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Thalassemia Major clinical trials

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NCT ID: NCT03161899 Completed - Thalassemia Major Clinical Trials

Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital

Start date: December 15, 2018
Phase:
Study type: Observational

Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha & Beta thalassemia. Alpha thalassemia: occurs when a gene or genes related to the alpha globin protein are missing or mutated. Beta-thalassemia syndromes are a group of hereditary blood disorders characterized by reduced or absent beta globin chain synthesis. Beta-thalassemias can be classified into: Silent carrier: completely asymptomatic with normal hematological parameters. Beta-thalassaemia minor (beta-thalassaemia trait): usually asymptomatic; diagnosis is made during a work-up for mild anemia. Beta-thalassaemia intermedia: usually a similar presentation to beta-thalassaemia major; symptoms are usually less pronounced and the course is usually more insidious. Beta-thalassaemia major : In which there is complete absence of hemoglobin A

NCT ID: NCT02986698 Enrolling by invitation - Hemoglobinopathies Clinical Trials

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

Start date: October 5, 2017
Phase: Phase 1
Study type: Interventional

The 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.

NCT ID: NCT02559648 Completed - Osteoporosis Clinical Trials

Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis

Start date: September 2014
Phase: Phase 2
Study type: Interventional

This is a single-site, randomized, placebo-controlled, double blind phase 2b clinical trial. Patients with Thalassemia will participate in this study and will be treated with Denosumab or placebo. The effect of Denosumab on lumbar spine BMD in patients with Thalassemia Major and Osteoporosis will be evaluated as compared with control (placebo) at 12 months.

NCT ID: NCT02308904 Completed - THALASSEMIA MAJOR Clinical Trials

Reproductive Capacity and Iron Burden in Thalassemia

Fertility thal
Start date: June 1, 2013
Phase: N/A
Study type: Interventional

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 understood. Despite remarkable progress in methodology for prediction of reproductive status and intervention for preserving fertility, implementation in thalassemia is lacking. The investigators hypothesize that iron toxicity to the anterior pituitary occurring in the process of transfusional iron loading is directly associated with a decline in gonadal function. The investigators expect pituitary MRI measurements of iron deposition as well as markers of oxidative damage to correlate with the functional studies of pituitary-gonadal axis performed in this study. This cross sectional study will examine the relation of pituitary iron deposition and pituitary volume; serum iron and oxidative stress measures, liver iron concentration (LIC), cardiac iron and chelation adequacy with pituitary and gonadal reproductive hormone levels (and spermatogenesis in adult male patients), in order to better define the association of iron burden and chelation patterns with fertility potential, in thalassemia patients with iron overload. The study will assess whether the current chelation treatment regimens, in particular during the pubertal developmental age, are adequate for preserving fertility and could lead to improved chelation routines for preventing the high prevalence of compromised fertility. In addition, by utilizing state-of-the-art markers for fertility status, findings from this study may improve current methods for screening for hypogonadism and reproductive potential and allow earlier intervention. The investigators propose to examine 26-30 patients, 12 years and older, with measures of fertility potential, and correlate them to their current iron burden parameters and to the cumulative iron effect as indicated by past iron overload patterns and chelation history.

NCT ID: NCT02307786 Recruiting - Thalassemia Major Clinical Trials

Long Term Outcomes in β Thalassemia Major

Start date: June 2014
Phase: N/A
Study type: Observational

Beta thalassemia (β-thalassemia) is the most common genetic disease worldwide. Individuals with thalassemia are born with a defect in hemoglobin. Hemoglobin is a protein in red blood cells that carries oxygen to vital organs such as the brain, heart, lungs and kidneys. Thalassemia major is a hereditary anemia characterized by little or no ß-globin production, which results in hemolysis (breakdown or destruction of red blood cells) due to the formation of unstable alpha-globin tetramers and ineffective erythropoiesis which is uniformly fatal in the absence of regular transfusions. Although improvements in conservative treatment have improved the prognosis of thalassemia considerably disease and transfusion related complications in affected patients progress over time, causing severe morbidity and shortened life expectancy. Substantial lifelong health care expenses are also involved, often a financial burden for families and unsustainable in most developing countries. The hypothesis is that patients who had beta thalassemia who have undergone a hematopoietic stem cell transplant (HSCT) and are >1 year post-HSCT will have less long term comorbidities and a higher quality of life (QOL) as compared to those with beta thalassemia who are maintained on supportive care. In order to assess quality of life, a quality of life questionnaire will be asked. Extraction of data from the patient's medical record will also be used to determine any comorbidities that have occurred after either a HSCT or supportive care therapy.

NCT ID: NCT02126046 Recruiting - Thalassemia Major Clinical Trials

Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major

Start date: September 2012
Phase: N/A
Study type: Interventional

Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool,we developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD.The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-thalassemia major.

NCT ID: NCT02090699 Completed - Thalassemia Major Clinical Trials

MR Imaging of Diffuse Myocardial Fibrosis in Transfusion-Dependent Anemias

MAFIO
Start date: July 2013
Phase: N/A
Study type: Observational

Cardiac failure is the major cause of death in patients with thalassemia and chronic blood transfusion-related iron overload. The treatment of thalassemia has been revolutionized over the past decade with the implementation of cardiac MRI based assessment of iron overload. This has enabled detection of cardiac iron overload prior to symptomatic heart failure and now allows for timely therapy which has resulted in a substantial decrease in mortality. However, currently implemented MR imaging techniques assess for iron content only and not for iron related diffuse fibrosis which play a role in iron related heart failure. Histopathologic studies indicate that patients with iron overload have diffuse interstitial fibrosis. Quantitative MR techniques have shown that patients with various cardiomyopathies demonstrate diffuse myocardial fibrosis and that these changes correlate with changes in cardiac function. The investigators propose that quantitative cardiac MRI for assessment of diffuse myocardial fibrosis can further improve our ability to detect early damage to the myocardium and prevent morbidity and mortality from cardiac iron overload. Detection of fibrosis in patients with thalassemia may allow for earlier identification of cardiomyopathy when compared to other techniques in clinical use including T2* analysis. Identification of fibrosis could affect patient management as it would allow for tailoring of iron chelation therapy and may lead to better understanding of the disease processes contributing to heart failure and arrhythmia in these patients.

NCT ID: NCT02083575 Not yet recruiting - Thalassemia Major Clinical Trials

Role of Vitamin C to Augment Iron Chelation With DFP or DFX

Start date: April 2014
Phase: Phase 2/Phase 3
Study type: Interventional

role of Vit C to Augment iron chelation with DFP or DFX in thalassemic patients.

NCT ID: NCT02049450 Completed - Thalassemia Major Clinical Trials

Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.

Start date: May 2014
Phase: Phase 2
Study type: Interventional

Patients with severe thalassemia (thalassemia major) present with severe anemia that requires life-long transfusion therapy, spleen enlargement that may lead to increased transfusion requirement, and other serious complications as early death, growth retardation, bone deformations and iron overload due to blood transfusions. Splenectomy can significantly reduce transfusion requirement in thalassemia patients, but it is associated with an increased risk of serious complications such as sepsis and thrombosis. Preliminary preclinical and clinical data suggest that JAK2 inhibition, by reducing spleen size, may improve hemoglobin levels, thereby eliminating the need for splenectomy and reducing transfusion requirement and related iron overload.

NCT ID: NCT01917708 Completed - Sickle Cell Disease Clinical Trials

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Start date: January 2014
Phase: Phase 1
Study type: Interventional

This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.