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

Late-onset Pompe Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02191917
• Other study ID #: 701-201
• Secondary ID: 2014-002158-38
• Status: Terminated
• Start date: October 21, 2014
• Est. completion date: June 22, 2016

Study information

• Verified date: April 2022
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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 .

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 8
• Est. completion date: June 22, 2016
• Est. primary completion date: June 22, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Late-onset Pompe Disease

Intervention

Procedure:
• Respiratory muscle strength measurements by different techniques.

Locations

Country	Name	City	State
United Kingdom	Royal Brompton Hospital	London
United States	University of Florida	Gainesville	Florida

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Maximal Inspiratory Pressure (MIP)	Pulmonary measure of change in maximal inspiratory pressure (MIP)	Baseline, Week 28
Secondary	Maximal Expiratory Pressure (MEP)	Pulmonary measure of change in maximal expiratory pressure (MEP)	Baseline, Week 28