Clinical trials: Transfusion-dependent Beta-Thalassemia
Beta Thalassemia Major
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
- Trial name
- NCT03947632
- Testing
- serum ferritin and hemoglobin test
- Volunteers
- 367 people
- Sponsor
- Aga Khan University
Beta-Thalassemia
Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen
repeated transfusions are the mainstay of disease management in most patients with transfusion dependent beta thalassemia.iron overload predispose to oxidative stress and tissue injury.
- Trial name
- NCT06509581
- Testing
- vitamin E
- Where
- Egypt
- Volunteers
- 180 people
- Sponsor
- Ain Shams University
Transfusion Dependent Beta Thalassemia
Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation.
- Trial name
- Thal-H (NCT07292259)
- Testing
- ADDITION OF THALIDOMIDE AND HYDROXYUREA
- Where
- 9 sites in Pakistan
- Volunteers
- 100 people
- Sponsor
- Pakistan Blood and Marrow Transplant (PBMT) Group
Thalassemia, Beta
Long-term Follow-up Study of BHC001 for TDT
Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
- Trial name
- NCT06314529
- Testing
- Assessments
- Where
- 2 sites in China
- Volunteers
- 6 people
- Sponsor
- Bioray Laboratories
Thalassemia Major
Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients.
- Trial name
- NCT03992001
- Testing
- Blood component A, Blood component B
- Where
- Italy
- Volunteers
- 55 people
- Sponsor
- Università degli Studi di Ferrara
Hereditary Anemias
Utility of Fibroscan in Estimating Hepatic Iron Concentration
In patients with hereditary anemias (e.g. thalassemias), defective red blood cells are produced due to an error in the genes, or DNA, that provide the instructions for their synthesis.
- Trial name
- NCT02067130
- Testing
- Fibroscan
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- University of British Columbia
Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
- Trial name
- NCT05693909
- Testing
- SP-420
- Where
- Denmark
- Volunteers
- 90 people
- Sponsor
- Pharmacosmos A/S
Beta-Thalassemia
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with…
- Trial name
- NCT03381833
- Testing
- LJPC-401
- Where
- 22 sites in United States, Australia and 6 more
- Volunteers
- 84 people
- Sponsor
- La Jolla Pharmaceutical Company
Chronic Iron Overload
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasirox (DFX) at dose of 20-40…
- Trial name
- NCT01825512
- Testing
- Deferiprone, Deferasirox
- Where
- 22 sites in Albania, Cyprus and 5 more
- Volunteers
- 435 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0…
- Trial name
- NCT02906202
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Genetix Biotherapeutics Inc.
Transfusion-dependent Beta-Thalassemia
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT05860595
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Thalassemia Major With Severe Transfusional Iron Overload
Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.
- Trial name
- NCT01709032
- Testing
- Deferasirox and deferiprone
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital of Philadelphia
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
β-thalassemia, Ineffective Erythropoiesis
Safety Study for Beta Thalassemia Subjects on PTG-300
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
- Trial name
- TRANSCEND (NCT04054921)
- Testing
- PTG-300
- Where
- 16 sites in United States, Greece and 6 more
- Volunteers
- 34 people
- Sponsor
- Protagonist Therapeutics, Inc.
Liver Iron Overload
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Trial name
- NCT01033747
- Testing
- Deferasirox
- Where
- 4 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Sickle Cell Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Thalassemia Major, Chronic Blood Transfusion Related Iron Overload
MR Imaging of Diffuse Myocardial Fibrosis in Transfusion-Dependent Anemias
Cardiac failure is the major cause of death in patients with thalassemia and chronic blood transfusion-related iron overload. The treatment of thalassemia has been revolutionized over the past decade with the implementation of…
- Trial name
- MAFIO (NCT02090699)
- Where
- Canada
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- University Health Network, Toronto
Beta Thalassaemia
Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)
OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene.
- Trial name
- NCT03275051
- Testing
- Safety and Efficacy assessments
- Where
- Italy
- Volunteers
- 9 people
- Sponsor
- IRCCS San Raffaele
Beta Thalassemia Intermedia
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.
- Trial name
- NCT04059406
- Testing
- sapablursen
- Where
- 19 sites in Australia, Greece and 3 more
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.