MRNA encoding the human glycogen debranching enzyme formulated in a lipid nanoparticle delivery ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 257 | Hepatic glycogenosis | 1 |
257. Hepatic glycogenosis
Clinical trials : 14 / Drugs : 27 - (DrugBank : 7) / Drug target genes : 3 - Drug target pathways : 8
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2021-000903-19-ES (EUCTR) | 26/11/2021 | 20/07/2021 | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 1;Phase 2 | France;United States;Canada;Spain;Germany;United Kingdom;Italy |