Basic Information

Gene symbol TNFRSF17 Synonyms BCM, BCMA, CD269, TNFRSF13A Type of gene protein-coding
Description TNF receptor superfamily member 17

GTO ID GTC0531
Trial ID NCT04561557
Disease Neuromyelitis Optica
Altered gene BCMA
Therapeutic/Target gene Target gene
TherapyCAR-T cell
Treatment CT103A
Generation2nd
PhaseEarly_Phase1
Recruitment statusRecruiting
TitleSafety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Idiopathic Inflammatory Diseases of the Nervous System
Year2020
CountryChina
Company sponsorTongji Hospital
Other ID(s)CARTinNS
Vector information
Vectorlentivirus
ConstructscFv-4-1BB-CD3ζ

Clinical Result

Cohort 1
Administration route intravenous infusion
Dosage 0.5 E6 cells/kg, 1 E6 cells/kg
Donor type Autologous
Pts 12
Age Adult, Older_Adult
Lymph depletion Yes
Outcome All patients generally reported improvement in disabilities and quality-of-life outcomes; 11 patients’ AQP-4 antibodies in serum showed a downward trend by the cutoff date.
Adverse reactions The most common events of grade 3 or higher were hematologic toxic effects. 58% developed infections, but no grade 4 infections occurred. Cytokine release syndrome was reported in all patients with only events of grade 1 or 2 observed.

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