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Clinical trials test new treatments โ€” sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

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23 recruiting trials

for Beta Thalassemia Transfusion Dependent

Long-term Follow-up Study of BHC001 for TDT NCT06314529n/a2 sites๐Ÿ“ Nanning๐Ÿ“ Changsha Bioray Laboratories ยท Tests: Assessments A Trial Testing SP-420 in Subjects With Transfusion-dependent ฮฒ-thalassemia or Low-risk Myelodysplastic Syndromes NCT05693909Phase 21 site๐Ÿ“ Copenhagen Pharmacosmos A/S ยท Tests: SP-420 T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias NCT03653338Phase 1, Phase 21 site๐Ÿ“ Pittsburgh Paul Szabolcs ยท Tests: CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia NCT07680803Phase 22 sites๐Ÿ“ Rome๐Ÿ“ Milan Fondazione Telethon ยท Tests: FT007 A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent ฮฒ-Thalassemia [TDT] NCT05762510EARLY Phase 11 site๐Ÿ“ Nanning First Affiliated Hospital of Guangxi Medical University ยท Tests: GMCN-508B (LentiRed) Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia NCT06490627Phase 21 site๐Ÿ“ Karachi National Institute of Blood and Marrow Transplant (NIBMT), โ€ฆ ยท Tests: Thalidomide, Thalidomide Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent ฮฒ-Thalassemia and Severe Sickle Cell Disease NCT05477563Phase 36 sites๐Ÿ“ New York๐Ÿ“ Charlotte๐Ÿ“ Nashville Vertex Pharmaceuticals Incorporated ยท Tests: CTX001 Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia NCT05773729NA1 site๐Ÿ“ Shanghai Shanghai BDgene Co., Ltd. ยท Tests: BD211 Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients NCT06219239NA1 site๐Ÿ“ Tianjin Institute of Hematology & Blood Diseases Hospital, China ยท Tests: KL003 cell injection Drug Product A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent ฮฒ-thalassemia NCT06772766Phase 12 sites๐Ÿ“ Nanning๐Ÿ“ Haikou Mabwell (Shanghai) Bioscience Co., Ltd. ยท Tests: 9MW3011, 9MW3011 placebo ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia NCT06364774Phase 1, Phase 21 site๐Ÿ“ Philadelphia Children's Hospital of Philadelphia ยท Tests: ALS20 Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent ฮฒ-Thalassemia NCT07884773Phase 31 site๐Ÿ“ Peshawar Khyber Medical University Peshawar ยท Tests: Thalidomide, Danazol, Prednisolone Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent ฮฒ-thalassemia NCT05776173NA1 site๐Ÿ“ Shanghai Shanghai BDgene Co., Ltd. ยท Tests: BD211 A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (ฮฑ- or ฮฒ-TDT) NCT07506863Phase 34 sites๐Ÿ“ Atlanta๐Ÿ“ New York๐Ÿ“ Philadelphia Agios Pharmaceuticals, Inc. ยท Tests: Mitapivat Matched Placebo, Mitapivat ฮ’-Thalassemia Treatment with KL003 Cell Injection NCT06280378Phase 1, Phase 22 sites๐Ÿ“ Shanghai๐Ÿ“ Tianjin Kanglin Biotechnology (Hangzhou) Co., Ltd. ยท Tests: KL003 Cell Injection Drug Product Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy NCT05991336n/a1 site๐Ÿ“ Tianjin Institute of Hematology & Blood Diseases Hospital, China ยท Tests: Gene therapy Long-term Follow-up Study of BRL-101 for TDT NCT06298630n/a7 sites๐Ÿ“ Guangzhou๐Ÿ“ Guangzhou๐Ÿ“ Shenzhen Bioray Laboratories ยท Tests: Assessments A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With ฮฑ- or ฮฒ-Non-Transfusion-Dependent Thalassemia NCT07517133Phase 35 sites๐Ÿ“ Palo Alto๐Ÿ“ Atlanta๐Ÿ“ New York Agios Pharmaceuticals, Inc. ยท Tests: Mitapivat, Placebo Matching Mitapivat Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT NCT01962415Phase 21 site๐Ÿ“ Pittsburgh Paul Szabolcs ยท Tests: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East NCT07215975n/a13 sites๐Ÿ“ Al Jahra๐Ÿ“ Andalusia๐Ÿ“ Kuwait City Bristol-Myers Squibb ยท Tests: Luspatercept Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe ฮฒ-Hemoglobinopathies NCT06647979Phase 11 site๐Ÿ“ Boston Daniel Bauer ยท Tests: autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequenโ€ฆ Evaluation of Physiological and Psychological Factors Involved in Exercise Intolerance in Patients With ฮฒ-TTD NCT07207577NA1 site๐Ÿ“ Chambรฉry Centre Hospitalier Metropole Savoie ยท Tests: Maximal incremental exercise test A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating ฮฒ-thalassemia Major NCT06465550Phase 13 sites๐Ÿ“ Guangzhou๐Ÿ“ Nanning๐Ÿ“ Shanghai Shanghai BDgene Co., Ltd. ยท Tests: BD211
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