294 → or search them.