Status:
TERMINATED
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Late-onset Pompe Patients Untreated or Treated With rhGAA
Eligibility:
All Genders
18+ years
Brief 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 dat...
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 procedure
- Diagnosed with late-onset Pompe disease based on current or previous genomic testing and/or endogenous GAA activity
- At least 18 years of age at study entry
- Willing and able to comply with all study procedures
Exclusion
- Requires non-invasive 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 study completion or data collection
- Unable to perform baseline efficacy assessments
Key Trial Info
Start Date :
December 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 1 2016
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT02221362
Start Date
December 1 2014
End Date
September 1 2016
Last Update
October 31 2016
Active Locations (24)
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1
Westmead Hospital, Dept. of Genetic Medicine
Westmead, New South Wales, Australia, 2145
2
Women's and Children's Hospital
Adelaide, South Australia, Australia, 5006
3
Monash Medical Centre
Clayton, Victoria, Australia, 3168
4
Porto Alegre, Brazil