Active clinical trials for "beta-Thalassemia"

This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.

Silymarin, a flavonolignan complex isolated from Silybum marianum, has a strong antioxidant, hepatoprotective and iron chelating activities. The present study has been designed to investigate the therapeutic activity of orally administered silymarin in patients with thalassemia major under conventional iron chelation therapy. A 6-month randomized, double-blind, clinical trial was conducted in 140 beta-thalassemia major patients in two well-matched groups. Patients are randomized to receive a silymarin tablet (140 mg) three times a day plus conventional desferrioxamine therapy or the same therapy but a placebo tablet instead of silymarin. Clinical laboratory tests of iron status and liver function are assessed at the beginning and the end of the trial.

safety and efficacy of different iron chelation therapy in transfusion dependent beta-thalaasemia patients with low serum ferritin and continued regular transfusion regimen.

This is a single group, open label study in 10 subjects who are 8 years of age or older with beta-thalassemia major. The objective of this study is to evaluate the safety and efficacy of autologous hematopoietic stem cell transduced with lentiviral vector for the treatment of beta-thalassemia major.

This is a randomized, open label, two arms superiority trial of a representative population of patients with a primary diagnosis of transfusion dependent thalassemia with evidence of moderate cardiac iron overload, defined as an average T2* MRI parameter at the mid inter-ventricular septum between 10 and 20ms.

Thalassemia major is a genetic disorder affecting hemoglobin synthesis, rendering individuals dependent upon lifelong blood transfusions. Consequently, iron overload occurs and patients have shortened life expectancy with the most common cause of death being heart failure. This trial tests whether the combination of traditional therapy (deferoxamine) with a newer drug (deferiprone) will prove more effective in removing cardiac iron than deferoxamine alone.

Beta Thalassemia is a major public health problem in Mediterranean countries.In Egypt, it is considered as the most common chronic hemolytic anemia.one of the major complications in this inherited disorder is iron overload which lead to oxidative stress and tissue damage. Regarding toxic effect of iron overload on liver, hepatomegaly is one of the most findings that resulting from hemosiderosis, extra medullary hematopoiesis, transmitted hepatitis B and C and cirrhosis. A lot of studies have been carried out recently to study the beneficial role of metformin in non-diabetic patients of different disorders as non-alcoholic fatty liver disease (NAFLD).Among several studies, it's demonstrated that metformin significantly improved insulin resistance, aminotransferase levels and liver morphology. The role of metformin in these studies is mainly thought to be antioxidant and anti-inflammatory effects. However, the role of Metformin on hepatic function in different populations with the same mechanism of liver injury should be further investigated. This clinical trial will be carried out on 60 patients with beta thalassemia major receiving regular blood transfusion and iron chelating therapy, either HCV positive or negative patients. They will be randomly distributed into either control group (group 1, n=30) receiving blood transfusion and taking iron chelating therapy or treatment group (group 2, n=30) receiving blood transfusion and taking iron chelating therapy along with metformin tablets (500 mg/twice daily) for 6 months.

Evaluate the therapeutic effects, health benefits, and immunological effect of Al-hijamah in children with beta thalassemia major. Evaluate the extent of clinical improvement, reductions in serum iron and ferritin, antioxidant status, in those patients.

Evaluate the therapeutic effects health benefits, and immunological effect of Nigella sativa in children with beta thalassemia major.

This is prospective randomized, double blind study designed to evaluate the use of zoledronic acid in the prevention prevention of bone loss post allogenic BMT done for beta-thalassemia major patients.