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

• Sponsor: BioMarin Pharmaceutical (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT02191917
• Collaborator: (none)
• Enrollment: 8
• Locations: 2
• Arm: 1
• Patients Per Site: 4

Study Details

Study Description

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 .

Condition or Disease	Intervention/Treatment	Phase
Late-onset Pompe Disease	Procedure: Respiratory muscle strength measurements by different techniques.	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 8 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Basic Science
• Study Start Date: Sep 1, 2014
• Anticipated Primary Completion Date: Sep 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Measurement of Respiratory Muscle Strength	Procedure: Respiratory muscle strength measurements by different techniques.

Outcome Measures

Primary Outcome Measures

1. Compare respiratory muscle strength values obtained using different techniques [24 weeks]

Secondary Outcome Measures

1. Measure change in respiratory muscle strength from Week 1 to Week 24 [24 weeks]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Florida	Gainesville	Florida	United States	32610
2	Royal Brompton Hospital	London		United Kingdom	SW36NP

Sponsors and Collaborators

• BioMarin Pharmaceutical

Investigators

• Study Director: Medical Monitor, MD, BioMarin Pharmaceutical

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: BioMarin Pharmaceutical
• ClinicalTrials.gov Identifier: NCT02191917
• Other Study ID Numbers: 701-201; 2014-002158-38
• First Posted: Jul 16, 2014
• Last Update Posted: Nov 3, 2016
• Last Verified: Nov 1, 2016

• Additional relevant MeSH terms: Glycogen Storage Disease Type II

Study Results

No Results Posted as of Nov 3, 2016