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

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ClinicalTrials.gov Identifier: NCT02191917
Recruitment Status : Terminated
First Posted : July 16, 2014
Last Update Posted : November 3, 2016
Sponsor:
Information provided by (Responsible Party):
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 .

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Study Start Date : September 2014
Estimated Primary Completion Date : September 2017


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



Primary Outcome Measures :
  1. Compare respiratory muscle strength values obtained using different techniques [ Time Frame: 24 weeks ]

Secondary Outcome Measures :
  1. Measure change in respiratory muscle strength from Week 1 to Week 24 [ Time Frame: 24 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02191917


Locations
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United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United Kingdom
Royal Brompton Hospital
London, United Kingdom, SW36NP
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
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Study Director: Medical Monitor, MD BioMarin Pharmaceutical

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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02191917     History of Changes
Other Study ID Numbers: 701-201
2014-002158-38 ( EudraCT Number )
First Posted: July 16, 2014    Key Record Dates
Last Update Posted: November 3, 2016
Last Verified: November 2016
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors