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Thalassaemia: nyitott vizsgálatok itt: Egyesült Államok

28 nyitott vizsgálatnak van helyszíne itt: Egyesült Államok. Regiszteradat; hogy ki vehet részt, azt csak a vizsgálat csapata dönti el.

  • 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)

    Kísérleti kezelés · 3. fázis · 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

    Regiszter frissítve: 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

    Kísérleti kezelés · 3. fázis · 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

    Regiszter frissítve: 2026-10-28

  • NCT04008368

    Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

    Kísérleti kezelés · 1/2. fázis · National Heart, Lung, and Blood Institute (NHLBI)

    National Institutes of Health Clinical Center, Bethesda

    Regiszter frissítve: 2026-10-26

  • NCT04099966

    AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

    Kísérleti kezelés · 2. fázis · Mitchell Cairo

    New York Medical College, Valhalla

    Regiszter frissítve: 2026-10-23

  • NCT07599176

    Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

    Kísérleti kezelés · 1/2. fázis · National Heart, Lung, and Blood Institute (NHLBI)

    National Institutes of Health Clinical Center, Bethesda

    Regiszter frissítve: 2026-10-13

  • NCT03937817

    Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

    Kutatás · National Institute of Allergy and Infectious Diseases (NIAID)

    National Institutes of Health Clinical Center, Bethesda

    Regiszter frissítve: 2026-10-02

  • NCT06313398

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

    Kísérleti kezelés · Korai 1. fázis · National Heart, Lung, and Blood Institute (NHLBI)

    National Institutes of Health Clinical Center, Bethesda

    Regiszter frissítve: 2026-09-28

  • NCT06609226

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

    Kísérleti kezelés · 3. fázis · 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

    Regiszter frissítve: 2026-09-18

  • NCT04872179

    International Registry of Patients With Alpha Thalassemia

    Kutatás · University of California, San Francisco

    University of California San Francisco, San Francisco

    Regiszter frissítve: 2026-09-02

  • NCT04398628

    ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

    Kutatás · American Thrombosis and Hemostasis Network

    Akron Children's Hospital - Showers Center for Cancer & Blood Disorders, Akron · University of New Mexico Ted R. Montoya Hemophilia & Thrombosis Program, Albuquerque · Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta, Atlanta · Emory/Children's Health Care of Atlanta, Atlanta · Children's Blood and Cancer Center of Central Texas, Austin · Johns Hopkins University Hemophilia Treatment Center, Baltimore · Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center, Boston · Western New York BloodCare, Buffalo · Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill · St. Jude Affiliate Clinic at Novant Health Hemby Children's Hospital, Charlotte · Rush University Medical Center, Chicago · Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center, Cincinnati · University of Cincinnati Medical Center Hemophilia Treatment Center, Cincinnati · University Hospitals Health System Cleveland, Cleveland · Nationwide Children's Hospital, Columbus, Columbus · Ohio State University Medical Center, Columbus · North Texas Hemophilia and Thrombosis Program - Pediatric Program / Center for Cancer & Blood Disorders, Dallas · North Texas Comprehensive Hemophilia Treatment Center, Dallas · Dayton Children's Hemostasis and Thrombosis Center, Dayton · Central Michigan Children's Hospital of Michigan, Detroit · Henry Ford Health System Bleeding and Thrombosis Treatment Center, Detroit · University of Florida Hemophilia Treatment Center, Gainesville · Hemophilia Outreach Center Green Bay, Green Bay · East Carolina University Hemophilia Treatment Center, Greenville · Connecticut Children's Medical Center, Hartford · Texas Children's Hemophilia & Thrombosis Center/Baylor College of Medicine, Houston · Gulf States Hemophilia and Thrombophilia Center, Houston · Northwell Health Hemostasis and Thrombosis Center at Long Island Jewish and Cohen Children's Medical Center, Hyde Park · Indiana Hemophilia and Thrombosis Center, Indianapolis · Iowa Hemophilia and Thrombosis Center, Iowa City · Children's Mercy Hospital, Kansas City, Kansas City · Cure 4 the Kids Foundation, Las Vegas · Arkansas Center for Bleeding Disorders, Little Rock · Orthopaedic Institute for Children HTC, Los Angeles · Childrens Hospital Los Angeles, Los Angeles · St. Jude Children's Research Hospital, Memphis · University of Miami Comprehensive Hemophilia Treatment Center, Miami · University of Miami Hospital and Clinics, Miami · Comprehensive Center for Bleeding Disorders, Milwaukee · Vanderbilt University Medical Center, Nashville · Newark Beth Israel Medical Center - Hemophilia Center, Newark · Yale Hemophilia Treatment Center, New Haven · Louisiana Center for Bleeding and Clotting Disorders, Tulane University, New Orleans · Weill Cornell Medical College-New York Presbyterian Hospital, New York · Children's Hospital of Kings Daughters, Norfolk · UCSF Benioff Children's Hospital Oakland, Oakland · Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders, Orlando · Bleeding and Clotting Disorders Institute, Peoria · Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania, Philadelphia · St. Christopher's Hospital for Children, Philadelphia · Children's Hospital of Philadelphia, Philadelphia · Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital, Phoenix · Hemophilia Center of Western Pennsylvania, Pittsburgh · Rhode Island Hospital Hemostasis and Thrombosis Center, Providence · Hemostasis and Thrombosis Center of Nevada, Reno · American Thrombosis and Hemostasis Network, Rochester · Mayo Comprehensive Hemophilia Center, Rochester · University of California at Davis Hemophilia Treatment Center, Sacramento · South Texas Comprehensive Hemophilia and Thrombophilia Treatment Center, San Antonio · Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program, San Bernardino · Rady Children's Hospital - San Diego, San Diego · Hemophilia & Thrombosis Treatment Center at UC San Diego Health, San Diego · University of California, San Francisco Hemophilia & Thrombosis Center, San Francisco · Memorial Health University Medical Center, Savannah · Maine Hemophilia and Thrombosis Center, Scarborough · Washington Center for Bleeding Disorders, Seattle · The John Bouhasin Center for Children with Bleeding Disorders, St Louis · Johns Hopkins All Children's Hospital, St. Petersburg · University of South Florida - Adult, Tampa · St. Joseph's Hospital Center for Bleeding & Clotting Disorders, Tampa · Montefiore Medical Center, The Bronx · Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital, Toledo · Georgetown University, Washington D.C. · Children's National Hemophilia Center, Washington D.C. · Delaware Hemophilia Treatment Center, Wilmington · Wake Forest University Health Sciences, Winston-Salem

    Regiszter frissítve: 2026-08-17

  • NCT03653338

    T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

    Kísérleti kezelés · 1/2. fázis · Paul Szabolcs

    Children's Hospital of Pittsburgh of UPMC, Pittsburgh

    Regiszter frissítve: 2026-08-13

  • NCT04208529

    A Long-term Follow-up Study in Participants Who Received CTX001

    Kísérleti kezelés · 3. fázis · 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

    Regiszter frissítve: 2026-08-11

  • NCT02675959

    Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

    Kísérleti kezelés · 2. fázis · 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

    Regiszter frissítve: 2026-07-27

  • NCT04143724

    Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

    Kísérleti kezelés · 2. fázis · Celgene

    Los Angeles · New York - Presbyterian Hospital, New York

    Regiszter frissítve: 2026-07-10

  • NCT04064060

    A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

    Kísérleti kezelés · 3. fázis · 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

    Regiszter frissítve: 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)

    Kísérleti kezelés · 2. fázis · 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

    Regiszter frissítve: 2026-06-03

  • NCT04416178

    Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

    Kutatás · St. Jude Children's Research Hospital

    St. Jude Children's Research Hospital, Memphis

    Regiszter frissítve: 2026-05-01

  • NCT05477563

    Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

    Kísérleti kezelés · 3. fázis · 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

    Regiszter frissítve: 2026-03-23

  • NCT06647979

    Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

    Kísérleti kezelés · 1. fázis · Daniel Bauer

    Boston Children's Hospital, Boston

    Regiszter frissítve: 2026-02-03

  • NCT04528355

    Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

    Kutatás · Paul Szabolcs

    UPMC Children's Hospital of Pittsburgh, Pittsburgh

    Regiszter frissítve: 2026-01-13

  • NCT01245179

    Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

    Kísérleti kezelés · 1. fázis · Abdullah Kutlar

    Augusta University, Augusta

    Regiszter frissítve: 2026-01-09

  • NCT01962415

    Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

    Kísérleti kezelés · 2. fázis · Paul Szabolcs

    UPMC Children's Hospital of Pittsburgh, Pittsburgh

    Regiszter frissítve: 2025-12-15

  • NCT07177300

    Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)

    Kísérleti kezelés · 4. fázis · Children's Hospital Medical Center, Cincinnati

    Cincinnati Children's Hospital Medical Center, Cincinnati

    Regiszter frissítve: 2025-09-16

  • NCT04432623

    The BENeFiTS Trial in Beta Thalassemia Intermedia

    Kísérleti kezelés · 1/2. fázis · Phoenicia BioScience

    Massachusetts General Hospital, Boston · Weil Cornell Medicine, New York · UCSF Benioff Children's Hospital at Oakland, Oakland · Susan Perrine, Weston

    Regiszter frissítve: 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

    Kutatás · 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

    Regiszter frissítve: 2025-04-02

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