PBGM01 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病1

19. ライソゾーム病


臨床試験数 : 854 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04713475
(ClinicalTrials.gov)
March 17, 20214/1/2021Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 GangliosidosisPhase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Subjects With Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 GangliosidosisGM1 Gangliosidosis;GM1 Gangliosidosis, Type I;GM1 Gangliosidosis, Type 2;Beta-Galactosidase-1 (GLB1) DeficiencyBiological: PBGM01Passage Bio, Inc.NULLRecruiting4 Months36 MonthsAll20Phase 1/Phase 2United States;Brazil;Canada;Turkey;United Kingdom