Beta-Thalassemia clinical trials at UCSF
2 in progress, 1 open to new patients
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A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who Have a β0/β0 Genotype
open to eligible people ages up to 50 years
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 15 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0 mutation at both alleles of the β-globin (HBB) gene (i.e. β0/β0). The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
Oakland, California and other locations
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An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
Sorry, in progress, not accepting new patients
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require regular red blood cell transfusion due to (β)-thalassemia. The study is divided into the Screening/Run-in Period, double-blind Treatment Period, double-blind Long-term Treatment Period, and Post-treatment Follow-up Period.
Oakland, California and other locations
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