Beta-Thalassemia clinical trials at UCSF
2 in progress, 1 open to new patients

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
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