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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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7 November 2016 |
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Main ID: |
NCT02221362 |
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Date of registration:
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06/08/2014 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
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Scientific title:
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A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease |
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Date of first enrolment:
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December 2014 |
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Target sample size:
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30 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT02221362 |
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Study type:
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Observational |
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Study design:
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Observational Model: Case Control, Time Perspective: Prospective
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Phase:
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N/A
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Countries of recruitment
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Australia
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Austria
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Brazil
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Canada
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Greece
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Ireland
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Korea, Republic of
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Poland
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Romania
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Serbia
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Singapore
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Slovenia
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Spain
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Taiwan
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Turkey
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Contacts
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Name:
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Medical Monitor, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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BioMarin Pharmaceutical |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- 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 Criteria:
- 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
Age minimum:
18 Years
Age maximum:
N/A
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Late-onset Pompe Patients Untreated or Treated With rhGAA
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Primary Outcome(s)
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To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease
[Time Frame: 264 Weeks]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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