UX701 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
171ウィルソン病2

171. ウィルソン病


臨床試験数 : 68 薬物数 : 79 - (DrugBank : 17) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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agemax
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PhaseCountries
1EUCTR2020-005266-34-ES
(EUCTR)
11/02/202206/09/2021A study to understand the safety and the effects of a virus that transfers a modified protein responsible for copper metabolism (copper-transporting P-type adenosine triphosphatase, ATP7B) in adults with Wilson disease.A Randomized, Double-blind, Placebo-controlled, Multicenter, Seamless, Adaptive, Safety, Dose-finding, and Phase 3 Clinical Study of UX701 AAV-mediated Gene Transfer for the Treatment of Wilson Disease - Cyprus2+ Wilson disease
MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: UX701 (5.0 × 10^12 genome copies/kg)
INN or Proposed INN: UX701
Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B
Product Name: UX701 (1.0 × 10^13 genome copies/kg)
INN or Proposed INN: UX701
Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B
Product Name: UX701 (2.0 × 10^13 genome copies/kg)
INN or Proposed INN: UX701
Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B
Ultragenyx Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
94Phase 1;Phase 2;Phase 3United States;Portugal;Spain;Austria;Italy;United Kingdom;France;Canada;Argentina;Brazil;Poland;Germany;Japan
2NCT04884815
(ClinicalTrials.gov)
September 27, 20217/5/2021Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson DiseaseA Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson DiseaseWilson DiseaseGenetic: UX701;Other: Placebo;Drug: oral corticosteroids;Drug: Placebo for oral corticosteroidsUltragenyx Pharmaceutical IncNULLRecruiting18 YearsN/AAll90Phase 1/Phase 2United States