Clinical trials: Thalassemia, Beta
Thalassemia, Beta
Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients
Objectives Primary objective: To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
- Trial name
- NCT06153784
- Testing
- Hydroxyurea and Thalidomide
- Where
- Pakistan
- Volunteers
- 603 people
- Sponsor
- Children's Hospital Karachi
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
Patients With Thalassemia Intermedia,, Congenital Dyserythropoietic Anemia Type I
GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin…
- Trial name
- GDF 15 (NCT01201135)
- Volunteers
- 80 people
- Sponsor
- Wolfson Medical Center
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Beta-Thalassemia, Thalassemia
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT05356195
- Testing
- CTX001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Thalassemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia.
- Trial name
- BEYOND (NCT03342404)
- Testing
- Luspatercept, Best Supportive Care (BSC)
- Where
- 16 sites in United States, Greece and 4 more
- Volunteers
- 145 people
- Sponsor
- Celgene
Beta-Thalassemia
Iron Status in BTM With Blood Transfusion
To assess the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.
- Trial name
- NCT04947540
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assiut University
Thalassemia Major
Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload…
- Trial name
- NCT05303506
- Testing
- serum ferritin and AST
- Volunteers
- 50 people
- Sponsor
- Assiut University
Liver Fibrosis, Beta Thalassemia Major
Elastography vs Iron Regulators in the Diagnosis of Hepatic Fibrosis- Iron Overload in Children With Beta- Thalassemia
The goal of this cross-sectional observational study is to evaluate the accuracy of serum ferritin and serum hepcidin versus non-invasive scores in the diagnosis of liver fibrosis and iron overload in beta- thalassemia children.
- Trial name
- NCT07153926
- Testing
- Serum ferritin, Serum hepcidin
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Tanta University
Sickle Cell Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Anemia (Iron-Loading), Beta-Thalassemia
Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone
To demonstrate the safety and effectiveness of orally-administered pyridoxal isonicotinoyl hydrazone (PIH) for the chronic treatment of iron overload.
- Conditions
- Anemia (Iron-Loading), Beta-Thalassemia, Hematologic Diseases, Hemoglobinopathies
- Trial name
- NCT00000588
- Testing
- Chelation therapy
- Volunteers
- 120 people
- Sponsor
- Case Western Reserve University
Beta Thalassemia Transfusion Dependent
Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia
Thalassemia is a group of recessively inherited hemoglobin disorders characterized by reduced or no production of hemoglobin and chronic anemia of varying severity.
- Trial name
- NCT07721480
- Testing
- HGI-001 Injection
- Volunteers
- 6 people
- Sponsor
- Prince of Wales Hospital, Shatin, Hong Kong
Thalassemia Major
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic…
- Trial name
- NCT05442346
- Testing
- γ-globin reactivated autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Bioray Laboratories
Transfusion-dependent Beta-Thalassemia
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT05860595
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZE-T (NCT04770779)
- Testing
- Mitapivat
- Where
- 75 sites in United States, Brazil and 17 more
- Volunteers
- 258 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Beta Thalassemia
Genetic Factors Affecting the Severity of Beta Thalassemia
Patients with genetic diseases can have widely differing severities. We are looking for genetic factors which influence the severity of beta thalassemia.
- Trial name
- NCT00159042
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- Deborah Rund
Thalassemia, Tuberculosis
Immunomodulatory Vitamin D in Thalassemia
Thalassemia becomes one of global health issue and so does Indonesia. In 2015, more than 7600 children were diagnosed as this hemoglobin genetic disease wherein anemia and lifetime blood transfusion contribute to their morbidity…
- Conditions
- Thalassemia, Tuberculosis, Vitamin D Deficiency
- Trial name
- ThalTB (NCT03919812)
- Testing
- Cholecalciferol supplementation
- Where
- Indonesia
- Volunteers
- 60 people
- Sponsor
- Universitas Padjadjaran
Congenital Hemolytic Anemia, Sickle Cell Disease
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT).
- Trial name
- NCT00061568
- Testing
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Confirmed Diagnosis of ß-thalassemia Major
ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene
The patient has inherited ß-thalassemia major through the genes. These genes have mistakes in them, so the body cannot make normal red blood cells. Stem cells are made in the bone marrow.
- Trial name
- NCT01639690
- Testing
- Autologous CD34+ cells transduced with TNS9.3.55
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- San Rocco Therapeutics
Beta-Thalassemia
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand).
- Trial name
- NCT02626689
- Testing
- Quality of Life (QOL) questionnaires, Healthcare Resource Utilization
- Where
- 5 sites in Greece, Italy and 3 more
- Volunteers
- 100 people
- Sponsor
- Celgene