Active clinical trials for "Thalassemia"

This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell disease and thalassemia), Blackfan-Diamond anemia and congenital or chronic hemolytic anemias; White blood cells defects/immune deficiencies e.g. chronic granulomatous disease, Wiskott-Aldrich syndrome,Osteopetrosis, Kostmann's syndrome (congenital neutropenia), Hereditary Lymphohistiocytosis (HLH); Platelets defects e.g.Congenital amegakaryocytic thrombocytopenia; Metabolic/storage disorders e.g. leukodystrophies,mucopolysaccharidoses as Hurler disease;Stem cell defects e.g.reticular agenesis, among many other rare similar conditions. The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood.

Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using different schedules of treatment, in the majority of the 13 clinical trials published between 1990 and 2008.No statistically significant difference was shown between these two interventions during, at most, 18 months of treatment.Three randomised trials that compared sequential DFP-DFO treatment versus DFO alone reported controversial results but this could be due to small sample sizes and short treatment duration. In fact, no trial with treatment duration longer than 18 months15, which reported on mortality, adverse events, serum ferritin concentrations, as well as costs has so far been published. This long-term sequential DFP-DFO treatment versus DFP alone treatment trial was conducted to assess the impacts of these chelation treatments on serum ferritin concentrations, mortality, adverse events, and costs in thalassemia major patients.

To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload and improving the quality of life for thalassemic patient

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.

We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.

This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.

Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).

Primary objectives: To determine the maximum tolerated dose (MTD) of CN128 for single oral administration in thalassemia patients aged 16 and above To study the pharmacokinetics of CN128 in thalassemia patients aged 16 and above Design: The study is designed as a randomized, double-blind, single ascending dose, phase Ia (first in human) trial. The study is consisted of: single dose ascending; dose escalation pharmacokinetics; metabolite structure identification in plasma, urine and feces. Subject inclusion criteria: Thalassemia patients with serum ferritin ≥500 μg/L Patients aged 16 and above Without blood transfusion within 1 week before admission, with hemoglobin ≥ 80 g/L Voluntarily participate in the experiment, and the process of obtaining informed consent form meeting the requirements of GCP Subject exclusion criteria: Hepatitis B surface antigen positive, hepatitis B core antibody positive and HBV-DNA positive, hepatitis C anti-HCV positive, HIV positive, Treponema pallidum positive Patients with history of active digestive tract diseases (including gastric ulcer, duodenal ulcer, gastroesophageal varices, ulcerative colitis, Crohn's disease, digestive tract tumors, familial genetic multiple intestinal polyps), history of digestive tract perforation, history of digestive tract surgery and influence on drug absorption, and other patients whom investigators believe to have potential intestinal complications Liver dysfunction (ALT or AST > 2.5×ULN); or renal dysfunction (serum creatinine > 1.5×ULN) Uncontrolled active infections Patients currently taking CYP3A strong inducer or inhibitor drugs or drugs that may prolong the QT interval without temporary suspension of use or temporary substitution of the said drugs ect. Usage: After fasting for at least 10 hours, the whole tablet was swallowed with 240 mL warm water on an empty stomach. Water was forbidden within 1 hour before and after the administration. Water was allowed 1 hour after administration. Pharmacokinetic assessment of CN128 administration: PK parameters of CN128 include AUC 0-t, AUC 0-∞, Cmax, Tmax, t1/2, CL/F, Vd/F, MRT ,λz etc. Safety and tolerability assessments: Evaluation was based on the incidence of adverse events (AE) after administration, termination information, laboratory test results, 12-lead electrocardiogram and vital signs. Statistics

Patients with transfusion dependent Beta Thalassemia suffer from a high incidence of Hepatitis C infection especially in developed countries as Egypt. In our patients we also found a high correlation between hepatitic C infection and Liver fibrosis. in this study we offer our patients treatment with Direct antiviral drugs and assessed the degree of fibrosis before and after treatment. We tested Hyalornic acid as a predictor of the degree of fibrosis before and after treatment.

It is known that postprandial hyperglycemia increases the cardiometabolic risk in both diabetic and non-diabetic patients. Moreover, there is insufficient data on the effectiveness of exercise on preventing Type II diabetes mellitus in individuals with insulin resistance and prediabetes. This study aims to examine the effectiveness of resistance exercise in limiting postprandial hyperglycemia and the necessity of prescribing medication particularly in patients with beta-thalassemia and insulin resistance.