Ultevursen ( DrugBank: - )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
90網膜色素変性症1
303アッシャー症候群1

90. 網膜色素変性症


臨床試験数 : 147 薬物数 : 176 - (DrugBank : 43) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 110
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05158296
(ClinicalTrials.gov)
December 8, 202123/11/2021Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius)Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision DisordersDrug: Ultevursen;Other: Sham-procedureProQR TherapeuticsNULLActive, not recruiting12 YearsN/AAll81Phase 2/Phase 3United States;Germany;Netherlands;United Kingdom

303. アッシャー症候群


臨床試験数 : 10 薬物数 : 11 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05158296
(ClinicalTrials.gov)
December 8, 202123/11/2021Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius)Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision DisordersDrug: Ultevursen;Other: Sham-procedureProQR TherapeuticsNULLActive, not recruiting12 YearsN/AAll81Phase 2/Phase 3United States;Germany;Netherlands;United Kingdom