Clinical trials: Thalassemia, Beta
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
Transfusion-dependent Beta-Thalassemia
Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
This is a prospective, dual-centre, single dose, Phase IIb, single arm, open label study. The proposed clinical trial involves a single infusion of autologous HSPCs genetically modified with the GLOBE lentiviral vector, using an…
- Trial name
- NCT07680803
- Testing
- FT007
- Where
- 2 sites in Italy
- Volunteers
- 9 people
- Sponsor
- Fondazione Telethon
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
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)
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
Oxidative Stress in Neonates, Near Infrared Spectroscopy
Transfusion-Related Changes in Oxidative Stress Biomarkers in Neonates
Reactive oxygen species (ROS), which include peroxides, are generated in the human body as by-products of cellular metabolism. In small amounts, they fulfill important physiological functions.
- Conditions
- Oxidative Stress in Neonates, Near Infrared Spectroscopy, Fetal Hemoglobin, Erythrocyte Transfusion
- Trial name
- NEO-REDOX (NCT07672275)
- Testing
- Peroxide analysis-TOC Omnignostica Forschungs GmbH, Near Infrared Imaging
- Where
- Austria
- Volunteers
- 170 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Beta-Thalassemia, Iron Overload
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients
This is a randomized, open label, two arms superiority trial of a representative population of patients with a primary diagnosis of transfusion dependent thalassemia with evidence of moderate cardiac iron overload, defined as an…
- Trial name
- CANALI (NCT02474420)
- Testing
- Amlodipine, Deferasirox
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- Kevin H.M. Kuo, MD, MSc, FRCPC
Sickle Cell Disease, Sickle B+ Thalassemia
The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.
The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Trial name
- NCT04691323
- Testing
- HU-Go app
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Sickle Cell Disease, Sickle Cell Anemia
Decision Aid for Therapeutic Options In Sickle Cell Disease
Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC
- Trial name
- NCT02326597
- Testing
- Decision Aid Tool, Standard Practice
- Where
- United States
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Emory University
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)
Hemoglobinopathies, Sickle Cell Disease
ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19
Patients with red blood cell disorders (RBCDs), such as Sickle cell disease (SCD) and Thalassemia, are chronic, life-threatening conditions that can become multi-organ complications over time, and are likely at an increased risk…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia, Sickle Beta Thalassemia
- Trial name
- NCT06831799
- Testing
- Non applicable, is a patient registry
- Where
- Spain
- Volunteers
- 684 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Thalassemia Major, Covid19
Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases
In Italy there are about 5000 patients with dependent transfusion thalassemia (source Italian Thalassemia and Hemoglobinopathies - SITE) and a smaller number, currently not definable, of patients with sickle cell anemia in…
- Trial name
- ThalaCoV (NCT04526405)
- Testing
- SARS-CoV-2 S1/S2 IgG
- Where
- Italy
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- University of Milano Bicocca
Beta-Thalassemia Major, Graft-versus-host Disease (GVHD)
Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major
The purpose of this study is to evaluate the basiliximab for prevention of graft-versus-host disease in unrelated allo-genetic hematopoietic stem cell transplantation for thalassemia major.
- Trial name
- NCT02342145
- Testing
- Basiliximab,, Tacrolimus, Methotrexate
- Where
- 3 sites in China
- Volunteers
- 205 people
- Sponsor
- Zhongming Zhang
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
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
Beta Thalassemia Major, Sickle Beta 0 Thalassemia
Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies
This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies).
- Conditions
- Beta Thalassemia Major, Sickle Beta 0 Thalassemia, Sickle Beta Plus Thalassemia, Sickle Beta Thalassemia
- Trial name
- NCT04776850
- Testing
- Bortezomib, Busulfan, Cyclophosphamide
- Sponsor
- M.D. Anderson Cancer Center
Transfusion Dependent Beta-Thalassaemia
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia.
- Trial name
- ET-01 (NCT04925206)
- Testing
- ET-01
- Where
- 4 sites in China
- Volunteers
- 8 people
- Sponsor
- EdiGene (GuangZhou) Inc.
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Transfusion Dependent Beta-Thalassaemia
A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.
This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Trial name
- NCT05752123
- Testing
- ET-01
- Where
- China
- Volunteers
- 2 people
- Sponsor
- EdiGene (GuangZhou) Inc.
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