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Active clinical trials for "beta-Thalassemia"

Results 41-50 of 249

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia...

Beta-ThalassemiaThalassemia4 more

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Active13 enrollment criteria

ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55...

Confirmed Diagnosis of ß-thalassemia Major

The patient has inherited ß-thalassemia major through the genes. These genes have mistakes in them, so the body cannot make normal red blood cells. Stem cells are made in the bone marrow. They are the earliest form of blood cells. This study is being done to see if the investigators can make the stem cells produce normal red blood cells and hemoglobin. The investigators do this by collecting the stem cells. The genes with mistakes are removed from the cells. These cells are then treated so they have the corrected gene for making normal hemoglobin. These treated cells are given back to the patient through an injection (shot) in the vein. This is also known as gene transfer. In order for the body to accept these cells, the patient will need to receive a low dose of a drug called busulfan. It is a drug that will prepare the body to receive the new stem cells. This study will let the investigators know: If it is safe to give the patient the treated stem cells If the treated stem cells will go into the bone marrow without causing side effects. Gene transfer has been used for the past five years. It has been successful in treating many blood disorders. At least 20 patients have received the type of treatment that the patient will get on this study. This treatment for B-thalassemia major was developed here at Memorial Sloan Kettering (MSK). It was studied for a long time in the lab before being given to patients.

Active35 enrollment criteria

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation...

Bone Marrow Failure SyndromeSevere Aplastic Anemia14 more

This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the time to neutrophil and platelet engraftment, to determine the time to immune reconstitution (including normalization of T, B and natural killer (NK) cell repertoire and Immunoglobulin G production), and to establish the incidence of infectious complications including bacterial, viral, fungal and atypical mycobacterial and other infections following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant disease.

Active27 enrollment criteria

Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia...

β-Thalassemia Major

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients

Not yet recruiting9 enrollment criteria

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent...

β-thalassemia

This study will be intented to evaluate the safety and efficacy of lentiviral vector transduction of β-globin genetically modified autologous CD34+ hematopoietic stem cells in patients with transfusion-dependent β-thalassemia.

Not yet recruiting29 enrollment criteria

Study of the Role of Genetic Modifiers in Hemoglobinopathies

Sickle Cell DiseaseThalassemia3 more

This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).

Recruiting6 enrollment criteria

Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia...

Beta-Thalassemia

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.

Not yet recruiting11 enrollment criteria

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent...

β-thalassemia

This study will be intented to evaluate the safety and efficacy of lentiviral vector transduction of β-globin genetically modified autologous CD34+ hematopoietic stem cells in patients with transfusion-dependent β-thalassemia.

Not yet recruiting28 enrollment criteria

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia...

Transfusion-dependent Beta-ThalassemiaGene Therapy

The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.

Recruiting24 enrollment criteria

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia...

Beta Thalassaemia

OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene. The TIGET-BTHAL is a phase I/II study evaluating safety and efficacy of OTL-300 in subjects with transfusion dependent beta-thalassemia for two years post gene-therapy. Subjects with rare disease who have undergone gene therapy are followed for efficacy and possible delayed adverse events. Thus, this study is designed to follow patients who have received gene therapy on TIGET-BTHAL for an additional six years (for a total of eight years).

Active3 enrollment criteria
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