A Study of Repiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
| Status: | Terminated |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 11/8/2017 |
| Start Date: | September 2014 |
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 BMN 701 .
test respiratory muscle strength initially and again after 24 weeks in subjects treated or
not treated with BMN 701 .
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 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 Criteria:
- 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
We found this trial at
2
sites
Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Barbara Smith, MD
Phone: 352-273-6855
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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London, SW3 6LL
Principal Investigator: Michael Polkey, MD
Phone: 0207 352 8121
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