Clinical trials: β-thalassemia Intermedia
Transfusion Dependent Beta Thalassemia, Hemoglobinopathies
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
- Conditions
- Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
- Trial name
- NCT05444894
- Testing
- EDIT-301
- Where
- 8 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Editas Medicine, Inc.
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.
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 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