RGX-121 ( 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2

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT04571970
(ClinicalTrials.gov)
March 11, 202123/9/2020RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome)Mucopolysaccharidosis Type II (MPS II)Genetic: RGX-121REGENXBIO Inc.NULLActive, not recruiting5 Years17 YearsMale6Phase 1/Phase 2United States;Canada
2NCT03566043
(ClinicalTrials.gov)
September 27, 20181/5/2018CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)Mucopolysaccharidosis Type II (MPS II)Genetic: RGX-121REGENXBIO Inc.NULLRecruiting4 Months5 YearsMale48Phase 2/Phase 3United States;Brazil