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Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)

NCT02824068 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 112

Last updated 2021-02-10

No results posted yet for this study

Summary

Long-term outcome in late-onset Pompe disease treated beyond 36 months (ATBIG-Pompe-Study), a multicenter, multinational, longitudinal, non-interventional observational study in subjects, at least 8 years old, diagnosed with late-onset Pompe disease retrospectively and prospectively collects data to understand clinical progression in terms of muscle and respiratory function, and clinical symptomology treated with alglucosidase alfa more than 36 months in 100 subjects.

Conditions

  • Pompe Disease

Interventions

DRUG

glucosidase alfa

Long-term use in an observational study of licenced drug

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

  • LMU Klinikum

    lead OTHER

Principal Investigators

  • Benedikt Schoser, MD · Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen Munich, Germany

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-06-01
Primary Completion
2019-12-31
Completion
2020-12-31

Countries

  • Germany
  • Italy
  • Taiwan

Study Locations

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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 NCT02824068 on ClinicalTrials.gov