Basic Information

Gene symbol FURIN Synonyms FUR, PACE, PCSK3, SPC1 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description furin, paired basic amino acid cleaving enzyme

Gene symbol TGFB1 Synonyms CED, DPD1, IBDIMDE, LAP, TGF-beta1, TGFB, TGFbeta Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description transforming growth factor beta 1

Gene symbol TGFB2 Synonyms G-TSF, LDS4, TGF-beta2 Type of gene protein-coding
Description transforming growth factor beta 2

GTO ID GTC0430
Trial ID NCT03495921
Disease Ewing Sarcoma
Altered gene FURIN|TGFB1|TGFB2
Therapeutic/Target gene Target gene
TherapyshRNA
Treatment Vigil|FANG Vaccine
Co-treatment Irinotecan|Temozolomide
PhasePhase3
Recruitment statusTerminated
TitleA Multi-Center Phase III, Randomized, Open-Label Trial of Vigil (Bi-shRNAfurin and GMCSF Augmented Autologous Tumor Cell Immunotherapy) in Combination With Irinotecan and Temozolomide as a Second-Line Regimen for Ewing's Sarcoma
Year2018
CountryUnited States
Company sponsorGradalis, Inc.
Other ID(s)CL-PTL-130

Clinical Result

Cohort1: Vigil_Irinotecan_Temozolomide
Administration route oral|intradermal injection
Dosage 1E6 cells/dose, on Day 15 of each cycle, 1 cycle = 21 days
Pts 3
Age Child, Adult, Older_Adult
Outcome Progression Free Survival (PFS):Median (95% Confidence Interval):2.8
Adverse reactions 2/3(All-cause mortality)
Cohort2: Irinotecan_Temozolomide
Administration route None
Dosage 0 cells/dose
Pts 2
Age Child, Adult, Older_Adult
Outcome Progression Free Survival (PFS):Median (95% Confidence Interval):9.1
Adverse reactions 1/2(All-cause mortality); 1/2(Respiratory, thoracic and mediastinal disorders)
Cohort3: Vigil
Administration route oral|intradermal injection
Dosage Vigil, 1E6 cells/dose, on Day 15 of each cycle, 1 cycle = 21 days
Pts 1
Age Child, Adult, Older_Adult
Adverse reactions No serious clinical adverse events

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