Clinical trials: Hereditary Haemochromatosis
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
Beta-Thalassemia
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood…
- Trial name
- NCT00061750
- Testing
- ICL670, deferoxamine
- Where
- 7 sites in United States
- Volunteers
- 595 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Liver Disease
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Hepatic steatosis may cause inflammation and fibrosis within the liver potentially leading to end-stage liver disease cirrhosis, liver failure and death.
- Trial name
- FALL (NCT05335603)
- Where
- Denmark
- Volunteers
- 335 people, healthy volunteers welcome
- Sponsor
- University of Copenhagen
Hemochromatosis, Type 4, Ferroportin Disease
Analysis of the Modulation of Serum Hepcidin Level in Response to Iron Oral Intake: Potential Interest for the Differential Diagnosis Between Ferroportin Disease and Dysmetabolic Hepatosiderosis.
The diagnosis of iron overload is a common problem. It is important to optimize the diagnosis to ensure support for patients and their relatives especially regarding genetic disease.
- Conditions
- Hemochromatosis, Type 4, Ferroportin Disease, Dysmetabolic Hepatosiderosis, Diagnosis
- Trial name
- NCT01949467
- Testing
- iron fumarate
- Where
- 4 sites in France
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Rennes University Hospital
Thalassemia, Iron Overload
Oral Nifedipine to Treat Iron Overload
This study will determine if nifedipine, a medication used to treat high blood pressure, can help treat iron overload, a condition in which the body contains too much iron.
- Conditions
- Thalassemia, Iron Overload, Hemochromatosis
- Trial name
- NCT00712738
- Testing
- Nifedipine, Phlebotomy, Urinalysis
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Down Syndrome
Infectious and Non-infectious Lower Respiratory Diseases in Children With Down Syndrome Followed in Pediatric Pulmonology Consultations in Ile-de-France
The purpose of this study is to describe infectious and non-infectious lower respiratory tract diseases in children with Down syndrome followed in pediatric pulmonology consultations in Ile de France.
- Trial name
- ARBT21 (NCT06336577)
- Testing
- lower respiratory tract disease
- Where
- France
- Volunteers
- 108 people
- Sponsor
- Centre Hospitalier Sud Francilien
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary…
- Trial name
- NCT07332091
- Testing
- Vamifeport
- Where
- 100 sites in United States, Australia and 16 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
Haemochromatosis
Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
Patients in maintenance treatment for HFE hemochromatosis since at least one year will be included in a two year study period and randomized in two groups experimental and control group.
- Trial name
- Quali-SAT (NCT04779593)
- Testing
- Clinical examination, SF36 questionnaire, AIMS2_SF questionnaire
- Where
- 12 sites in France
- Volunteers
- 239 people
- Sponsor
- Rennes University Hospital
Refractory Hemochromatosis Rheumatism
Treatment of Refractory Hemochromatosis Rheumatism by Anakinra: a Preliminary Phase II Study
Treatment of refractory hemochromatosis rheumatism by Anakinra. Prospective, multicenter, non-randomised, single-arm, open-label, phase II trial.
- Trial name
- THERA (NCT02263638)
- Testing
- Anakinra
- Where
- 3 sites in France
- Volunteers
- 17 people
- Sponsor
- Rennes University Hospital
Hereditary Hemochromatosis, Hyperferritinemia
Quality and Biologic Characteristics of Red Blood Concentrates Obtained From Individuals With Elevated Ferritin.
Iron overload in hereditary hemochromatosis (HH) is treated by phlebotomy. It is unclear, if individuals with hyperferritinemia due to hereditary hemochromatosis or to secondary causes are suitable as blood donors.
- Trial name
- NCT05742035
- Testing
- Venipuncture
- Where
- 2 sites in Switzerland
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Interregionale Blutspende SRK
Hemochromatosis
Evaluation of a New MR Pulse Sequence to Quantify Liver Iron Concentration
The purpose of this study is to determine if a new MR pulse sequence is accurate in determining how much iron is in the liver.
- Trial name
- NCT00587535
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
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
Hemochromatosis
Erythrocyte Apheresis Versus Phlebotomy in Hemochromatosis
Primary hemochromatosis is the most frequent hereditary condition in Scandinavia. The condition may result in serious organ damage which can be prevented by therapy, but only few patients develop such organ damage.
- Trial name
- NCT00509652
- Testing
- Arm 1: Erythrocyte apheresis, Arm 2: Whole blood phlebotomy
- Where
- 3 sites in Norway
- Volunteers
- 67 people
- Sponsor
- University of Bergen
Hemochromatosis
Study of the Cellular Diffusion of Tacrolimus Across the Membrane of Mononuclear Cells
Pharmacokinetics of tacrolimus are highly variable and may result in graft rejection (underdosing) or toxicity (overdosing). The risk of transplant rejection and the toxicity of tacrolimus can be reduced by pharmacological…
- Trial name
- DIFF-TAC (NCT03654794)
- Testing
- Cell pharmacokinetics of tacrolimus
- Where
- France
- Volunteers
- 26 people
- Sponsor
- Rennes University Hospital
Iron Deficiency Anemia of Pregnancy, Iron Storage Disease
Analysis of the Effect of Iron Supplements on Iron Deficiency Anemia in Pregnancy
Subjects were tested for hemoglobin, ferritin, serum iron, transferrin saturation and reticulocyte count during routine prenatal examination at 24-26 weeks of gestation, and blood samples were taken for serum hepcidin detection…
- Trial name
- NCT05804071
- Testing
- Niferex
- Where
- China
- Volunteers
- 452 people, healthy volunteers welcome
- Sponsor
- Qianfoshan Hospital
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
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
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
Anemia (Iron-Loading), Beta-Thalassemia
Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
To determine whether deferoxamine prevented the complications of transfusional iron overload.
- Conditions
- Anemia (Iron-Loading), Beta-Thalassemia, Hematologic Diseases, Hemoglobinopathies
- Trial name
- NCT00000595
- Testing
- deferoxamine
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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