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: | https://clinicaltrials.gov/ct2/show/NCT03432364 |