View clinical trials related to Iron Overload.
Filter by:The purpose of this research is to see if a new automated magnetic resonance imaging (MRI) method will be able to improve the images taken of the liver. Participants will have either known or suspected liver disease, known or suspected iron overload syndrome, or be a healthy adult. Participants will be in the research study for one day.
Aim of the work 1. Assessment of hepatic affection in patients with ESRD (end stage renal disease) on regular dialysis with iron indices suggesting iron overload. 2. Comparison between HCV -negative HD patients with high and normal TSAT as regard liver iron concentration(LIC) and degree of fibrosis.
All patients were investigated using a 3T MRI and 1,5 T MRI scanners. For myocardium, pancreas and pituitary gland iron overload quantification in children we have used special sequences for T2*-mapping. Miocardium, pancreas and pituitary gland T2* relaxometry maps were calculated automatically by commertial application ReportCARD Functool (GE Healthcare) and integrated Philips T2* maps. Then for selected ROI T2* data acquisition in milisecond [ms] were performed and calculated automatically.
This study enrolls patients with myelodysplastic syndrome (MDS) and myelofibrosis (MFS), with transfusional iron overload and treats them with the investigational iron chelator, SP-420. SP-420 may be better tolerated and safer than commercially available iron chelators. Iron chelation therapy (ICT) has been shown to improve outcomes in iron overload, but adherence is poor due to problems related to ease of administration, tolerability, and safety.
The overall goal of this project is to develop and validate a novel technique for Magnetic Resonance Imaging (MRI)-based Quantitative Susceptibility Mapping (QSM) of the abdomen, for non-invasive assessment of liver iron deposition. In this work, study team will develop and optimize advanced data acquisition and image reconstruction methods to enable QSM of the abdomen. Further, investigators will determine the accuracy, repeatability, and reproducibility of abdominal QSM for iron quantification in patients with liver iron overload. Excessive accumulation of iron in various organs, including the liver, which affects both adult and pediatric populations, is toxic and requires treatment aimed at reducing body iron stores. Accurate assessment of liver iron concentration is critical for the detection and staging of iron overload as well as for longitudinal monitoring during treatment. In summary, this project will develop a novel MRI-based QSM technique designed for the abdomen and will validate it in pediatric and adult patients with liver iron overload. Upon successful validation, QSM will provide accurate, repeatable, and reproducible quantification of LIC based on a fundamental property of tissue.
1. Primary objectives: • To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above. 2. Design: - The study is designed as a single arm and opened phase IIa clinical trial, so as to investigate the safety and efficacy of CN128. - A total of 50 eligible subjects are planned to be enrolled, and orally administration of CN128 for 24 weeks or 48 weeks according to the administration plan. The treatment period is from day 0 to 24 weeks, and the extended treatment period was from 25 weeks to 96 weeks. - Subjects' medication status, uncomfortable symptoms, concomitant medication or non-drug therapy were recorded daily. 3. Subject inclusion criteria: - Thalassemia patients. - The number of blood transfusion per month ≥1. Or hemoglobin can not be maintained at 90g/L above, if blood transfusions is less than once per month. - Serum ferritin ≥ 500 µg/L - Patients aged 16 and above - Volunteer for the trial and sign the informed consent. 4. Subject exclusion criteria: - Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and alanine transaminase (ALT) beyond normal range) - Active gastrointestinal disease history (including: gastric ulcer, duodenal ulcer, stomach or esophageal varices, ulcerative colitis, Crohn's disease, gastrointestinal cancer, familial genetic multiple intestinal polyps), and History of gastrointestinal perforation, gastrointestinal surgery that influence drug absorption, and other potential intestinal complications considered by researchers; - ALT or Aspartate transaminase (AST) > 2.5 × Upper limit of normal (ULN), or serum creatinine > 1.5 × ULN; - Neutropenia patient (neutrophil count < 1.5 × 109 / L); - Active infection uncontrolled; - The patients who are currently taking CYP3A strong inducer or strong inhibitor drugs, or the drug that may extend the QT interval, or the drug that may decrease neutrophil count, but can not temporarily interrupt the use of such drugs; - Congenital long QT syndrome or known family history of long QT syndrome; QTc > 480 ms; clinically significant ventricular or atrial fast arrhythmia; - The patients who can not accept MRI as detection means, such as claustrophobic for MRI, pacemaker, and those using ferromagnetic metal implants. - Birth planner (including male subjects) within or within 3 months after the end of the trial; - Participated in other clinical trials in the three months before taking the test preparation, except for non-interventional studies; - Pregnant or lactating women; - Unsuitable to participate in the trial considered by the researchers. 5. Usage: - All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan. - All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan. 6. Safety assessments: Safety evaluations include adverse events, adverse reactions, severe adverse events, and severe adverse reactions; growth (weight, height); total and free testosterone in men, follicle-generating hormone and luteinizing hormone in women; vital signs and electrocardiogram; hearing, laboratory tests (blood routine analytes, blood biochemistry, coagulation function, thyroid and para-thyroid function, urine routine analytes.), urine pregnancy test (women of childbearing age),Levels of drug exposure during the study. 7. Efficacy assessments: Efficacy evaluations include serum ferritin, liver iron content (MRI R2) and cardiac iron content (MRI T2*). 8. Statistics: - Subject characteristic distribution Demographic characteristics, general conditions, and baseline conditions (pre-treatment) of enrolled subjects were analyzed.The measurement data are described by means, standard deviation, minimum value and maximum value, while the qualitative data list frequency and percentage. - Safety analysis Descriptive statistical analysis was used for safety endpoints. - Effectiveness analysis Mean, standard deviation, median, minimum and maximum values were described and 95% confidence intervals were calculated. Paired T-test was used to compare each time point with the baseline if necessary. The 95% confidence interval was calculated by using Clopper-Pearson method for the proportion of patients.
A national survey on the prevalence and natural history of endocrine complications in thalassemia transfusion--dependent patients treated with deferasirox was designed, in order to assess a larger population during a longer follow up and improve the quality of previous investigations.
Hematopoietic Stem Cell Transplantation (HSCT) is currently a standard procedure for a wide range of blood-oncological diseases and genetic disorders. Recent improvements in transplant technologies, infection prevention and graft-versus-host-disease (GVHD) management procedures have significantly reduced the transplant-related mortality (TRM). However, approximately 50% of pediatric patients may develop liver dysfunction before HSCT and 74% to 85.5% after HSCT, with a TRM related to liver dysfunction reaching 46%. The liver and pancreas complications still remain too high for the difficulties and diagnostic inefficiencies and, consequently, for the lack of targeted and safer therapies. The diagnostic problems can be summarized in 3 major points: a) the histological examination of liver and pancreas parenchyma cannot be routinely performed because of the organ anatomy and the relative risk of the bioptic procedures; b) the lack of specific biomarkers or advanced imaging techniques appropriate for the diagnosis of HSCT complications; c) the multifactorial causes of organ complications, as well as drug toxicities, GVHD, siderosis, ductopenia (considered as an index of hepatic GVHD), the accumulation of potentially toxic substances favored by siderosis and ductopenia. In more than 50% of HSCT patients, siderosis and/or ductopenia may represent common pathological conditions. Furthermore, international guidelines issued by onco-hematology and transplantation scientific societies recommend a chelating treatment with deferasirox in all hematological and oncological patients undergoing an intense transfusion regimen. However, in the presence of siderosis and marked ductopenia, patients receiving deferasirox may experience both severe renal and hematological toxicities and lack of effectiveness of the chelating treatment. Therefore, the principal aim of the present retrospective study will be the evaluation of the transplant-related mortality (TRM) in patients requiring a chelation treatment according to the Italian guidelines in pediatric patients
Iron demand: The average daily demand to fit the cell biological metabolism is balanced between intake and lost which about 1-2 mg.
Iron demand: The average daily demand to fit the cell biological metabolism is balanced between intake and lost which about 1-2 mg.