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Trial record 50 of 103 for:    Pompe Disease

Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

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ClinicalTrials.gov Identifier: NCT02801539
Recruitment Status : Recruiting
First Posted : June 16, 2016
Last Update Posted : March 29, 2019
Sponsor:
Collaborator:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
Duke University

Brief Summary:
This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to decide if sham-RMT is a useful control condition for RMT and to choose the best ways to measure the health benefits of RMT in LOPD.

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II Device: RMT therapy using modified RMT device Device: Sham-RMT therapy using modified RMT device Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 28 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Supportive Care
Official Title: Respiratory Muscle Training in Late-Onset Pompe Disease
Actual Study Start Date : February 2, 2017
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2019


Arm Intervention/treatment
Experimental: Respiratory muscle training (RMT)
Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.
Device: RMT therapy using modified RMT device
The exercises provided by the RMT device is intended to strengthen breathing muscles.

Sham Comparator: Sham-RMT
Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.
Device: Sham-RMT therapy using modified RMT device
The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles.




Primary Outcome Measures :
  1. Change in respiratory strength [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
    RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).

  2. Feasibility of sham-RMT, as measured by program adherence [ Time Frame: 12 weeks ]
    As measured by program adherence (good is considered greater than or equal to 80%). Helps determines if a future efficacy trial is warranted and if sham-RMT may be a good control condition for a future trial.


Secondary Outcome Measures :
  1. Change in maximum expiratory pressures (MEP) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
    Change pretest to posttest

  2. Change in 6-minute walk test (6MWT) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
    Change pretest to posttest

  3. Change in Gait, Stairs, Gower, and Chair (GSGC) scale [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
    Change pretest to posttest

  4. Change in peak cough flow (PCF) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
    Change pretest to posttest

  5. Change in Rasch-built Pompe-specific Activity Score (R-PAct) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
    Change pretest to posttest

  6. Change in diaphragm thickness [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
    change pretest to posttest



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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:

  • Age ≥ 18 years
  • Diagnosis of LOPD
  • On enzyme replacement therapy for ≥ 26 weeks at pretest
  • Able to follow directions for study participation
  • Able to complete a home-based RMT regimen

Exclusion Criteria:

  • Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic condition that would prevent meaningful study participation as determined at the discretion of the principle investigator
  • Inability to give legally effective consent
  • Inability to read and understand English

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): NCT02801539


Contacts
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Contact: Amy Walker 919-684-1732 amy.walker1@duke.edu
Contact: Kelly Crisp kelly.crisp@duke.edu

Locations
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United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Principal Investigator: Harrision Jones         
Sponsors and Collaborators
Duke University
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Investigators
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Principal Investigator: Harrison Jones, PhD Division of Head and Neck Surgery & Communication Sciences, Duke University

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT02801539     History of Changes
Other Study ID Numbers: Pro00068728
1R21AR069880-01 ( U.S. NIH Grant/Contract )
First Posted: June 16, 2016    Key Record Dates
Last Update Posted: March 29, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors