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

NCT02221362 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 30

Last updated 2016-10-31

No results posted yet for this study

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.

Conditions

  • Late-onset Pompe Patients Untreated or Treated With rhGAA

Sponsors & Collaborators

Principal Investigators

  • Medical Monitor, MD · BioMarin Pharmaceutical

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-12-31
Primary Completion
2016-09-30
Completion
2016-09-30

Countries

  • Australia
  • Brazil
  • Canada
  • Greece
  • Ireland
  • Poland
  • Romania
  • Serbia
  • Singapore
  • Slovenia
  • South Korea
  • Spain
  • Taiwan

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02221362 on ClinicalTrials.gov