A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Late-onset Pompe Patients Untreated or Treated With rhGAA Clinical Trial

Official title: A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Status Terminated

Clinical Trial Summary

Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease prospectively collects data to understand clinical progression in terms of respiratory function, symptomology, genotype, biochemistry, endurance and selected subject-reported measures for 24 weeks followed by a 240 week additional observation period for up to 100 subjects.

Clinical Trial Description

n/a

Study Design

Observational Model: Case Control, Time Perspective: Prospective

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Late-onset Pompe Patients Untreated or Treated With rhGAA

• NCT number: NCT02221362
• Study type: Observational
• Source: BioMarin Pharmaceutical
• Status: Terminated
• Phase: N/A
• Start date: December 2014
• Completion date: September 2016