Clinical trials: Transfusion-dependent Thalassemia
Transfusion Dependent Beta-Thalassaemia
Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Trial name
- NCT04390971
- Testing
- ET-01
- Where
- 3 sites in China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Transfusion Dependent Beta Thalassemia
A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with…
- Trial name
- NCT06678165
- Testing
- YOLT-204
- Where
- China
- Volunteers
- 3 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Hemochromatosis
Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent Patients
Systematical (retro- and prospective) investigation of the long-term safety (toxicity assessment according to CTCAE v3.0) and efficacy of deferiprone either given alone or in combination with desferrioxamine
- Trial name
- NCT00349453
- Testing
- Deferiprone (L1), Desferrioxamine
- Where
- 13 sites in Switzerland
- Volunteers
- 24 people
- Sponsor
- Lipomed
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia Majors (Beta-Thalassemia Major)
Effect of Omega 3 Fatty Acids on Heart and Liver in Beta Thalassemia Patients
the aim of this interventional study to show the effect of taking omega 3 for about 6 months for patients beta thalassemia major maintained on standard of care and receiving the iron chelator deferasirox on liver, cardiac…
- Trial name
- NCT07863791
- Testing
- Omega 3 Fish Oil supplements, Deferasirox
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Ain Shams University
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Anemia
A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India
- Trial name
- NCT05891249
- Testing
- Luspatercept
- Where
- 9 sites in India
- Volunteers
- 85 people
- Sponsor
- Bristol-Myers Squibb
Iron Overload, Beta-Thalassemia
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Trial name
- NCT02274233
- Testing
- SP-420
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 24 people
- Sponsor
- Sideris Pharmaceuticals
α-thalassemia
the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia
This is an open label study to evaluate the safety and efficacy of α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major Patients
- Trial name
- NCT05851105
- Testing
- α-globin restored autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Shenzhen Hemogen
Non-transfusion Dependent Thalassemia
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload.
- Trial name
- THALASSA (NCT00873041)
- Testing
- deferasirox
- Where
- 21 sites in United States, Greece and 7 more
- Volunteers
- 166 people
- Sponsor
- Novartis Pharmaceuticals
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
Thalassemia, Beta, Thalassemia Major
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
- Trial name
- NCT06041620
- Testing
- VGB-Ex01
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Beta-Thalassemia Major, Transfusion-Dependent Thalassemia
The Effect of Motivational Interviewing on Treatment Adherence and Self-Efficacy Among Omani Adults With Thalassemia
Beta-thalassemia major is a chronic hereditary blood disorder that requires lifelong treatment, including regular blood transfusions and iron chelation therapy.
- Trial name
- MITAS (NCT07660224)
- Testing
- Motivational Interviewing
- Volunteers
- 45 people
- Sponsor
- Oman Ministry of Health
β-thalassemia
the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
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
- NCT05864170
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Shenzhen Hemogen
Thalassemia, Iron Overload
Long-term Clinical Study of CN128 in Thalassemia Patients
1. Primary objectives: To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above. 2.
- Trial name
- NCT04614779
- Testing
- CN128 Tablets
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Hangzhou Zede Pharma-Tech Co., Ltd.
Fetal Hemoglobin, Thalassemia Major
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing…
- Conditions
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- Trial name
- BTM (NCT06490627)
- Testing
- Thalidomide
- Where
- Pakistan
- Volunteers
- 54 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who…
- Trial name
- NCT05991336
- Testing
- Gene therapy
- Where
- China
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
This study is researching an experimental drug called REGN7999 (called "study drug"). The study is focused on patients with non-transfusion dependent beta-thalassemia.
- Trial name
- FERVENT-1 (NCT06421636)
- Testing
- REGN7999
- Where
- 26 sites in United States, Georgia and 7 more
- Volunteers
- 19 people
- Sponsor
- Regeneron Pharmaceuticals
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
Transfusion-dependent Beta-Thalassemia, Blood Transfusion
Use of the Hemanext One® Hypoxic Red Blood Cell Storage System for Transfusion in Thalassemia Patients
Transfusion-dependent thalassemia (TDT) requires lifelong, regular red blood cell (RBC) transfusions. Conventionally stored RBCs develop biochemical and structural "storage lesions," driven largely by oxidative stress, which may…
- Trial name
- NCT07055503
- Where
- Greece
- Volunteers
- 30 people
- Sponsor
- University Research Institute for the Study of Genetic & Malignant Disorders in Childhood