256. Muscle glycogenosis Clinical trials / Disease details


Clinical trials : 180 Drugs : 153 - (DrugBank : 30) / Drug target genes : 31 - Drug target pathways : 134

  
1 trial found
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PhaseCountries
1EUCTR2016-003475-21-GB
(EUCTR)
17/05/201703/01/2017A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa (NeoGAA)
Product Code: GZ402666
INN or Proposed INN: Avalglucosidase Alfa (NeoGAA)
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
34Phase 2United States;France;Taiwan;Germany;Japan;United Kingdom