Thalassemia clinical trials

The registry lists 250 studies for Thalassemia across more than 10 countries. 43 are recruiting or about to start. 117 of them are mainly about Thalassemia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10. Counting its narrower condition beta-Thalassemia too: 497 studies, 101 open.

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Status

Completed: 143
Recruiting: 28
Terminated: 21
Status unknown: 21
Active, not recruiting: 15
Not yet recruiting: 12

Phase

Phase 2: 80
Phase 1: 38
Phase 3: 23
Phase 4: 19
Early Phase 1: 6

Where

United States: 104
Italy: 35
China: 32
United Kingdom: 27
Thailand: 24
Greece: 23

Who runs them

Novartis Pharmaceuticals (20) · HaEmek Medical Center, Israel (11) · Carelon Research (7) · National Heart, Lung, and Blood Institute (NHLBI) (7) · Assiut University (7) · UCSF Benioff Children's Hospital Oakland (6) · Agios Pharmaceuticals, Inc. (6) · University of California, San Francisco (6)

What is being tested here

Drugs under test in 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
Thalidomide	2	1	8
Exagamglogene autotemcel	2	4	4
Dexamethasone	2	0	2
Cyclophosphamide	1	1	7
Hydroxyurea	1	1	5
Fludarabine	1	1	5
Sirolimus	1	0	2
Mitapivat	1	0	2
Abatacept	1	0	2
Tacrolimus	1	0	2

Drug classes tested here Corticosteroids 3 open · Antimetabolites 2 open · Alkylating agents 1 open · Iron chelators 24 · Calcium channel blockers 3

Coming up

9phase 3 studies under way

18studies 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
Dec 2026A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia · Kind Pharmaceuticals LLC
Dec 2026A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major · Shanghai BDgene Co., Ltd.
Dec 2026Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia · University of California, San Francisco
Dec 2026the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia · Shenzhen Hemogen
Jan 2027A Blood Stem Cell Transplant for Sickle Cell Disease · City of Hope Medical Center
Feb 2027A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) · Memorial Sloan Kettering Cancer Center
Feb 2027Low-Dose Liposomal Amphotericin B for Invasive Fungal Infection Prophylaxis in Neutropenic Children · Haikou Affiliated Hospital of Central South University Xiangya School of Medicine

Who these studies take

Of the 28 Thalassemia studies recruiting now, 7 (25%) set no upper age limit and 18 (64%) stop at 75 or younger. 22 (79%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.

Recruiting studies that take each age, of 28
Age	Women	Men
10	20 (71%)	20 (71%)
18	23 (82%)	23 (82%)
30	20 (71%)	20 (71%)
40	13 (46%)	13 (46%)
50	13 (46%)	13 (46%)
65	12 (43%)	12 (43%)
75	11 (39%)	11 (39%)
85	10 (36%)	10 (36%)

Across all 250 studies, recruiting or not: 55% stop at 75 or younger and 52% at 65; 70% take children; 3% are for women only and <1% for men only; 16% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Thalassemia studies take

Of 25 phase 2 interventional studies under Thalassemia, half reached primary completion within 30 months of starting, counting those still running.

Start to primary completion	Every sponsor	Industry	Non-industry
Phase 2	30 mo19–67 · 25 studies	—	—
Interventional, no phase	17 mo3–87 · 18 studies	—	—
Observational	53 mo29–74 · 23 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.

Newly registered2

Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-ThalassemiaOct 21, 2026NCT07884773
Native T1 Mapping and ECV in Chronic Hemolytic AnemiaOct 20, 2026NCT07882303

Studies

Recruiting first, then most recently updated. Showing 50 of 250. Browse all 250 → or search them.