Clinical trials: Thalassemia
Thalassemia (Transfusion Delendent)
Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
The CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the…
- Trial name
- CENTAurus (NCT02069886)
- Testing
- deferasirox
- Sponsor
- Novartis Pharmaceuticals
Transfusional Iron Overload, Iron Overload
Treatment of Iron Overload Requiring Chelation Therapy
The purpose of this open-label study is to assess liver iron concentration using MRI imaging in subjects with beta-thalassemia when administered with either SPD602 or deferasirox for the treatment of chronic transfusional iron…
- Conditions
- Transfusional Iron Overload, Iron Overload, Iron Chelation, Beta-thalassemia
- Trial name
- NCT01927913
- Testing
- SPD602, Deferasirox
- Sponsor
- Shire
Transfusion-dependent Thalassemia, Non-transfusion-dependent Thalassemia
Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet
Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox dispersible tablet (DT) in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by…
- Trial name
- Jupiter (NCT02993224)
- Testing
- Deferasirox dispersable tablet (DT), Deferasirox film coated tablet (FCT)
- Where
- 17 sites in Egypt, Lebanon and 5 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Healthy Volunteers Sickle Cell Disease, Thalassemia
A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants
The study aims to test if a new medicine called etavopivat potentially affects other medicines in healthy participants. The purpose of the study is to investigate whether the use of etavopivat affects the breakdown and metabolism…
- Trial name
- NCT06813924
- Testing
- Etavopivat, Digoxin, Pitavastatin
- Where
- United States
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
- Trial name
- NCT05693909
- Testing
- SP-420
- Where
- Denmark
- Volunteers
- 90 people
- Sponsor
- Pharmacosmos A/S
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Anemia, Pulmonary Hypertension
Heart Disease in Sickle Cell Anemia
This study will explore what may cause people with sickle cell anemia to have heart problems and an increased risk of sudden death. People 18 years of age and older with sickle cell anemia may be eligible for this study.
- Trial name
- NCT00113152
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Sickle Cell Disease, Sickle Cell-Beta0-Thalassemia
Isoquercetin in Sickle Cell Anemia
This research study is being done to assess the safety and effectiveness of isoquercetin to reduce levels of soluble P-Selectin in patients with sickle cell disease. Isoquercetin is a naturally occurring flavonoid-or vitamin.
- Trial name
- NCT04474626
- Testing
- Isoquercetin
- Volunteers
- healthy volunteers welcome
- Sponsor
- Jeffrey Zwicker, MD
Sickle Cell Disease, Beta Thalassemia
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease.
- Trial name
- NCT00408447
- Testing
- Busulfan, Fludarabine, Alemtuzumab
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Columbia University
Sickle Cell Disease, Beta Thalassemia Intermedia
Pumilio1 (PUM1) Expression, Sickle Cell Anemia, β-thalassemia Intermedia
1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3.
- Trial name
- NCT05883254
- Testing
- RNA Binding Protein Pumilio1 (PUM1) Expression by reverse transcriptase quantatative PCR
- Volunteers
- 80 people
- Sponsor
- Assiut University
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
Beta Thalassemia
Effects of Spirulina on Cardiac Functions in Children With Beta Thalassemia Major
The aim of this study is to evaluate the cardioprotective effect of spirulina in children with beta thalassemia.
- Trial name
- NCT02597595
- Testing
- spirulina
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Tanta University
Thalassemia, Sickle Cell Anemia
Blood Transfusions in Thalassemia Patients, Complications and Adverse Effects
Patients suffering from Thalassemia or another hemoglobinopathies required regular blood transfusions. The complications and adverse effects of blood transfusions can be classified as immediate and late.
- Trial name
- NCT00971880
- Testing
- Clinical medical record summary
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- HaEmek Medical Center, Israel
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
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
Beta-Thalassemia
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with…
- Trial name
- NCT03381833
- Testing
- LJPC-401
- Where
- 22 sites in United States, Australia and 6 more
- Volunteers
- 84 people
- Sponsor
- La Jolla Pharmaceutical Company
Thalassemia, Iron Overload
Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with…
- Trial name
- NCT00901199
- Testing
- Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Elliott Vichinsky
B Thalassemia
Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia
to evaluate thyroid function ,HbA1C,\&lipid profile in thalassemic patient to correlate thyroid function , Hba1c , lipid profile e ferritin level in thalassemic patient
- Trial name
- NCT06377865
- Volunteers
- 72 people
- Sponsor
- Assiut University
Thalassemia
Pharmacotherapy Prescribing Pattern and the Adherence Level to Iron Chelation Therapy in Thalassemia
Thalassemias are a heterogeneous grouping of genetic disorders that result in dysfunctional Hb, reduced RBC life span leading to chronic anemia . Thalassemia is endemic in the Middle East.
- Trial name
- NCT06210139
- Where
- Iraq
- Volunteers
- 199 people
- Sponsor
- Reem Ali Shaker
Beta Thalassemia Major
Effect of Spirulina on Liver Iron Concentration in Beta Thalassemic Children With Hepatitis C
Thalassemics can develop liver fibrosis because of iron overload and hepatitis C infection. The latter is the main risk factor for liver fibrosis in transfusion dependent thalassemics.
- Trial name
- NCT02744560
- Testing
- spirulina
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Tanta University