Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Thalassemia: early-phase trial registry
5 of 21 indexed Thalassemia trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #797 of 28,707 conditions by registered trial count.
21 US clinical trials · 5 currently recruiting · #797 of 28,707 conditions by registered trial count
Thalassemia: 21 US clinical trials tracked, 5 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.
Enrollment posture brief
Registry condition thalassemia
OPEN 5 | RECR 24% | LATE 3 | EARLY 11 | RANK #797 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Dehydration (24% recruiting · 21 trials)
Thalassemia lists 5 open slots (24% of 21 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Thalassemia on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Thalassemia is recruiting below its size-band peers.
5 of 21 indexed trials (23.8%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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Sponsorship of Thalassemia trials is spread across many organizations.
UCSF Benioff Children's Hospital Oakland accounts for 2 of 21 trials (9.5%). 17 distinct sponsors appear on at least one study for this condition label.
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Most Thalassemia trials plan to enrol fewer than 100 people.
Median target enrolment is 26 among the 20 trials that report a genuine target (sentinel values excluded). 15 of those (75%) plan for fewer than 100 participants.
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Thalassemia research is mostly interventional.
15 interventional and 6 observational studies make up this index (71.4% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
21 total, page 1 of 1
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 Francisco
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 Francisco
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 |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 7 |
| Phase 3 | 3 |
Top Sponsors
Named interventions
Most-linked intervention names on Thalassemia records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Thalassemia trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Thalassemia page
21 registered trials, 5 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 5 Thalassemia trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- UCSF Benioff Children's Hospital Oakland sponsors the most Thalassemia trials on record, review their full trial history before evaluating a specific study. See UCSF Benioff Children's Hospital Oakland's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Thalassemia condition card totals 21 registered trials; most-linked intervention Cyclophosphamide; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.