Clinical trials: Β-thalassemia
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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
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
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
Β-thalassemia
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia.
- Trial name
- NCT06655662
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- 3 sites in China
- Volunteers
- 8 people
- Sponsor
- Shenzhen Hemogen
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
Sickle Cell Disease, Thalassemia, Beta
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow.
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Trial name
- NCT05736419
- Testing
- Fludarabine, Cyclophosphamide, Tacrolimus
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Transfusion-dependent β-thalassemia Patients, Cardiac Iron Overload
Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload
The primary efficacy endpoint of this interventional study was to evaluate the number of patients achieving a complete response (CR), defined as patients switching from intensive deferasirox -DFO treatment, at any time point…
- Trial name
- NCT01459718
- Testing
- Deferasirox, Deferoxamine (DFO)
- Where
- 4 sites in Greece
- Volunteers
- 32 people
- Sponsor
- Novartis Pharmaceuticals
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
Sickle Cell Disease, Beta Thalassemia Intermedia
Pumilio1 (PUM1) Expression, Sickle Cell Anemia, β-thalassemia Intermedia
1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3.
- Trial name
- NCT05883254
- Testing
- RNA Binding Protein Pumilio1 (PUM1) Expression by reverse transcriptase quantatative PCR
- Volunteers
- 80 people
- Sponsor
- Assiut University
Beta-Thalassemia
CS-101 in Patients With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06328764
- Testing
- CS-101
- Where
- China
- Volunteers
- 10 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
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
β-thalassemia Intermedia
Apotransferrin in Patients With β-thalassemia
The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced…
- Trial name
- NCT03993613
- Testing
- human apotransferrin
- Where
- Netherlands
- Volunteers
- 10 people
- Sponsor
- Prothya Biosolutions
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.
Thalassemia Major
NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major
The effect of N_acetylcystein as an antioxidant on iron overload and frequency of blood transfusion in β-thalassemia major patients at Assiut Childern Hospital University And its cosubmitted for partial fulfillment of master…
- Trial name
- NCT05777733
- Testing
- n-acetylcystine
- Volunteers
- 100 people
- Sponsor
- Assiut University
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
- Trial name
- NCT06479616
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Fudan University
Beta Thalassemia Major, Beta Thalassemia Intermedia
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Trial name
- NCT01571635
- Testing
- SOTATERCEPT (ACE-011)
- Where
- 18 sites in France, Greece and 2 more
- Volunteers
- 46 people
- Sponsor
- Celgene
β-Thalassemia, Pulmonary Function
Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemia
The aims of this study are: 1. To determine the correlation between pulmonary functions and physical fitness in children with β-thalassemia. 2.
- Conditions
- β-Thalassemia, Pulmonary Function, Physical Fitness
- Trial name
- NCT05494333
- Testing
- no intervention
- Where
- Egypt
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Cairo University