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

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

8 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-10-21

Study Completion Date

2016-06-22

Brief Summary

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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 .

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Detailed Description

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Conditions

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Late-onset Pompe Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Measurement of Respiratory Muscle Strength

Respiratory muscle strength measurements by different techniques.

Intervention Type PROCEDURE

Interventions

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Respiratory muscle strength measurements by different techniques.

Intervention Type PROCEDURE

Eligibility 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 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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No