Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle...
Sickle Cell DiseaseThalassemia2 moreBackground: - Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs. They want to know if it helps a body to better accept donor stem cells. Objectives: - To see if low dose radiation (300 rads), oral cyclophosphamide, pentostatin, and sirolimus help a body to better accept donor stem cells. Eligibility: - People 4 and older with beta-thalassemia or sickle cell disease that can be cured with transplant, and their donors. Design: Participants and donors will be screened with medical history, physical exam, blood test, tissue and blood typing, and bone marrow sampling. They will visit a social worker. Donors: may receive an intravenous (IV) tube in their groin vein. will receive a drug injection daily for 5 or 6 days to move the blood stem cells from the bone marrow into general blood circulation. will undergo apheresis: an IV is put into a vein in each arm. Blood is taken from one arm, a machine removes the white blood cells that contain blood stem cells, and the rest is returned through the other arm. Participants: may undergo red cell exchange procedure. will remain in the hospital for about 30 days. will receive a large IV line that can stay in their body from transplant through recovery. will receive a dose of radiation, and transplant related drugs by mouth or IV. will receive blood stem cells over 8 hours by IV. will take neuropsychological tests and may complete questionnaires throughout the transplant process. must stay near NIH for 4 months. They will visit the outpatient clinic weekly. will have 5 follow-up visits for 3 years after transplant, then annually.
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
Alpha Thalassemia MajorHemoglobinopathy; With Thalassemia7 moreThe investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.
A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants...
Hematologic DiseasesThis is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent...
Non-Transfusion-dependent Alpha-ThalassemiaNon-Transfusion-dependent Beta-ThalassemiaThe primary purpose of this study is to compare the effect of mitapivat versus placebo on anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).
A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia...
Transfusion Dependent Beta-ThalassaemiaThis is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.
A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion...
Transfusion Dependent Beta-ThalassaemiaThis is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent...
Transfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-ThalassemiaThe primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
β Thalassemia MajorThis is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
Sickle Cell DiseaseBeta ThalassemiaThe primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease. Patients will receive a chemotherapy regimen with busulfan, fludarabine, and alemtuzumab followed by an infusion of stem cells, either from a family-related or cord-blood matched donor.
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124...
Non-transfusion-dependent ThalassemiaLow Risk Myelodysplastic Syndrome1 moreThis study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c. doses and multiple doses in healthy male and female subjects. Up to 7 cohorts of 56 patients with Thalassaemia and up to 7 cohorts of 56 patients with MDS will be enrolled. Each subject will receive single or multiple doses of SLN124 or placebo given by subcutaneous (s.c) injection.