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ST-400 Investigational product

Phase 1

Transfusion Dependent Beta-thalassemia | Gene therapy | Hematology |Sangamo Therapeutics, Inc.|Last Updated: Dec 14, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment5
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03432364A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)PHASE1 COMPLETED 5 — — Mar 29, 2018Nov 17, 2022Dec 14, 20236 United States
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Study Endpoints
Primary Endpoints
Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 Weeks After the ST-400 Infusion
Up to 156 weeks after the ST-400 infusion

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

Secondary Endpoints
Clinical Laboratory Measurement of Hemoglobin (Hb) Fractions (A and F in g/dL)
Baseline, Weeks 26, 52, and 156 after ST-400 infusion
Clinical Laboratory Measurements of Percent (%) HbF
Baseline, Weeks 26, 52, and 156 after ST-400 infusion
Annualized Frequency of Packed RBC Transfusions
From Baseline (2 years prior to screening/consent), to ST-400 Infusion (Day 0), after hematopoietic reconstitution and up to 156 weeks (post ST-400 infusion)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ST-400 Investigational productEXPERIMENTALST-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
Interventions
NameTypeDescription
ST-400 Investigational productGENETICSingle dose of ST-400 following chemotherapy conditioning with busulfan
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Eligibility Criteria
Age Range18 Years — 40 Years
SexALL
Healthy VolunteersNo
Study Sites6

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...

Countries:United States
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