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All 51 Thalassemia trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic DisordersMost sites in California, Ohio and FloridaRecruitingAmerican Thrombosis and Hemostasis Network76US sites
31 states - A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or ThalassaemiaMost sites in California, New York and North CarolinaRecruitingPhase 3Ages 2+Novo Nordisk A/S11US sites
7 states - 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)Most sites in New Jersey and New YorkRecruitingPhase 2Ages 2-50Memorial Sloan Kettering Cancer Center6US sites
2 states - A Study of Participants with Β-Thalassemia Treated with Betibeglogene AutotemcelMost sites in California, Minnesota and New YorkRecruitingGenetix Biotherapeutics6US sites
5 states - A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent ThalassemiaMost sites in California, Georgia and New YorkRecruitingPhase 3Ages 1-17Agios Pharmaceuticals5US sites
5 states - A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)Most sites in Georgia, New York and PennsylvaniaRecruitingPhase 3Ages 1-17Agios Pharmaceuticals4US sites
4 states - Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell DiseaseMost sites in New York, North Carolina and TennesseeRecruitingPhase 3Ages 12-35Vertex Pharmaceuticals Incorporated3US sites
3 states - AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell DepletionOnly in New YorkRecruitingPhase 2Ages 1 Day-30Mitchell Cairo1US site
- Collection of Human Biospecimens for Basic and Clinical Research Into Globin VariantsOnly in MarylandRecruitingAges 18-70National Institute of Allergy and Infectious Diseases (NIAID)1US site
- Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin LabelingOnly in MarylandRecruitingEarly Phase 1Ages 18-100National Heart, Lung, and Blood Institute (NHLBI)1US site
- Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)Only in OhioRecruitingPhase 4Ages 6 Months+Children's Hospital Medical Center, Cincinnati1US site
- Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-HemoglobinopathiesOnly in MassachusettsRecruitingPhase 1Ages 13-40Daniel Bauer1US site
- International Registry of Patients With Alpha ThalassemiaOnly in CaliforniaRecruitingUniversity of California, San Francisco1US site
- Partial Stem Cell Transplant for Sickle Cell Disease From Matched DonorsOnly in MarylandRecruitingPhase 1/2Ages 4-65National Heart, Lung, and Blood Institute (NHLBI)1US site
- Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-ThalassemiaOnly in New YorkRecruitingPhase 2Ages 6-17Celgene1US site
- Study of the Role of Genetic Modifiers in HemoglobinopathiesOnly in MassachusettsRecruitingAges 2+Cyprus Institute of Neurology and Genetics1US site
- T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other AnemiasOnly in PennsylvaniaRecruitingPhase 1/2Ages 5-40Paul Szabolcs1US site
- A Long-term Follow-up Study in Participants Who Received CS-101RecruitingAges 3-17Children's Hospital of Fudan UniversityNo US site yet
- A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemiaRecruitingPhase 1Ages 18-65Mabwell (Shanghai) Bioscience Co.No US site yet
- A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia MajorRecruitingPhase 1Ages 3-35Shanghai BDgene Co.No US site yet
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Who runs the Thalassemia trials in the US, and what stage they are in
Of the 17 Thalassemia trials with a US site, 4 are in Phase 3, the last stage before a company can ask the FDA for approval. Earlier Phase 1 or 2 tests of safety and dosing make up 7 of them. One is a Phase 4 study of an already approved treatment. The remaining 5 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 6 of them, and universities, hospitals, foundations and government groups run 11. The sponsors with the most are Agios Pharmaceuticals (2) and National Heart, Lung, and Blood Institute (NHLBI) (2). The most recently listed one first appeared on ClinicalTrials.gov on May 20, 2026.
Thalassemia trials in the US, state by state
Of the 17 Thalassemia trials with a US site, 15 accept children under 18. Their open sites are in 30 states and Washington, DC, most often New York, California and Pennsylvania. A trial counts once in each state where it has an open site, and some trials accept both children and adults.
Show the other 21 states
US hospitals running the most Thalassemia trials
Centers that run trials for a rare condition often see more people with it than most clinics do, so this list can also help you find doctors who know Thalassemia.
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania4 trials
- National Institutes of Health Clinical CenterBethesda, Maryland3 trials
- Boston Children's HospitalBoston, Massachusetts2 trials
- Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank HospitalAtlanta, Georgia2 trials
- University of Texas Southwestern Medical CenterDallas, Texas2 trials
- Weill Cornell Medical College/ New York PresbyterianNew York, New York2 trials
- Akron Children's Hospital - Showers Center for Cancer & Blood DisordersAkron, Ohio1 trial
- American Thrombosis and Hemostasis NetworkRochester, New York1 trial
- Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's HospitalPhoenix, Arizona1 trial
- Arkansas Center for Bleeding DisordersLittle Rock, Arkansas1 trial
Help getting to a Thalassemia trial far from home
Ask the study team whether the trial pays back travel, parking and hotel costs, because many sponsors do. The FDA's guidance on paying research volunteers says paying back these costs does not count as pressuring someone to join.
These nonprofits also help patients reach care, and each one sets its own rules.
- Mercy Medical Angels
- Pays for gas cards, ground rides and airline tickets anywhere in the US, and it lists clinical trials as a reason to travel. Household income at or below 300% of the federal poverty guidelines, and usually a trip of 50 miles or more each way.
- Air Charity Network
- Volunteer pilots in all 50 states fly patients for free in small private planes, and members also fly people taking part in clinical trials. You must be able to walk and sit upright for the flight, show financial need and have a backup plan if weather cancels it.
- Ronald McDonald House
- Gives families a room near the hospital while a child gets care, through 394 House programs around the world. Families of a sick child, with rules set by each local chapter.
- Healthcare Hospitality Network
- Keeps a map of hospitality houses near hospitals, and most member houses charge little or nothing to patients and families. Each house sets its own rules, so check with the house near your study site.
Program details were checked on each program's website on October 24, 2026.
Questions about finding a Thalassemia trial near you
Are there Thalassemia clinical trials near me?
As of November 13, 2026, 51 Thalassemia clinical trials are recruiting now and 22 are not yet recruiting. In the US, 17 of them have at least 1 open site, across 30 states and Washington, DC. Enter your ZIP code on this page to rank every one of them by the distance to its closest open site, with the sites shown on a map.
Which states have Thalassemia clinical trial sites?
Recruiting Thalassemia trials list open sites in 30 states and Washington, DC. New York, California and Pennsylvania have the most, and the table on this page lists every state.
Are there Thalassemia trials for children near me?
Of the 17 Thalassemia trials with a US site, 15 accept children under 18. Their open sites are in 30 states and Washington, DC, most often New York, California and Pennsylvania. Each trial sets its own age limits, so check the ages listed on a trial before you contact it.
Who pays for travel to a Thalassemia clinical trial?
Many trial sponsors pay back travel, parking and hotel costs, so ask the study team before you rule out a far-away site. The FDA's guidance says paying back these costs does not count as pressuring someone to join. Charities such as Mercy Medical Angels and Air Charity Network can also help with rides and flights.
What if there is no Thalassemia trial near me?
Some trials send staff to your home or use a local lab, so fewer visits happen at the study site. You can also sign up on this page to get an email when a Thalassemia trial opens a new site or changes status.
How is the distance to a trial site measured?
Distances are straight-line miles from the center of your ZIP code to each study site's listed location, not driving distance. Your browser looks up the ZIP code's coordinates with a free map service, Zippopotam.us, and the ZIP code is never sent to or stored by Trial Friend.
FDA-approved for Thalassemia: Aqvesme (mitapivat), Casgevy (exagamglogene autotemcel), Reblozyl (luspatercept), Zynteglo (betibeglogene autotemcel), Exjade / Jadenu (deferasirox), Ferriprox (deferiprone), Tepadina (thiotepa).
Clinical trials near you for related conditions
Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of November 13, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out. Children means the trial's age group includes people under 18.