Clinical trials: Beta Thalassemia Major
Beta-Thalassemia, Thalassemia
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD).
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn
- Trial name
- NCT05477563
- Testing
- CTX001
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Iron Overload Due to Repeated Red Blood Cell Transfusions, Thalassemia Majors (Beta-Thalassemia Major)
Evaluate the Efficacy and Safety of Irochel for the Management of Iron Overload in Thalassemia Patients in Comparison With Deferoxamine
This research study was an experimental, randomized, controlled clinical trial to evaluate the safety and efficacy of coded Unani formulation Irochel for the management of IOL due to repetitive blood transfusion in comparison…
- Trial name
- NCT07782931
- Testing
- Irochel, Deferoxamine
- Where
- Pakistan
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- Hamdard University
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Preterm Birth, Oxygen Toxicity
Fetal Haemoglobin and Cerebral and Peripheral Oxygenation.
The aim of this study is to investigate the relationship between cerebral and peripheral oxygenation and oxygen extraction, as measured by NIRS (near-infrared spectroscopy ), and the FHbF (fraction of fetal hemoglobin) and…
- Conditions
- Preterm Birth, Oxygen Toxicity, Fetal Hemoglobin
- Trial name
- HbFIN (NCT04802629)
- Testing
- Near-infrared spectroscopy
- Where
- Austria
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Myelodysplastic Syndromes, Beta-thalassemia
A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia
The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic…
- Trial name
- NCT07681440
- Testing
- Luspatercept
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Bristol-Myers Squibb
Beta Thalassemia Major, Congenital Anemias
Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
Betathalassemia major is a disease of the blood and bone marrow. You were born with it and it has made you unable to make normal hemoglobin and red cells. You have been receiving red blood cell transfusions all your life.
- Trial name
- NCT00658385
- Testing
- GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Beta Thalassemia
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia
- Trial name
- NCT00790127
- Testing
- HQK-1001
- Where
- 2 sites in Lebanon, Thailand
- Volunteers
- 21 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Beta-thalassemia Major
Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients
Background: Three iron chelators now available on the market differ in toxicity and organ specificity; evidence on standardized chelation protocol remains inconclusive, but patients with transfusion-dependent beta-thalassemia…
- Trial name
- NCT02198508
- Testing
- DFX(Deferasirox), DFP(Deferiprone)
- Volunteers
- 13 people
- Sponsor
- China Medical University Hospital
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Beta Thalassemia Intermedia, Sickle Cell Disease
The BENeFiTS Trial in Beta Thalassemia Intermedia
Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases…
- Trial name
- PB04-001 (NCT04432623)
- Testing
- Benserazide Only Product
- Where
- 5 sites in United States, Canada
- Volunteers
- 36 people
- Sponsor
- Phoenicia BioScience
β-thalassemia Major
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector…
- Trial name
- NCT05776173
- Testing
- BD211
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Thalassemia Major
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.
- Trial name
- NCT03171831
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- China
- Volunteers
- 30 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Thalassemia Major
Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform.
- Trial name
- NCT02126046
- Testing
- unrelated CB following haplo-identical hematopoietic stem cells transplantation
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Nanfang Hospital, Southern Medical University
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
EKLF Mutations in Beta Thalassemia Patients
Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients
Detection of KLF1 gene mutations in patients with beta thalassemia considering the alpha and beta molecular status of these patients.
- Trial name
- NCT06433570
- Testing
- multiplex PCR
- Volunteers
- 100 people
- Sponsor
- Assiut University
Beta-Thalassemia
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells.
- Trial name
- NCT06364774
- Testing
- ALS20
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Thalassemia Major, Transfusion-dependent Anemia
The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron…
- Trial name
- NCT05462548
- Testing
- Luspatercept Injectable Product
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Sun Yat-sen University
Beta Thalassemia Intermedia
Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon
Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body.
- Trial name
- LB-04-THAL (NCT01642758)
- Testing
- Sodium 2,2 dimethylbutyrate
- Where
- Lebanon
- Volunteers
- 10 people
- Sponsor
- Boston University