Clinical trials: Beta-thalassemia
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
Thalassemia Majors (Beta-Thalassemia Major)
Gonadal Function in Thalassemic Patient
Patient with transfusion depandant beta thalassemia with accumulation of iron in their body affect pituitary gland and gonades lead to impaired their functions .
- Trial name
- NCT07288762
- Testing
- serum inhibin B serum INSL3 serum AMH
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Sohag University
Beta Thalassemia
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia
- Trial name
- NCT00790127
- Testing
- HQK-1001
- Where
- 2 sites in Lebanon, Thailand
- Volunteers
- 21 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Extremely Premature Infant, Anemia Neonatal
Heterologous Cord Blood-Derived Red Blood Cell for Transfusion in Extremely Preterm Infants
Anemia is a condition in which there are not enough red blood cells to carry oxygen throughout the body. It is very common in extremely preterm infants (born before 28 weeks of pregnancy), and many of these babies require red…
- Conditions
- Extremely Premature Infant, Anemia Neonatal, Blood Transfusion, Umbilical Cord Blood
- Trial name
- NCT07502781
- Testing
- Cord Blood Red Blood Cells, Standard Adult Donor Red Blood Cells
- Where
- Spain
- Volunteers
- 176 people
- Sponsor
- Hospital Clinic of Barcelona
Beta-Thalassemia
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients
Beta-thalassemias are hereditary blood disorders caused by reduced or absent synthesis of hemoglobin beta chains, with variable outcomes ranging from severe anemia to clinically asymptomatic individuals.
- Trial name
- SIRTHALACLIN (NCT03877809)
- Testing
- Sirolimus 0.5 mg
- Where
- 2 sites in Italy
- Volunteers
- 26 people
- Sponsor
- Rare Partners srl Impresa Sociale
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Beta Thalassemia
5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia
This study will evaluate the safety and effectiveness of 5-azacytidine and phenylbutyrate for treating thalassemia major. Patients with this disease have abnormal production of hemoglobin (the oxygen-carrying protein in red blood…
- Trial name
- NCT00005934
- Testing
- 5-Azacytidine
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Anemia, Beta-Thalassemia
Prevalence Of Microalbuminuria Among Children Suffering From Sickle Cell Nephropathy and Sickle Cell/Beta-Thalassemia
Sickle cell nephropathy is a known complication of sickle cell anemia (SCA) manifested by increase in glomerular filtration rate (glomerular hyperfiltration) and results in proteinuria and chronic renal failure.
- Conditions
- Sickle Cell Anemia, Beta-Thalassemia, Microalbuminuria
- Trial name
- NCT01092169
- Testing
- No intervention
- Sponsor
- Hillel Yaffe Medical Center
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
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.
- Trial name
- NCT01740531
- Testing
- S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells
- Where
- 3 sites in Italy, Turkey (Türkiye)
- Volunteers
- 86 people
- Sponsor
- Cerus Corporation
Beta-thalassemia Major Complicated With Diabetes
Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus
Beta-thalassemia represents a group of recessive inherited hemoglobin disorders characterized by reduced synthesis of β-globin chain.
- Trial name
- NCT03851055
- Testing
- Zinc
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
Beta-Thalassemia
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells.
- Trial name
- NCT06364774
- Testing
- ALS20
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Iron Overload, Thalassemia Major
"Iron Overload and Endocrinological Diseases"
Patients with hemochromatosis or Thalassemia develop progressive tissue and organs damages secondary to iron overload. Iron overload can result both from transfusional hemosiderosis and excess gastrointestinal iron absorption.
- Conditions
- Iron Overload, Thalassemia Major, Thalassemia Intermedia, Hemochromatosis
- Trial name
- NCT06137079
- Where
- Italy
- Volunteers
- 62 people
- Sponsor
- Azienda Ospedaliero-Universitaria di Modena
Thalassemia
The Role of OCTA in Patients Affected by Beta Thalassemia
To investigate, using optical coherence tomography angiography (OCTA), the retinal and choriocapillaris vascular features in patients affected by Beta Thalassemia.
- Trial name
- NCT04582110
- Testing
- Optical coherence tomography angiography
- Where
- Italy
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- Federico II University