Clinical trials: B Thalassemia, open to volunteers
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who…
- Trial name
- NCT05991336
- Testing
- Gene therapy
- Where
- China
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Beta-Thalassemia
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s).
- Trial name
- NCT03351829
- Testing
- Gene-modified autologous hematopoeitic stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Transfusion-dependent Beta-Thalassemia
β-Thalassemia Treatment With KL003 Cell Injection
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with…
- Trial name
- NCT06280378
- Testing
- KL003 Cell Injection Drug Product
- Where
- 2 sites in China
- Volunteers
- 41 people
- Sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd.
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids-T (NCT07506863)
- Testing
- Mitapivat
- Where
- 4 sites in United States
- Volunteers
- 54 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
- Trial name
- NCT05777993
- Testing
- Mitapivat
- Where
- 23 sites in Belgium, Brazil and 6 more
- Volunteers
- 55 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids (NCT07517133)
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 45 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
- Trial name
- NCT06479616
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Fudan University
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Beta-Thalassemia, Thalassemia
Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia
This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone.
- Trial name
- NCT07884773
- Testing
- Thalidomide, Danazol, Prednisolone
- Where
- Pakistan
- Volunteers
- 172 people
- Sponsor
- Khyber Medical University Peshawar
Hemolytic Anemia, Thalassemia
Native T1 Mapping and ECV in Chronic Hemolytic Anemia
This cross-sectional study aims to evaluate the diagnostic performance of native T1 mapping and extracellular volume fraction for the detection of myocardial iron overload and fibrosis in patients with chronic hemolytic anemia…
- Conditions
- Hemolytic Anemia, Thalassemia, Iron Overload
- Trial name
- NCT07882303
- Volunteers
- 54 people
- Sponsor
- Assiut University
Sickle Cell Disease, Beta-thalassemia
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions.
- Trial name
- NCT07599176
- Testing
- Total Body Irradiation, Alemtuzumab, Abatacept
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Beta Thalassemia Transfusion Dependent
Evaluation of Physiological and Psychological Factors Involved in Exercise Intolerance in Patients With β-TTD
The goal of this clinical trial is to compare physical ability of patients with transfusion-dependent β-thalassemia to control subjects.
- Trial name
- THALEX (NCT07207577)
- Testing
- Maximal incremental exercise test
- Where
- France
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Metropole Savoie
B Thalassemia Major
Oral Health Status in Children With β-Thalassemia Major
To evaluate the effectiveness of a structured oral health educational program on oral hygiene status and oral health-related quality of life (OHRQoL) among children diagnosed with β-thalassemia.
- Trial name
- NCT07614217
- Testing
- oral health educational program for Immediate Intervention Group, routine oral hygiene instructions
- Volunteers
- 46 people
- Sponsor
- Cairo University
Alpha and Beta Thalassemia, Sickle Cell Disease
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Background: Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders.
- Conditions
- Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
- Trial name
- NCT03937817
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sickle Cell Disease, Thalassemia
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemoglobinopathy
- Trial name
- NCT06313398
- Testing
- Biotin-labeled red blood cells
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Alpha-Thalassemia, Alpha Thalassemia Major
International Registry of Patients With Alpha Thalassemia
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of…
- Conditions
- Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
- Trial name
- ATM Registry (NCT04872179)
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- University of California, San Francisco
Thalassemia Particularly Transfusion Dependent Thalassemia
Clinical, Economic,and Psychosocial Burden of Thalassemia in Sohag
This study aims to assess the clinical, economic, and psychosocial burden of thalassemia among affected children in Sohag. Clinically, the study will evaluate disease severity, transfusion requirements, iron overload…
- Trial name
- HBT-sohag (NCT07796022)
- Volunteers
- 100 people
- Sponsor
- Randa Elham Hamam
β-thalassemia
A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
- Trial name
- NCT07215975
- Testing
- Luspatercept
- Where
- 13 sites in Kuwait, Oman and 2 more
- Volunteers
- 200 people
- Sponsor
- Bristol-Myers Squibb
Hematologic Disorder, Bleeding Disorder
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly.
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia
- Trial name
- NCT04398628
- Where
- 76 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- American Thrombosis and Hemostasis Network