Clinical trials: Beta-Thalassemia Major
Beta-Thalassemia, Insulin Resistance
Resistance Exercise on Postprandial Hyperglycemia in Patients With B-thalassemia Exhibiting Resistance to Insulin
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…
- Conditions
- Beta-Thalassemia, Insulin Resistance, PreDiabetes, Diabetes Mellitus, Type 2
- Trial name
- NCT03889977
- Testing
- Resistance exercise
- Where
- Greece
- Volunteers
- 6 people
- Sponsor
- University of Thessaly
Thalassemia
Endocrine and Nutritional Assessment in B Thalassemia Major
B Thalassemia patients developed short stature and low weight in spite no evident endocrine abnormalities. One hypothesis is that they developed some degree of malnutrition,then the purpose of this study is to assess the…
- Trial name
- NCT00456690
- Testing
- Nutritional recommendations
- Where
- Israel
- Volunteers
- 133 people
- Sponsor
- HaEmek Medical Center, Israel
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
This study is researching an experimental drug called REGN7999 (called "study drug"). The study is focused on patients with non-transfusion dependent beta-thalassemia.
- Trial name
- FERVENT-1 (NCT06421636)
- Testing
- REGN7999
- Where
- 26 sites in United States, Georgia and 7 more
- Volunteers
- 19 people
- Sponsor
- Regeneron Pharmaceuticals
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Transfusion Dependent Beta Thalassemia
Glucagon-Like Peptide 1 Receptor Agonist in Diabetes Mellitus Management in Children and Adolescents With Transfusion-Dependent Thalassemia
Blood transfusion and iron-chelation therapy have prolonged and improved the quality of life in patients with β-thalassemia. The improvement was mainly due to the decrease in mortality from heart failure Such a treatment…
- Trial name
- NCT07370922
- Testing
- Dulaglutide 0.75Mg/0.5Ml Inj Pen, control group (insulin)
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
Sickle Cell Disease
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor.
- Trial name
- LBH589 (NCT01245179)
- Testing
- panobinostat
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Abdullah Kutlar
Beta Thalassemia Major
Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified
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…
- Trial name
- NCT03276455
- Testing
- Autologous CD34+ cells genetically modified
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Nanfang Hospital, Southern Medical University
β-thalassemia, Ineffective Erythropoiesis
Safety Study for Beta Thalassemia Subjects on PTG-300
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
- Trial name
- TRANSCEND (NCT04054921)
- Testing
- PTG-300
- Where
- 16 sites in United States, Greece and 6 more
- Volunteers
- 34 people
- Sponsor
- Protagonist Therapeutics, Inc.
Beta-Thalassemia
CS-101 in Patients With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06328764
- Testing
- CS-101
- Where
- China
- Volunteers
- 10 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Osteoporosis, Thalassemia Major
Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TI
An Italian Multicentric randomized, open-label therapeutic trial evaluating the efficacy and safety of Neridronate in the treatment of Osteoporosis in patients with Thalassemia Major and Severe Thalassemia Intermedia.
- Conditions
- Osteoporosis, Thalassemia Major, Thalassemia Intermedia
- Trial name
- Nerixia (NCT01140321)
- Testing
- Neridronate
- Where
- 8 sites in Italy
- Volunteers
- 120 people
- Sponsor
- Ente Ospedaliero Ospedali Galliera
Beta Thalassemia Major, Beta Thalassemia Intermedia
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Trial name
- NCT01571635
- Testing
- SOTATERCEPT (ACE-011)
- Where
- 18 sites in France, Greece and 2 more
- Volunteers
- 46 people
- Sponsor
- Celgene
Microcytic Hypochromic Anemia
Red Cell Distribution Width Index Versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .
Red Cell Distribution Width Index versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .
- Trial name
- NCT03868306
- Testing
- CBC , Iron study and Haemoglobin electrophoresis .
- Volunteers
- 100 people
- Sponsor
- Assiut University
Beta Thalassemia Major Anemia
The Potential Hepatoprotective Effect of Metformin in Patients With Beta Thalassemia Major
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…
- Trial name
- NCT02984475
- Testing
- Metformin
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Cairo University
B Thalassemia
Effect of Metoprolol on Thalassemia Cardiomyopathy
effect of B blocker was first evaluated in patient with cardiomyopathy not induced by ischemia and idiopathic which as the most common causes of cardiomyopathy.
- Trial name
- NCT01863173
- Testing
- metoprolol
- Where
- Iran
- Volunteers
- 45 people
- Sponsor
- Shiraz University of Medical Sciences
Thalassemia
Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E Patients
The purpose of this study is to determine the efficacy of antioxidant cocktails on the alleviation of oxidative stress and iron overload in beta-thalassemia/Hb E patients.
- Trial name
- NCT01597765
- Testing
- curcuminoids, N-acetylcysteine, deferiprone and vitamin E, Curcuminoids and alpha-tocopherol
- Where
- Thailand
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Mahidol University
Thalassemia
The Role of OCTA in Patients Affected by Beta Thalassemia
To investigate, using optical coherence tomography angiography (OCTA), the retinal and choriocapillaris vascular features in patients affected by Beta Thalassemia.
- Trial name
- NCT04582110
- Testing
- Optical coherence tomography angiography
- Where
- Italy
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- Federico II University
Endocrine Disease, Fertility Decreased Male
Sperm DNA Damage in β-thalassemia Major: Is There a Role for Antioxidants?
Accumulation of iron in patients with beta thalassemia major causes free radical formation which leads to damage of biological membranes. Sperm DNA damage may result from these generated antioxidants.
- Conditions
- Endocrine Disease, Fertility Decreased Male, DNA Damage
- Trial name
- NCT03014882
- Testing
- L-Carnitine and N-acetyl cysteine
- Where
- 3 sites in Egypt
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Ain Shams University
Thalassemia, Beta
Long-term Follow-up Study of BRL-101 for TDT
Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.
- Trial name
- NCT06298630
- Testing
- Assessments
- Where
- 7 sites in China
- Volunteers
- 45 people
- Sponsor
- Bioray Laboratories
Sickle Cell Disease, Thalassemia, Beta
Study of the Role of Genetic Modifiers in Hemoglobinopathies
This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia Alpha, Hemoglobinopathies
- Trial name
- INHERENT (NCT05799118)
- Testing
- GWAS
- Where
- 26 sites in United States, Angola and 14 more
- Volunteers
- 30,000 people
- Sponsor
- Cyprus Institute of Neurology and Genetics
β-thalassemia Major
An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.
- Trial name
- MACS2163 (NCT01724138)
- Testing
- Deferasirox
- Sponsor
- Novartis Pharmaceuticals