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ST-400 Investigational product · 1 trial · 1 indication
Safety and tolerability assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 weeks after the ST-400 infusion
| Arm | Type | Description |
|---|---|---|
| ST-400 Investigational product | EXPERIMENTAL | ST-400 Investigational product is composed of autologous CD34+ hematopoietic stem/progenitor cells that are genetically modified ex vivo at the erythroid-specific enhancer of the BCL11A gene |
| Name | Type | Description |
|---|---|---|
| ST-400 Investigational product | GENETIC | Single dose of ST-400 following chemotherapy conditioning with busulfan |
Inclusion Criteria: 1. Informed Consent 2. Clinical diagnosis of TDT with ≥ 8 documented RBC transfusion events per year on an annualized basis in the 2-years prior to screening 3. Confirmed beta-thalassemia diagnosis by molecular genetic testing 4. Clinically stable and eligible to receive conditi...
ST-400 is an investigational gene therapy developed by Sangamo Therapeutics for transfusion-dependent beta-thalassemia, a hematologic disorder requiring regular blood transfusions. It is a cell-based gene therapy product studied in a single Phase 1/2 clinical trial conducted in the United States. ST-400 has not been established as an approved treatment.
ST-400 was studied as a potential treatment for transfusion-dependent beta-thalassemia, a condition in which patients require chronic red blood cell transfusions. The Phase 1/2 trial enrolled adults 18 years and older of all sexes who were not healthy volunteers. The study assessed safety, tolerability, and efficacy in this patient population.
ST-400 is developed by Sangamo Therapeutics, Inc., a biopharmaceutical company whose common stock trades under the ticker SGMOQ. Sangamo sponsored the clinical program evaluating ST-400 in transfusion-dependent beta-thalassemia.
ST-400 is in Phase 1/2 development. Its single clinical trial, NCT03432364, was a Phase 1/Phase 2 study that has been completed. ST-400 is an investigational gene therapy and is not an approved product; the completed early-stage study does not by itself establish approval status.
ST-400 has been evaluated in one clinical trial, NCT03432364, titled a study to assess the safety, tolerability, and efficacy of ST-400 for treatment of transfusion-dependent beta-thalassemia. The trial was a completed Phase 1/Phase 2, uncontrolled, open-label study with 5 enrolled participants, conducted in the United States.
The ST-400 Phase 1/2 trial, NCT03432364, enrolled 5 participants. It was an uncontrolled study without randomization or double blinding, and it enrolled adults aged 18 years and older with transfusion-dependent beta-thalassemia in the United States. The trial has been completed.