Clinical trials: Beta-Thalassemia Major
Iron Overload, Beta-Thalassemia
SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
The purpose of this study is to test the safety and tolerability of SP-420 and it's efficacy in terms of lowering iron in subjects with Beta-thalassemia or other rare anemias who need regular blood transfusions.
- Trial name
- NCT03801889
- Testing
- SP-420
- Where
- 4 sites in Canada, Lebanon and 2 more
- Sponsor
- Abfero Pharmaceuticals, Inc
Malaria, Severe Malaria
Examination of Protective Factors Against Severe Malaria
This study, sponsored by the National Institutes of Health and the University of Bamako in Mali, Africa, will examine factors that may protect against progression of malaria from mild to severe disease.
- Trial name
- NCT00342043
- Where
- Mali
- Volunteers
- 4,850 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Beta-Thalassemia
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961),No investigational drug product will be administered in the study.
- Trial name
- NCT06717932
- Where
- China
- Volunteers
- 8 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Beta-thalassemia Major, Sickle Cell Disease
Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload
Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: The…
- Conditions
- Beta-thalassemia Major, Sickle Cell Disease, Iron Hemosiderosis
- Trial name
- NCT01511848
- Testing
- DFP (ferriprox) and deferasirox (ICL 670), DFP, DFO
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Ain Shams University
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0…
- Trial name
- NCT02906202
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Genetix Biotherapeutics Inc.
Beta Thalassemia Major
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
the aim of this study is to evaluate the effect of Spirulina compared to Amlodipine on cardiac iron overload and cardiac functions in multi-transfused children with beta thalassemia major
- Trial name
- NCT02671695
- Testing
- Spirulina, Amlodipine
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Tanta University
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Hemoglobin SC Disease, Sickle Cell Anemia
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders
Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation…
- Trial name
- NCT00005783
- Testing
- Recombinant-methionyl human stem cell factor
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Hemoglobinopathies, Anemia, Sickle Cell
Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia
The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high…
- Conditions
- Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia
- Trial name
- NCT00034528
- Testing
- Busulfan, Fludarabine, FK506
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Background: Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs.
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Trial name
- NCT02105766
- Testing
- Alemtuzumab, Sirolimus, Cyclophosphamide
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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 Major
Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients.
- Trial name
- NCT03992001
- Testing
- Blood component A, Blood component B
- Where
- Italy
- Volunteers
- 55 people
- Sponsor
- Università degli Studi di Ferrara