NCT02191917

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

A Study of Respiratory Muscle Strength, Including Effort-Independent Measures, in Subjects With Late-Onset Pompe Disease

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 8 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 .

Conditions

• Late-onset Pompe Disease

Interventions

Type	Name	Description
PROCEDURE	Respiratory muscle strength measurements by different techniques.

Timeline

• Start date: 2014-10-21
• Primary completion: 2016-06-22
• Completion: 2016-06-22
• First posted: 2014-07-16
• Last updated: 2023-01-09
• Results posted: 2023-01-09

Locations

2 sites across 2 countries: United States, United Kingdom