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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 (17) clinical trials

Allogeneic SCT of CordIn in Patients With Hemoglobinopathies

CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The trial ends when the last patient completes their last visit. The overall study objectives are to evaluate the safety and efficacy of CordIn™: single ex-vivo expanded cord blood unit transplantation ...

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

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

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 β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. The study will evaluate the efficacy ...

Phase

L-Glutamine Therapy for Sickle Cell Anemia

Sickle cell anemia is one of the most common and devastating hereditary disorders with significant morbidity and mortality affecting individuals of African-American heritage. No safe effective therapy is yet available. An ideal agent would be one that is readily available, effective, and safe even with chronic use. Early studies using ...

Phase

Factors Promoting Increased Rate and Success of Pregnancy in the Thalassemia Population in Toronto

Treatment of patients with beta thalassemia in North America has altered dramatically during the past 40 years, with improvements in transfusion therapy and introduction of iron chelation therapy. Thalassemia patients now enjoy an increased life expectancy to the fifth and sixth decades of life, with fertility and childbearing becoming important ...

Phase N/A

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 Pilot Study of HSCT for Patients With High-Risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen

Severe hemoglobinopathies such as sickle cell disease (SCD) and Thalassemia are associated with considerable morbidity, organ damage and premature mortality. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only therapy that can cure a hemoglobinopathy. The applicability of HSCT for hemoglobinopathies is limited by the paucity of suitable donors, and ...

Phase N/A

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 β-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

Iron Balance Study of Deferasirox Deferoxamine and the Combination of Both

The study involves a 34-day hospital stay during which we will conduct metabolic iron balance studies wherein deferasirox and deferoxamine are evaluated separately and in combination, each subject serving as his/her own control. In the studies currently proposed, deferasirox (20, 30 or 40 mg/kg, administered p.o. 30 minutes before breakfast) ...

Phase