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ClinicalTrials.gov 5 recruiting now official registry

Thalassemia clinical trials

5 of 21 indexed Thalassemia trials are currently recruiting.

21 US clinical trials · 5 currently recruiting

Thalassemia is the subject of 21 registered US clinical trials on ClinicalTrials.gov, 5 of them currently open to new participants. 3 are in Phase 3-4 (later-stage) and 11 in Phase 1-2 (earlier-stage). The most active sponsor is UCSF Benioff Children's Hospital Oakland, running 2 of these trials.

The research picture

Thalassemia has 21 registered US clinical trials, 5 of them open to new participants right now, about 24% of the total.

5
recruiting participants now
24%
of trials open to enrollment
3
in Phase 3–4 (later-stage)
2
top sponsor: UCSF Benioff Children's Hospital Oakland
Phase 14Phase 27Phase 33

Active & Recent Trials

21 total, page 1 of 1

RECRUITING Phase 3 480 participants

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Novo Nordisk A/S

NCT06609226

RECRUITING Early Phase 1 100 participants

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

National Heart, Lung, and Blood Institute (NHLBI)

NCT06313398

RECRUITING Phase 3 26 participants

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

RECRUITING Phase 2 24 participants

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

RECRUITING NA 20 participants

Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia

University of California, San Francisco

NCT07064941

ACTIVE NOT RECRUITING Phase 2 56 participants

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

ACTIVE NOT RECRUITING Phase 2 20 participants

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

Agios Pharmaceuticals

NCT03692052

ACTIVE NOT RECRUITING Phase 3 16 participants

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Vertex Pharmaceuticals Incorporated

NCT05356195

ACTIVE NOT RECRUITING Phase 1 3 participants

A Blood Stem Cell Transplant for Sickle Cell Disease

City of Hope Medical Center

NCT03249831

COMPLETED 423 participants

Multi-Center Study of Iron Overload: Survey Study (MCSIO)

UCSF Benioff Children's Hospital Oakland

NCT01913548

COMPLETED 165 participants

Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia

Children's Hospital Los Angeles

NCT03715972

COMPLETED 157 participants

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

Westat

NCT05255445

COMPLETED Phase 2 53 participants

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

Forma Therapeutics

NCT04987489

COMPLETED 50 participants

Genes Influencing Iron Overload State

St. Jude Children's Research Hospital

NCT01158794

COMPLETED Phase 2 27 participants

Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension

Carelon Research

NCT00872170

COMPLETED 25 participants

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

University of California, San Francisco

NCT03609827

COMPLETED Phase 1 22 participants

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Duke University

NCT00744692

COMPLETED Phase 2 22 participants

Stem Cell Transplant for Hemoglobinopathy

Masonic Cancer Center, University of Minnesota

NCT00176852

COMPLETED 20 participants

Multi-Center Study of Iron Overload: Pilot Study

UCSF Benioff Children's Hospital Oakland

NCT01114776

COMPLETED Phase 2 2 participants

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

Dana-Farber Cancer Institute

NCT00153985

COMPLETED Phase 1

Combination Iron Chelation Therapy

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NCT00004982

Phase Distribution

PhaseTrial count
Phase 1 4
Phase 2 7
Phase 3 3

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Thalassemia Trial Landscape

21 indexed Thalassemia studies · 5 recruiting (24%).

Phase mix: 3 late (III/IV) vs 11 early (I/II).

Top sponsor: UCSF Benioff Children's Hospital Oakland (2 trials), 9 others listed. 21 shown below, recruiting first.

How to read this condition's trial landscape

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.

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.

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.

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.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.