Open-label, Single-arm Study to Assess Safety, Tolerability, and Efficacy of ST-400 Autologous Hematopoietic Stem Cell Transplant for Treatment of Transfusion-dependent Beta thalassemia

NCT#/Phase: NCT03432364
Phase 1/2
IRB#: 2013-018
Description: This is a Phase 1/2, open-label, multi-center, single-arm study conducted in six subjects with a diagnosis of transfusion dependent Beta thalassemia. Eligible subjects will be enrolled and undergo apheresis to collect autologous CD34+ stem cells. The CD34+ stem cells will be treated ex vivo by transfection with Zinc Finger Nuclease mRNAs SB-mRENH1 and SB-mRENH2 to manufacture the study drug, ST-400. Subjects will receive conditioining therapy with IV Busulfan before being infused with ST-400.
Inclusion: Individuals with confirmed Beta thalassemia by genetic testing between 18 to 40 years of age receiving > 8 RBC transfusions per year for at least two years prior to enrollment. Patients must be clinically stable to undergo transplant and willing and able to use contraception for 3 months prior to ST-400 infusion through 6 months post infusion.
Enrollment Status: Open for enrollment
Sponsor: Sangamo Therapeutics, Inc.
Principal Investigator(s): Mark Walters, MD
Contact(s): Marci Moriarty, RN (510-428-3885 x5396)
Julie Decaris (510-428-3885 x2739)
Study Link: