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Thalassemia Clinical Trials

A listing of Thalassemia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (47) clinical trials

Haploidentical Hematopoietic Stem Cell Transplantation

Research subjects will undergo reduced intensity conditioning (Hydroxyurea, ATG, Fludarabine, Thiotepa, Melphalan) followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34+ positively selected using the CliniMACS device. Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and ...

Phase

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

This is single site, dose- escalation study of SCD-101 in participants with homozygous sickle cell disease (S/S) or S/beta 0 Thalassemia. All participants will be monitored for safety, tolerability, and dose-limiting toxicities. The study is divided into two parts. Part A is an open-label, non-randomized, non-placebo-controlled dose escalation study with ...

Phase

A Study Evaluating the Efficacy and Safety of the LentiGlobin BB305 Drug Product in Subjects With Transfusion-Dependent -Thalassemia Who do Not Have a 0/ 0 Genotype

This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 23 subjects 50 years of age with transfusion-dependent -thalassemia (TDT), also known as -thalassemia major, who do not have a 0 mutation at both alleles of the hemoglobin (HBB) gene. The study will evaluate the efficacy and safety of ...

Phase

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Sickle cell disease is a genetic disorder in which a mutation in the beta chain of human hemoglobin results in abnormal blood hemoglobin, causing red blood cells to sickle under stress with resulting symptoms including severe pains and strokes. Beta thalassemia is another genetic disorder in which there are abnormal ...

Phase

A Study Evaluating the Efficacy and Safety of the LentiGlobin BB305 Drug Product in Subjects With Transfusion-Dependent -Thalassemia Who Have a 0/ 0 Genotype

This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 15 subjects 12 and 50 years of age with transfusion-dependent -thalassemia (TDT), who have a 0 mutation at both alleles of the -globin (HBB) gene (i.e. 0/0). The study will evaluate the efficacy and safety of autologous hematopoietic stem ...

Phase

Bone Marrow for Hemoglobinopathy Research

These studies are designed to evaluate the potential of retroviral vector mediated gene transfer, gene editing, or drug treatment to correct the pathophysiology of sickle cell anemia and -thalassemia. CD34+ cells purified from bone marrow of research participants with a sickle cell syndrome or a thalassemia syndrome will be subjected ...

Phase N/A

Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major

Allo-geneic stem cell transplantation(allo-HSCT) cure thalassemia major by destroying the original hematopoietic and immune systems with a large dose of chemotherapy, rebuilding a new system to correct the abnormal hematopoietic globin chain synthesis which leads to hemolysis. Currently, it is the only curative means. According to donors, allo-HSCT could be ...

Phase

Study on the Mechanism of Colla Corri Asini in the Treatment of Thalassemia Patients With Pregnancy Anemia

Thalassemia is a type of hemolytic anemia disease caused by genetic defect of synthesis in one or more globin chains. Among all the single genetic disorders, thalassemia has the highest incidence rate in the world and causes heavy burdens on public health system. In China, the southern provinces suffer from ...

Phase

Reproductive Capacity and Iron Burden in Thalassemia

The improved long-term survival of thalassemia major (TM) patients has resulted in increased focus on the ability to preserve fertility. While the association of iron toxicity with vital organ dysfunction, heart and liver, has been extensively investigated, the correlation of reproductive capacity and extent of iron overload is not well ...

Phase N/A

Fetal Hemoglobin Induction Treatment Metformin

This is a dose escalation, pilot study for subjects with sickle cell anemia (SCA) disease and non-transfusion dependent thalassemia (NTDT) to determine if metformin has a beneficial effect on the treatment of SCA and NTDT patients. In addition the following items will also occur: Data Collection will occur from subject's ...

Phase N/A