Thalassaemia trials in United States

In United States 28 Also abroad 73

28 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

Phase 3Taking people
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)
Where
Atlanta, Dallas, New York, Philadelphia
Age
1 year to 17 years
People
54 worldwide
Run by
Agios Pharmaceuticals, Inc.
See details and how to joinNCT07506863 · EU 2025-524707-71-00
Phase 3Taking people
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
Where
Atlanta, Dallas, New York, Palo Alto, Philadelphia
Age
1 year to 17 years
People
45 worldwide
Run by
Agios Pharmaceuticals, Inc.
See details and how to joinNCT07517133 · EU 2025-524706-15-00
Phase 1/2Taking people
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Where
Bethesda
Age
2 years to 80 years
People
32
Run by
National Heart, Lung, and Blood Institute (NHLBI)
See details and how to joinNCT04008368
Phase 2Taking people
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Where
Valhalla
Age
1 day to 30 years
People
20
Run by
Mitchell Cairo
See details and how to joinNCT04099966
Phase 1/2Taking people
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
Where
Bethesda
Age
4 years to 65 years
People
90
Run by
National Heart, Lung, and Blood Institute (NHLBI)
See details and how to joinNCT07599176
No phaseTaking people
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Where
Bethesda
Age
18 years to 70 years
People
300
Run by
National Institute of Allergy and Infectious Diseases (NIAID)
See details and how to joinNCT03937817
Early phase 1Taking people
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Where
Bethesda
Age
18 years to 100 years
People
100
Run by
National Heart, Lung, and Blood Institute (NHLBI)
See details and how to joinNCT06313398
Phase 3Taking people
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Where
Atlanta, Augusta, Birmingham, Boston, Brooklyn, Charleston, Charlotte, Chicago, Cincinnati, Durham, Farmington, Greenville, Hollywood, Houston, Los Angeles, Memphis, Miami, Milwaukee, New Orleans, New York, North Canton, Oakland, Orange, Phoenix, Richmond, St Louis, Tacoma, The Bronx, Washington D.C., Winston-Salem
Age
2 years and over
People
480 worldwide
Run by
Novo Nordisk A/S
See details and how to joinNCT06609226 · EU 2024-510805-27-00
No phaseTaking people
International Registry of Patients With Alpha Thalassemia
Where
San Francisco
People
500
Run by
University of California, San Francisco
See details and how to joinNCT04872179
No phaseTaking people
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Where
Akron, Albuquerque, Atlanta, Austin, Baltimore, Boston, Buffalo, Chapel Hill, Charlotte, Chicago, Cincinnati, Cleveland, Columbus, Dallas, Dayton, Detroit, Gainesville, Green Bay, Greenville, Hartford, Houston, Hyde Park, Indianapolis, Iowa City, Kansas City, Las Vegas, Little Rock, Los Angeles, Memphis, Miami, Milwaukee, Nashville, Newark, New Haven, New Orleans, New York, Norfolk, Oakland, Orlando, Peoria, Philadelphia, Phoenix, Pittsburgh, Providence, Reno, Rochester, Sacramento, San Antonio, San Bernardino, San Diego, San Francisco, Savannah, Scarborough, Seattle, St Louis, St. Petersburg, Tampa, The Bronx, Toledo, Washington D.C., Wilmington, Winston-Salem
People
3,000
Run by
American Thrombosis and Hemostasis Network
See details and how to joinNCT04398628
Phase 1/2Taking people
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Where
Pittsburgh
Age
5 years to 40 years
People
5
Run by
Paul Szabolcs
See details and how to joinNCT03653338
Phase 3Enrolling by invitation
A Long-term Follow-up Study in Participants Who Received CTX001
Where
Charlotte, Chicago, Memphis, Nashville, New York, Palo Alto, Philadelphia, San Antonio
Age
2 years to 64 years
People
160 worldwide
Run by
Vertex Pharmaceuticals Incorporated
See details and how to joinNCT04208529 · EU 2024-512654-19-00
Phase 2Taking people
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
Where
Gainesville, Los Angeles, Milwaukee, Valhalla
Age
6 months to 21 years
People
40
Run by
New York Medical College
See details and how to joinNCT02675959
Phase 2Taking people
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
Where
Los Angeles, New York
Age
6 years to 17 years
People
99 worldwide
Run by
Celgene
See details and how to joinNCT04143724 · EU 2022-502499-22-00
Phase 3Taking people
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
Where
Boston, Chicago, Cleveland, Detroit, Houston, Los Angeles, Nashville, New York, Oakland, Philadelphia, Stanford, Tampa
Age
18 years and over
People
665 worldwide
Run by
Celgene
See details and how to joinNCT04064060
Phase 2Taking people
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)
Where
Basking Ridge, Commack, Harrison, Middletown, Montvale, Rockville Centre
Age
2 years to 50 years
People
24
Run by
Memorial Sloan Kettering Cancer Center
See details and how to joinNCT05736419
No phaseTaking people
Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders
Where
Memphis
Age
13 years and over
People
352
Run by
St. Jude Children's Research Hospital
See details and how to joinNCT04416178
Phase 3Taking people
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Where
Charlotte, Nashville, New York
Age
12 years to 35 years
People
26 worldwide
Run by
Vertex Pharmaceuticals Incorporated
See details and how to joinNCT05477563 · EU 2024-514641-12-00
Phase 1Taking people
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Where
Boston
Age
13 years to 40 years
People
10
Run by
Daniel Bauer
See details and how to joinNCT06647979
No phaseTaking people
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Where
Pittsburgh
Age
2 months to 60 years
People
50
Run by
Paul Szabolcs
See details and how to joinNCT04528355
Phase 1Taking people
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Where
Augusta
Age
18 years and over
People
18
Run by
Abdullah Kutlar
See details and how to joinNCT01245179
Phase 2Taking people
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Where
Pittsburgh
Age
2 months to 55 years
People
100
Run by
Paul Szabolcs
See details and how to joinNCT01962415
Phase 4Taking people
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
Where
Cincinnati
Age
6 months and over
People
50
Run by
Children's Hospital Medical Center, Cincinnati
See details and how to joinNCT07177300
Phase 1/2Enrolling by invitation
The BENeFiTS Trial in Beta Thalassemia Intermedia
Where
Boston, New York, Oakland, Weston
Age
18 years and over
People
36 worldwide
Run by
Phoenicia BioScience
See details and how to joinNCT04432623
No phaseEnrolling by invitation
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Where
Atlanta, Aurora, Charleston, Charlotte, Cleveland, Columbus, Dallas, Fort Worth, Minneapolis, Nashville, New Haven, New York, Oakland, Philadelphia, St. Petersburg
Age
12 years to 50 years
People
54 worldwide
Run by
Editas Medicine, Inc.
See details and how to joinNCT06363760