Clinical trials: Thalassemia Intermedia
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
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
Thalassemia
L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia
- Trial name
- NCT03402191
- Testing
- L-arginine, Sildenafil
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Sherief Abd-Elsalam
Transfusion-dependent Beta-Thalassemia
Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients
The goal of this Non-Randomized Clinical Trial is to determine the effects of thalidomide on red blood cells in transfusion dependent beta thalassemia patients.
- Trial name
- NCT06146478
- Testing
- Thalidomide
- Where
- Pakistan
- Volunteers
- 200 people
- Sponsor
- Blood Care Clinic
Body Composition in Thalassemia Major
Body Composition in Pediatric Patients With Thalassemia Major
To assess body composition (fat mass, lean mass, and bone mass) in pediatric patients with beta thalassemia major using appropriate tools (e.g.,Bioelectrical impedance analysis (BIA)).
- Trial name
- BC-THAL (NCT07204015)
- Volunteers
- 115 people
- Sponsor
- Assiut University
Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome
This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c.
- Conditions
- Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome, Very-Low Risk Myelodysplastic Syndrome
- Trial name
- NCT04718844
- Testing
- SLN124
- Where
- 20 sites in Germany, Israel and 5 more
- Volunteers
- 44 people
- Sponsor
- Silence Therapeutics plc