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

Terminated

• Conditions: Late-onset Pompe Disease
• Interventions: Procedure: Respiratory muscle strength measurements by different techniques.

• Registration Number: NCT02191917
• Lead Sponsor: BioMarin Pharmaceutical

Brief 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 .

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-07-15
• Study First Submitted QC: 2014-07-15
• Study First Posted: 2014-07-16
• Study Start: 2014-10-21
• Primary Completion: 2016-06-22
• Study Completion: 2016-06-22
• Results First Submitted: 2017-06-22
• Status Verified: 2022-04
• Results First Submitted QC: 2022-04-11
• Last Update Submitted: 2022-04-11
• Results First Posted: 2023-01-09
• Last Update Posted: 2023-01-09

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 8

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Measurement of Respiratory Muscle Strength	Respiratory muscle strength measurements by different techniques.	-

Primary Outcome Measures

Name	Time	Method
Maximal Inspiratory Pressure (MIP)	Baseline, Week 28	Pulmonary measure of change in maximal inspiratory pressure (MIP)

Secondary Outcome Measures

Name	Time	Method
Maximal Expiratory Pressure (MEP)	Baseline, Week 28	Pulmonary measure of change in maximal expiratory pressure (MEP)

Trial Locations

Locations (2)

University of Florida; 🇺🇸 Gainesville, Florida, United States

Royal Brompton Hospital; 🇬🇧 London, United Kingdom