Clinical trials: β-thalassemia
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, 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
Hematologic Diseases
A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.
- Trial name
- NCT05015920
- Testing
- BD211 Drug Product
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Beta Thalassemia Major
Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism
Hematopoietic stem cell transplantation is currently the only way to cure thalassemia, one of its main obstacles is the rejection after transplantation, chimerism continued to decline, which eventually lead to transplant failure.
- Trial name
- NCT03101423
- Testing
- Interleukin-2, Donor Regulatory T-Lymphocytes
- Where
- China
- Volunteers
- 30 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Pateints of β-thalassemia Major With Iron Overload
Comparison of Deferasirox and Desferoxamine in Patients of β-Thalassemia Major With Iron Overload
This study was planned to compare deferasirox and desferrioxamine in terms of mean serum ferritin levels in patients of β-thalassemia major having Iron overload.
- Trial name
- NCT06468423
- Testing
- Deferasirox (DFX), Deferoxamine (DFO)
- Where
- Pakistan
- Volunteers
- 142 people
- Sponsor
- RESnTEC, Institute of Research
Thalassemia, Iron Deficiency
Detection of β Thalassemia Carriers by Red Cell Parameters Obtained From the H2 Automatic Counter
β thalassemia is an autosomal recessive hemoglobinopathy and considered as the most widespread genetic mutation. According to the World Health Organization (WHO) between 1.5-7% of the world population are carriers for this…
- Trial name
- NCT00481221
- Testing
- Observation of results from laboratory tests
- Where
- Israel
- Volunteers
- 30,000 people
- Sponsor
- HaEmek Medical Center, Israel
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.
Myelodysplastic Syndrome, Beta Thalassemia
A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia
The purpose of this observational study is to assess the real-world safety of luspatercept in Korean participants with myelodysplastic syndrome (MDS) or beta thalassemia.
- Trial name
- NCT06073860
- Testing
- Luspatercept
- Where
- 2 sites in South Korea
- Volunteers
- 104 people
- Sponsor
- Bristol-Myers Squibb
Transfusional Iron Overload
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional…
- Trial name
- NCT01044186
- Testing
- ICL670
- Where
- 18 sites in United States, Greece and 1 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
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
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
Transfusion-dependent β-thalassemia Patients
Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
β-thalassemia is one of the most common inherited hemoglobinopathies worldwide and a major public health issue that severely impacts birth quality, human health, and social progress.
- Trial name
- NCT07338344
- Testing
- Luspatercept combined with low-dose thalidomide
- Where
- 8 sites in China
- Volunteers
- 78 people
- Sponsor
- Rongrong Liu
β-thalassemia
A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
- Trial name
- NCT07215975
- Testing
- Luspatercept
- Where
- 13 sites in Kuwait, Oman and 2 more
- Volunteers
- 200 people
- Sponsor
- Bristol-Myers Squibb
β-thalassemia Major
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major.
- Trial name
- NCT01745120
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 6 sites in United States, Australia and 1 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Confirmed Diagnosis of ß-thalassemia Major
ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene
The patient has inherited ß-thalassemia major through the genes. These genes have mistakes in them, so the body cannot make normal red blood cells. Stem cells are made in the bone marrow.
- Trial name
- NCT01639690
- Testing
- Autologous CD34+ cells transduced with TNS9.3.55
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- San Rocco Therapeutics
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
Thalassemia, Beta
Long-term Follow-up Study of BRL-101 for TDT
Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.
- Trial name
- NCT06298630
- Testing
- Assessments
- Where
- 7 sites in China
- Volunteers
- 45 people
- Sponsor
- Bioray Laboratories
Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)
YOLT-204 in Patients With Hemoglobinopathies
This is a single-arm, open-label, single-dose, dose-escalation trial that plans to enrol 3-18 patients with transfusion-dependent β-thalassaemia (TDT) or sickle-cell disease (SCD).
- Trial name
- NCT07190001
- Testing
- YOLT-204
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Transfusional Iron Overload, β-thalassemia Major
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed.
- Conditions
- Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
- Trial name
- NCT00390858
- Testing
- Deferasirox
- Where
- 4 sites in France, Italy
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
β-thalassemia
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector…
- Trial name
- NCT06465550
- Testing
- BD211
- Where
- 3 sites in China
- Volunteers
- 9 people
- Sponsor
- Shanghai BDgene Co., Ltd.