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항목
CGTStandard DatabasestalePhase 2
stem cells (stem cells)
Genzyme, a Sanofi Company·Acute Lymphoblastic Leukemia
62 trials
Overview
Program
stem cells
Overview
Company
Genzyme, a Sanofi Company
Overview
Modality
CGT
Overview
Target
Acute Lymphoblastic Leukemia
Overview
Indication
Acute Lymphoblastic Leukemia; Acute Myelocytic Leukemia
Overview
Phase
PHASE_2
Overview
Status
DISCONTINUED
Overview
Content status
Standard Database
Overview
Data Confidence
Data Confidence · Medium
Overview
Development Signal
Development Signal · Emerging
Overview
Approval status
Investigational
Technology
Vector
lentiviral barcoded library
MoA
Mechanism
targeting the STAT3 pathway
MoA
Biomarker
biomarker changes following a single intrathecal injection of autologous bone marrow-derived mesenchymal stem
PK/PD
Species
Mouse
PK/PD
Animal (cat.)
Mouse, In vitro
PK/PD
Experiment
tumor growth inhibition
Toxicology
Species
Mouse
Toxicology
Animal (cat.)
Mouse, In vitro
Toxicology
Major finding
Safety and preliminary efficacy of autologous bone marrow-derived mesenchymal stem cell transplantation in hereditary cerebellar ataxia: phase I/IIa clinical trial.. Hereditary cerebellar ataxias (HCA) encompass a spectrum of pathological conditions affecting the cerebellum. Currently, there is growing interest in the potential role of mesenchymal stem cells (MSCs) as an investigational therapeuti…
Clinical
Safety signal
Safety and preliminary efficacy of autologous bone marrow-derived mesenchymal stem cell transplantation in hereditary cerebellar ataxia: phase I/IIa clinical trial.. Hereditary cerebellar ataxias (HCA) encompass a spectrum of pathological conditions affecting the cerebellum. Currently, there is growing interest in the potential role of mesenchymal stem cells (MSCs) as an investigational therapeuti…
Clinical
Reported PFS
35.5
Clinical
Reported OS
68
Clinical
Result source
ClinicalTrials.gov NCT02208037
Clinical
Program phase
PHASE_2
Clinical
Trial ref
NCT02208037