257. 肝型糖原病 Hepatic glycogenosis Clinical trials / Disease details


臨床試験数 : 14 薬物数 : 27 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 8

  
1 trial found
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1EUCTR2021-000903-19-ES
(EUCTR)
26/11/202120/07/2021A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III)A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients with GSD III Glycogen Storage Disease Type III (GSD III)
MedDRA version: 20.1;Level: PT;Classification code 10053250;Term: Glycogen storage disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: mRNA encoding the human glycogen debranching enzyme formulated in a lipid nanoparticle delivery
Product Code: UX053
INN or Proposed INN: Not yet available
Other descriptive name: mRNA encoding the human glycogen debranching enzyme
Ultragenyx Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 1;Phase 2France;United States;Canada;Spain;Germany;United Kingdom;Italy