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A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

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Trial Status Terminated

Trial Identifier

NCT02191917

Condition

Late-onset Pompe Disease

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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 BMN 701 .

Eligibility Criteria

Birth Sex

All

Age

From 18 Years

Healthy Volunteers

No

Drug/Treatment:

Respiratory muscle strength measurements by different techniques.

Study Type:

Observational

Number of Participants:

8

Study Started:

2014-10-21

Study Updated:

2023-01-09

Trial Locations

  • University of Florida

    Gainesville, Florida, United States

  • Royal Brompton Hospital

    London, , United Kingdom

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 testin

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