Thalassaemia: open trials in United States

20 open trials have a site in United States. Registry data; only the trial team decides who can take part.

NCT07506863
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)
Trial Treatment · Phase 3 · Agios Pharmaceuticals, Inc.
Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital, Atlanta · University of Texas Southwestern Medical Center, Dallas · Weill Cornell Medical College-New York Presbyterian, New York · Children's Hospital of Philadelphia, Philadelphia
Registry updated 2026-10-29
NCT07517133
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
Trial Treatment · Phase 3 · Agios Pharmaceuticals, Inc.
Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital, Atlanta · University of Texas Southwestern Medical Center, Dallas · Weill Cornell Medical College-New York Presbyterian, New York · Stanford Medicine, Palo Alto · Children's Hospital of Philadelphia, Philadelphia
Registry updated 2026-10-28
NCT04008368
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Trial Treatment · Phase 1/2 · National Heart, Lung, and Blood Institute (NHLBI)
National Institutes of Health Clinical Center, Bethesda
Registry updated 2026-10-26
NCT04099966
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Trial Treatment · Phase 2 · Mitchell Cairo
New York Medical College, Valhalla
Registry updated 2026-10-23
NCT07599176
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
Trial Treatment · Phase 1/2 · National Heart, Lung, and Blood Institute (NHLBI)
National Institutes of Health Clinical Center, Bethesda
Registry updated 2026-10-13
NCT03937817
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Research · National Institute of Allergy and Infectious Diseases (NIAID)
National Institutes of Health Clinical Center, Bethesda
Registry updated 2026-10-02
NCT06609226
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Trial Treatment · Phase 3 · Novo Nordisk A/S
Children's Healthcare Atlanta, Atlanta · Emory University School of Medicine, Atlanta · Center for Blood Disorders Augusta University, Augusta · Univ of Alabama Birmingham, Birmingham · Boston Medical Center, Boston · NYC Health+Hospitals, Brooklyn · Medical University of South Carolina Charleston, Charleston · Atrium Levine Children's/Atrium Health, Charlotte · Univer Of Illinois at Chicago, Chicago · Cincinnati Child's Hsp Med Ctr, Cincinnati · Duke University_Durham, Durham · University of Connecticut, Farmington · East Carolina Univ-Greenville, Greenville · East Carolina University_Greenville, Greenville · Foundation for Sickle Cell Disease Research, Hollywood · Texas Children's Hospital_Houston, Houston · UT Health University of Texas, Houston · Children's Hospital Los Angeles - Endocrinology, Los Angeles · Methodist University Hospital, Memphis · Univ of Miami/SCCC, Miami · Versiti, CCBD_Milwaukee, Milwaukee · Children's Hosp-New Orleans, New Orleans · Columbia University Medical Center_New York_0, New York · Weill Cornell Med Coll-NYPH, New York · Neuro-Behavioral Clinical Research, North Canton · UCSF Oakland Benioff ChildHosp, Oakland · Children's Hosp Of Orange, Orange · University of California Irvine, Orange · Phoenix Children's Hsptl, Phoenix · Virginia Comm Univ Medical Ctr, Richmond · Washington University St. Louis, St Louis · Mary Bridge Children's Health, Tacoma · Jacobi Medical Center, The Bronx · Montefiore Medical Center, The Bronx · Children's National Medical Center, Washington D.C. · Atrium Health-Wake Forest Bapt, Winston-Salem
Registry updated 2026-09-18
NCT03653338
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Trial Treatment · Phase 1/2 · Paul Szabolcs
Children's Hospital of Pittsburgh of UPMC, Pittsburgh
Registry updated 2026-08-13
NCT04208529
A Long-term Follow-up Study in Participants Who Received CTX001
Trial Treatment · Phase 3 · Vertex Pharmaceuticals Incorporated
Levine Children's Hospital - Hematology, Charlotte · Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology, Chicago · St. Jude Children's Research Hospital, Memphis · TriStar Medical Group Children's Specialists - Pediatric Oncology, Nashville · New York Presbyterian Hospital - Morgan Stanley Children's Hospital, New York · Herbert Irving Pavilion - Hematology, New York · Lucile Packard Children's Hospital, Palo Alto · The Children's Hospital of Philadelphia - Hematology, Philadelphia · Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital, San Antonio
Registry updated 2026-08-11
NCT02675959
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
Trial Treatment · Phase 2 · New York Medical College
University of Florida, Gainesville · University of California, Los Angeles, Los Angeles · Medical College of Wisconsin, Milwaukee · New York Medical College, Valhalla
Registry updated 2026-07-27
NCT04143724
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
Trial Treatment · Phase 2 · Celgene
Los Angeles · New York - Presbyterian Hospital, New York
Registry updated 2026-07-10
NCT04064060
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
Trial Treatment · Phase 3 · Celgene
Boston · Chicago · Cleveland · Detroit · The University of Texas MD Anderson Cancer Center, Houston · Los Angeles · Vanderbilt-Ingram Cancer Center, Nashville · New York · Oakland · Philadelphia · Stanford · Tampa
Registry updated 2026-07-01
NCT05736419
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)
Trial Treatment · Phase 2 · Memorial Sloan Kettering Cancer Center
Memorial Sloan Kettering at Basking Ridge (Consent Only), Basking Ridge · Memorial Sloan Kettering Suffolk - Commack (Consent Only), Commack · Memorial Sloan Kettering Westchester (Consent only), Harrison · Memorial Sloan Kettering Monmouth (Consent Only), Middletown · Memorial Sloan Kettering Bergen (Consent Only), Montvale · Memorial Sloan Kettering Nassau (All Protocol Activities), Rockville Centre
Registry updated 2026-06-03
NCT05477563
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Trial Treatment · Phase 3 · Vertex Pharmaceuticals Incorporated
Levine Children's Hospital - Hematology, Charlotte · TriStar Medical Group Children's Specialists - Pediatric Oncology, Nashville · New York Presbyterian Hospital - Morgan Stanley Children's Hospital, New York
Registry updated 2026-03-23
NCT06647979
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Trial Treatment · Phase 1 · Daniel Bauer
Boston Children's Hospital, Boston
Registry updated 2026-02-03
NCT01245179
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Trial Treatment · Phase 1 · Abdullah Kutlar
Augusta University, Augusta
Registry updated 2026-01-09
NCT04432623
The BENeFiTS Trial in Beta Thalassemia Intermedia
Trial Treatment · Phase 1/2 · Phoenicia BioScience
Massachusetts General Hospital, Boston · Weil Cornell Medicine, New York · UCSF Benioff Children's Hospital at Oakland, Oakland · Susan Perrine, Weston
Registry updated 2025-08-15
NCT06363760
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Research · Editas Medicine, Inc.
Children's Healthcare of Atlanta, Atlanta · Children's Hospital Colorado, Aurora · Medical University of South Carolina, Charleston · Atrium Health, Charlotte · Cleveland Clinic, Cleveland · Nationwide Children's Hospital, Columbus · Texas Oncology - Baylor Charles A. Sammons Cancer Center, Dallas · Cook Children's, Fort Worth · University of Minnesota, Minneapolis · Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers, Nashville · Smilow Cancer Hospital, New Haven · Columbia University Medical Center, New York · Columbia University Medical Center - Department of Pediatrics, New York · UCSF Benioff Children s Hospital, Oakland · Children's Hospital of Philadelphia, Philadelphia · Johns Hopkins All Children's Hospital, St. Petersburg
Registry updated 2025-04-02
NCT06271512
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Research · Genetix Biotherapeutics Inc.
Texas Children's Hospital, Houston · University of Minnesota, Minneapolis · Cohen Children's Medical Center, New Hyde Park · UCSF Benioff Children's Hospitals, Oakland · Stanford University, Palo Alto · Children's Hospital of Philadelphia, Philadelphia
Registry updated 2025-02-11
NCT06664541
Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Research · Georgetown University
Georgetown University Medical Center, Washington D.C.
Registry updated 2024-10-29

All Thalassaemia trials and the map

This page is not medical advice. Talk to your doctor before contacting a trial.

Report an error