Clinical trials: Beta-Thalassemia Major
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
Transfusion Dependent Beta Thalassemia
A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with…
- Trial name
- NCT06678165
- Testing
- YOLT-204
- Where
- China
- Volunteers
- 3 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical 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
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness…
- Trial name
- NCT06664541
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Georgetown University
Thalassemia Major
Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major.
- Trial name
- AnloThal (NCT01125254)
- Testing
- Amlodipine
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- University of Campinas, Brazil
Beta Thalassemia
Genetic Variants Affecting the Clinical Severity of Beta Thalassemia
β-thalassemia is one of the most common single gene disorder in Southern China. The phenotypic severity of beta thalassemia widely varies from mild to severe forms.
- Trial name
- NCT04918056
- Testing
- Hematological Analysis and Genetical Analysis
- Where
- China
- Volunteers
- 1,300 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Sickle Cell Disease
Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own…
- Trial name
- GRASP-EA (NCT07640815)
- Testing
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Where
- United States
- Sponsor
- David Williams
Thalassemia
Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia Intermedia
The definition of Thalassemia Intermedia is not generally accepted and the specific clinical and laboratory characteristics varies between patients.
- Trial name
- NCT01443312
- Testing
- Laboratory analysis.
- Where
- Israel
- Volunteers
- 28 people
- Sponsor
- HaEmek Medical Center, Israel
Beta-Thalassemia, Hepatitis C
Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus
Patients with transfusion dependent Beta Thalassemia suffer from a high incidence of Hepatitis C infection especially in developed countries as Egypt.
- Trial name
- NCT03961828
- Testing
- Ledipasvir 90 MG / Sofosbuvir 400 MG [Harvoni]
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Tanta University
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis
Sickle Cell Disease, Sickle Cell Hemoglobin C
Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among…
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Trial name
- NCT04678037
- Testing
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Anesthesia, Pediatric Cancer
the Efficacy and Safety of Sugammadex in Children 0-2 Years Old
Sugammadex is a selective antidote to muscle relaxants rocuronium bromide and vecuronium bromide. Sugammadex is a modified gamma-cyclodextrin, a compound that selectively binds rocuronium bromide and vecuronium bromide.
- Conditions
- Anesthesia, Pediatric Cancer, Pediatric Tumor, Pediatric Hepatoblastoma
- Trial name
- NCT03728543
- Testing
- Sugammadex
- Where
- Russia
- Volunteers
- 62 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Beta-Thalassemia
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.
- Trial name
- NCT06065189
- Testing
- CS-101 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Children's Hospital of Fudan University
Incentive Spirometer, Pulmonary Function
Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major
The study was done to: 1. Investigate the effect of respiratory training on functional lung capacity and 2. To detect the effect of respiratory training on pulmonary functions in children with β-thalassemia major. 3.
- Conditions
- Incentive Spirometer, Pulmonary Function, Functional Capacity, Child, Only
- Trial name
- NCT07112703
- Testing
- Incentive spirometer exercises, Diaphragmatic breathing, Pursed lip breathing:
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Rawan Mohammed Khairy Mostafa Elsawy
Beta Thalassemia Major Anemia
Thrombin Generation in Beta-thalassemia Major
This study aims to evaluate the persistence of a hypercoagulable state in chronically-transfused patients with beta thalassemia major, by using the thrombin generation test (performed in whole blood and plasma).
- Trial name
- TG-THAL (NCT04962984)
- Testing
- blood sampling
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Central Hospital, Nancy, France
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, 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
Beta-Thalassemia
Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
OBJECTIVES: I. Determine whether arginine butyrate with or without epoetin alfa can stimulate gamma-globin chain production to a degree that decreases anemia and results in hematologic improvement in patients with thalassemia…
- Trial name
- NCT00006136
- Testing
- arginine butyrate, epoetin alfa
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 15 people
- Sponsor
- Boston University
Diabetes, Prediabetes
The Use of CGMS to Detect Alterations of Blood Glucose in Thalassemic Patients
The aim of the study is to: Assess the pattern of glucose homeostasis in patients with B thalassemia . To detect early impairment in glucose metabolism and prediabetic state in B thalassemia patients using continuous glucose…
- Conditions
- Diabetes, Prediabetes, B-Thalassemia
- Trial name
- NCT03591900
- Testing
- insulin pump/CGMS
- Volunteers
- 200 people
- Sponsor
- Ain Shams University
Beta Thalassemia Major
Effect of Spirulina on Liver Fibrosis by Transient Elastography 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
- NCT02744105
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Tanta University