Clinical trials: Thalassemia Intermedia
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
β-thalassemia Major
A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-Thalassemia Major
- Trial name
- NCT07489196
- Testing
- CS-101 injection
- Where
- 3 sites in China
- Volunteers
- 20 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Transfusion Dependent Beta-thalassemia
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism…
- Trial name
- NCT06606886
- Testing
- Tablet Metformin (Group A), Tablet Hydroxyurea (Group B)
- Where
- Pakistan
- Volunteers
- 96 people
- Sponsor
- Bahria University
β-Thalassemia Intermedia
Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia
The purpose of this study is determination of the efficacy of combination of hydroxyurea with L-carnitine or magnesium in improving hematologic parameters and cardiac status in patients with β-thalassemia intermedia in comparison…
- Trial name
- NCT00809042
- Testing
- hydroxyurea, L-carnitine and hydroxyurea, hydroxyurea and magnesium chloride
- Where
- Iran
- Volunteers
- 120 people
- Sponsor
- Shiraz University of Medical Sciences
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).
- Trial name
- NCT04770753
- Testing
- Mitapivat
- Where
- 68 sites in United States, Brazil and 16 more
- Volunteers
- 194 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Beta-Thalassemia, Hemosiderosis
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions.
- Trial name
- NCT00171171
- Testing
- deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 252 people
- Sponsor
- Novartis
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
Cognitive Change, Beta-Thalassemia
Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia
This study aims to: Assess the cognitive function in children with beta thalassemia Evaluate the fatigue in beta thalassemic children Assess the health related quality of life measures in children with beta thalassemia.
- Conditions
- Cognitive Change, Beta-Thalassemia, Fatigue, Quality of Life
- Trial name
- NCT05469230
- Where
- Egypt
- Volunteers
- 168 people, healthy volunteers welcome
- Sponsor
- Cairo University
Beta Thalassemia Major
the Potential Immunomodulatory Effects of Spirulina on Thalassemic Children
The aim of this study is to evaluate the possible immunologic effects of spirulina in children with beta thalassemia.
- Trial name
- NCT02674607
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Tanta University
β-thalassemia
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
- Trial name
- NCT05745532
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Hemogen
Infant, Extremely Premature, Erythrocyte Transfusion
Red Blood Cells From Umbilical Cord for Transfusion of Preterm Infants
This study has been designed to demonstrate that red blood cell from umbilical cord blood (UCB-RBC) is a safe and available product for extremely preterm infants (EPI) transfusion and that transfusion of UCB-RBC is non-less…
- Conditions
- Infant, Extremely Premature, Erythrocyte Transfusion, Umbilical Cord Issue
- Trial name
- NCT05612919
- Testing
- Red blood cell from umbilical cord blood, Red blood cell from adult donor
- Where
- Spain
- Volunteers
- 41 people
- Sponsor
- Hospital Clinic of Barcelona
Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome
Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
The primary purpose of this study is to determine the safety and tolerability of SLN124 for the treatment of non-transfusion-dependent (NTD) β-thalassaemia and low risk myelodysplastic syndrome.
- Trial name
- NCT04176653
- Where
- 5 sites in Bulgaria, United Kingdom
- Sponsor
- Silence Therapeutics plc
Beta-Thalassemia
Thrombophilia In Beta Thalassemia
β-thalassemia disease is one of the most common congenital hemolytic anemia commonly found in the malarial belt areas including the Mediterranean, the Middle East, Africa, Southeast Asian countries, and China.
- Trial name
- NCT04219449
- Testing
- PT, Protein C, Platelet aggregation by ADP and arachidonic acid
- Volunteers
- 100 people
- Sponsor
- Assiut University
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 Majors (Beta-Thalassemia Major), Haplo-identical Donors
Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
- Trial name
- NCT06734520
- Testing
- super-transplantation in 3 severe β -thalassemia pediatric patients.
- Where
- 2 sites in China
- Volunteers
- 3 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Anemia, Cooley's, Beta-Thalassemia
Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)
The purpose of the TCRN is to accelerate research in the management of thalassemia, standardize existing treatments, and evaluate new ones in a network of clinical centers in North America.
- Conditions
- Anemia, Cooley's, Beta-Thalassemia, Hematologic Diseases, Thalassemia
- Trial name
- NCT00000623
- Testing
- Deferoxamine, Deferiprone, Arginine
- Where
- 5 sites in United States, Canada
- Volunteers
- 1,000 people
- Sponsor
- Carelon Research
Beta-thalassemia Major
Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients
Background: Three iron chelators now available on the market differ in toxicity and organ specificity; evidence on standardized chelation protocol remains inconclusive, but patients with transfusion-dependent beta-thalassemia…
- Trial name
- NCT02198508
- Testing
- DFX(Deferasirox), DFP(Deferiprone)
- Volunteers
- 13 people
- Sponsor
- China Medical University Hospital
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
Thalassemia Major
Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation
This study is a multicenter, open-label, two-period crossover design that evaluates the safety, tolerability, pharmacokinetics and preliminary evidence of iron chelating activity of DST-0509 as compared to Jadenu and Exjade in…
- Trial name
- NCT03637556
- Testing
- DST-0509, Jadenu, Exjade
- Where
- 4 sites in United States, Thailand
- Volunteers
- 25 people
- Sponsor
- DisperSol Technologies, LLC
Iron Overload, Beta-Thalassemia
SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
The purpose of this study is to test the safety and tolerability of SP-420 and it's efficacy in terms of lowering iron in subjects with Beta-thalassemia or other rare anemias who need regular blood transfusions.
- Trial name
- NCT03801889
- Testing
- SP-420
- Where
- 4 sites in Canada, Lebanon and 2 more
- Sponsor
- Abfero Pharmaceuticals, Inc