Clinical Trials for Hemoglobinopathies

Discarded Bone Marrow for Hematology Research

NCT ID: NCT04671212

This clinical trial, titled "Discarded Bone Marrow for Hematology Research," aims to collect and study bone marrow samples from individuals with blood disorders known as hemoglobinopathies, like si...

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

NCT ID: NCT06363760

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

NCT ID: NCT05477563

This clinical trial is studying a new treatment called CTX001 for people with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The treatment involves modifying a patie...

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

NCT ID: NCT06839456

This clinical trial is studying a new approach to help prevent serious infections in patients with certain types of blood cancers, such as leukemia and lymphoma, after they receive a stem cell tran...

Allo HSCT for High Risk Hemoglobinopathies

NCT ID: NCT06872333

This clinical trial, called "Allo HSCT for High Risk Hemoglobinopathies," is studying a treatment called allogeneic hematopoietic stem cell transplantation (HSCT) for patients with serious blood di...

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT ID: NCT05356195

This clinical trial is studying a new treatment called CTX001 for children with a serious blood disorder known as transfusion-dependent beta-thalassemia (TDT). TDT is a condition where the body doe...

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

NCT ID: NCT04208529

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

NCT ID: NCT04644016

This clinical trial is studying the use of cord blood transplants for children and young adults with serious blood cancers or certain non-cancer conditions. The goal is to understand how safe this ...

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

NCT ID: NCT05329649

This clinical trial is looking at a new treatment called CTX001 for children with severe sickle cell disease (SCD), especially for those who have not responded well to or cannot tolerate the standa...

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT ID: NCT03128996

This clinical trial is investigating a type of bone marrow transplant called familial HLA-mismatched bone marrow transplant for young patients under 21 years old who have serious non-cancer-related...

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

NCT ID: NCT00920972

This clinical trial is studying a new way to prepare patients for a bone marrow transplant using a combination of medications called Campath, Fludarabine, and Melphalan. The goal is to find out if ...

Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)

NCT ID: NCT01666080

This clinical trial is looking at a new treatment approach for patients who need a second or greater stem cell transplant after not getting good results from their first transplant. The trial uses ...

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

NCT ID: NCT06107400

This clinical trial is studying a new treatment called RM-004 for patients with Hemoglobin H-Constant Spring disease, a type of genetic blood disorder that affects how hemoglobin, the protein that ...

Study of the Role of Genetic Modifiers in Hemoglobinopathies

NCT ID: NCT05799118

This clinical trial is studying how certain genetic factors might affect conditions like sickle cell disease and thalassemia, which are types of inherited blood disorders. Researchers will analyze ...