Status:
TERMINATED
A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Late-onset Pompe Disease
Eligibility:
All Genders
18+ years
Brief Summary
Study 701-201 is a study in patients with late-onset Pompe disease (LOPD). The study will test respiratory muscle strength initially and again after 24 weeks in subjects treated or not treated with BM...
Eligibility Criteria
Inclusion
- Willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any study-related procedures
- Documented diagnosis with late-onset Pompe disease - At least 18 years of age at study entry
- Willing and able to comply with all study procedures
Exclusion
- Requires ventilatory support while awake and in the upright position
- Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise patient well-being, study completion, or data collection
- Allergy to tools or procedures used for respiratory muscle testing
Key Trial Info
Start Date :
October 21 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 22 2016
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT02191917
Start Date
October 21 2014
End Date
June 22 2016
Last Update
January 9 2023
Active Locations (2)
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1
University of Florida
Gainesville, Florida, United States, 32610
2
Royal Brompton Hospital
London, United Kingdom, SW36NP