Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Thalassemia
Open-data reference.
21 US clinical trials · 5 currently recruiting
Active & Recent Trials
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Novo Nordisk A/S
NCT06609226
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
National Heart, Lung, and Blood Institute (NHLBI)
NCT06313398
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Vertex Pharmaceuticals Incorporated
NCT05477563
A 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
NCT05736419
Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia
University of California, San Francis
NCT07064941
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
National Heart, Lung, and Blood Institute (NHLBI)
NCT02105766
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
Agios Pharmaceuticals
NCT03692052
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Vertex Pharmaceuticals Incorporated
NCT05356195
A Blood Stem Cell Transplant for Sickle Cell Disease
City of Hope Medical Center
NCT03249831
Multi-Center Study of Iron Overload: Survey Study (MCSIO)
UCSF Benioff Children's Hospital Oakland
NCT01913548
Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia
Children's Hospital Los Angeles
NCT03715972
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients
Westat
NCT05255445
A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
Forma Therapeutics
NCT04987489
Genes Influencing Iron Overload State
St. Jude Children's Research Hospital
NCT01158794
Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension
Carelon Research
NCT00872170
Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
University of California, San Francis
NCT03609827
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Duke University
NCT00744692
Stem Cell Transplant for Hemoglobinopathy
Masonic Cancer Center, University of Minnesota
NCT00176852
Multi-Center Study of Iron Overload: Pilot Study
UCSF Benioff Children's Hospital Oakland
NCT01114776
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
Dana-Farber Cancer Institute
NCT00153985
Combination Iron Chelation Therapy
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NCT00004982
Phase Distribution
| Phase | Trial count |
|---|---|
| Early Phase 1 | 4 |
| Phase 2 | 7 |
| Phase 3 | 3 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Thalassemia Trial Landscape
ClinicalTrials.gov lists 21 US studies indexed under Thalassemia, and 5 of those are currently open to recruitment — roughly 24% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Thalassemia shows 3 late-stage studies (Phase 3 and Phase 4 combined) alongside 11 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Thalassemia is led by UCSF Benioff Children's Hospital Oakland with 2 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 21 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Thalassemia?
PlainTrial tracks 21 US clinical trials for Thalassemia, of which 5 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Thalassemia?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.