A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT ID: NCT02221362
Last Updated: 2016-10-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
30 participants
OBSERVATIONAL
2014-12-31
2016-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_CONTROL
PROSPECTIVE
Study Groups
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Observational
No interventions
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
* 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
18 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Monitor, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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Westmead Hospital, Dept. of Genetic Medicine
Westmead, New South Wales, Australia
Women's and Children's Hospital
Adelaide, South Australia, Australia
Monash Medical Centre
Clayton, Victoria, Australia
Porto Alegre, , Brazil
Recife, , Brazil
Ribeirão Preto, , Brazil
Alberta Children's Hospital
Calgary, Alberta, Canada
Alexandroupoli, , Greece
Athens, , Greece
Larissa, , Greece
Thessaloniki, , Greece
St. Vincent's University Hospital
Dublin, , Ireland
Copernicus, St. Adalbert Hospital
Gdansk, , Poland
Diamond Clinic
Krakow, , Poland
Insitute of Psychiatry and Neurology
Warsaw, , Poland
Spitalul Clinic Judetean de Urgenta "Sf. Apostol Andrei"
Constanța, , Romania
Belgrade, , Serbia
National University Hospital
Singapore, , Singapore
Ljubljana, , Slovenia
Seoul National University Hospital
Seoul, , South Korea
Hospital de la Santa Creu I Sant Pau
Barcelona, , Spain
Hospital General Universitario Gregorio Maranon
Madrid, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
Mackay Memorial Hospital
Taipei, , Taiwan
Countries
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Other Identifiers
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701-901
Identifier Type: -
Identifier Source: org_study_id