Clinical trials: Genetic Hemochromatosis
Iron Overload, Excessive Body Iron Burden
Massive Iron Deposit Assessment
Iron overload is a severe complication of multiple blood transfusions. As the body has no physiologic mechanism for clearing iron, repeated transfusions cause iron accumulation in organs and lead to iron toxicity.
- Trial name
- NCT01572922
- Testing
- R2*-UTE, R2*-GRE, Liver biopsy
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- St. Jude Children's Research Hospital
Diabetes Mellitus, Heart Disease
Deferoxamine for the Treatment of Hemochromatosis
When patients receive repeated blood transfusions the level of iron in the patient s blood can rise. When iron is processed in the body a protein known as hemosiderin can begin collecting in the organs.
- Conditions
- Diabetes Mellitus, Heart Disease, Hemochromatosis, Thalassemia
- Trial name
- NCT00001203
- Where
- United States
- Volunteers
- 151 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
- Trial name
- NCT01090323
- Testing
- ICL670
- Where
- 39 sites in United States, Canada and 3 more
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Hemochromatosis, Iron Overload
MRI QSM Imaging for Iron Overload
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…
- Trial name
- NCT04631718
- Testing
- Magnetic Resonance Imaging (MRI)-based Quantitative Susceptibility Mapping (QSM)
- Where
- 2 sites in United States
- Volunteers
- 63 people
- Sponsor
- University of Wisconsin, Madison
Beta-Thalassemia, Hemosiderosis
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions.
- Trial name
- NCT00171171
- Testing
- deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 252 people
- Sponsor
- Novartis
Transfusional Iron Overload
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional…
- Trial name
- NCT01044186
- Testing
- ICL670
- Where
- 18 sites in United States, Greece and 1 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Genetic/Metabolic Liver Diseases
China Registry for Genetic / Metabolic Liver Diseases
CR-GMLD registry started on June 13, 2015 to collect cases of genetic/metabolic liver diseases from tertiary or secondary hospitals in mainland China.
- Trial name
- NCT03131427
- Testing
- Standard of care
- Where
- 12 sites in China
- Volunteers
- 20,000 people
- Sponsor
- Beijing Friendship Hospital
Hemochromatosis, Cerebrovascular Accident
Stroke With Transfusions Changing to Hydroxyurea
The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with…
- Conditions
- Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
- Trial name
- NCT00122980
- Testing
- Red Cell Transfusions, Iron Chelation, Hydroxyurea
- Where
- 28 sites in United States
- Volunteers
- 134 people
- Sponsor
- St. Jude Children's Research Hospital
Glomerulonephritis, Membranous, Neonatal Diseases and Abnormalities
Research Network for Neonatal Diseases Induced by Tissular Fetomaternal Alloimmunization
Problems of compatibility between a mother and her child are frequent. The most well-known case can be illustrated by the fetomaternal blood group incompatibility (rhesus factor) which can induce severe anemia of the fetus.
- Conditions
- Glomerulonephritis, Membranous, Neonatal Diseases and Abnormalities, Hemochromatosis
- Trial name
- NCT00199628
- Where
- France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Limoges
Beta-thalassemia Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
Hemochromatosis
Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients
Comparison of efficacy and toxicity of the combination treatment of deferiprone and desferrioxamine with the single agent treatment of either drug
- Trial name
- NCT00350662
- Testing
- Deferiprone (L1), Desferrioxamine
- Where
- 2 sites in Egypt, Turkey (Türkiye)
- Volunteers
- 95 people
- Sponsor
- Lipomed
Blood Disease, Hemochromatosis
Hemochromatosis--Genetic Prevalence and Penetrance
To examine the cost effectiveness of hereditary hemochromatosis (HH) screening in primary care.
- Trial name
- NCT00006312
- Sponsor
- University of Rochester
Iron Overload
Iron Overload in Stem Cell Transplant Recipients
The goal of this study is to examine the impact of iron overload in patients undergoing a bone marrow transplant. We believe that the iron status in these patients is associated with complications for transplant survivors.
- Trial name
- NCT00806715
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Boston Children's Hospital
Hemochromatosis
Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent Patients
Systematical (retro- and prospective) investigation of the long-term safety (toxicity assessment according to CTCAE v3.0) and efficacy of deferiprone either given alone or in combination with desferrioxamine
- Trial name
- NCT00349453
- Testing
- Deferiprone (L1), Desferrioxamine
- Where
- 13 sites in Switzerland
- Volunteers
- 24 people
- Sponsor
- Lipomed
Hemochromatosis, Iron Overload
Iron Overload in African Americans
Iron overload (hemochromatosis) is a condition which causes the intestines to take too much iron into the body from food or pills. The extra iron can build up in the liver, heart, joints, pancreas, sex organs, and other organs.
- Trial name
- NCT00001455
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Thalassemia, Brain Function (Measured by ERP Level)
A Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients.
β-thalassemia syndromes are a group of hereditary disorders characterized by a genetic deficiency in the synthesis of beta-globin chains.
- Trial name
- NCT01935661
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease
Liver Fibrosis in Sickle Cell Disease
Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions.
- Trial name
- NCT02007746
- Testing
- Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- University of Miami
Iron Overload, Hemochromatosis
Confounder-Corrected Quantitative MRI Biomarker of Hepatic Iron Content
The purpose of this multi-site research is to validate a rapid magnetic resonance based confounder-corrected R-2 mapping method as a quantitative imaging biomarker of liver iron concentrations.
- Conditions
- Iron Overload, Hemochromatosis, Hemosiderosis
- Trial name
- NCT02025543
- Testing
- MRI
- Where
- 4 sites in United States
- Volunteers
- 207 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison
Transfusional Iron Overload, Transfusional Hemosiderosis
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions. The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a…
- Trial name
- CORDELIA (NCT00600938)
- Testing
- Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox
- Where
- 21 sites in Canada, China and 9 more
- Volunteers
- 197 people
- Sponsor
- Novartis Pharmaceuticals
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals