Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)
Primary Purpose
Glycogen Storage Disease Type II
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
RMT therapy using modified RMT device
Sham-RMT therapy using modified RMT device
Sponsored by
About this trial
This is an interventional supportive care trial for Glycogen Storage Disease Type II
Eligibility 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
Sites / Locations
- Duke University Medical Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Sham Comparator
Arm Label
Respiratory muscle training (RMT)
Sham-RMT
Arm Description
Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.
Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.
Outcomes
Primary Outcome Measures
Change in respiratory strength
RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).
Feasibility of sham-RMT, as measured by program adherence
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
Change in maximum expiratory pressures (MEP)
Change pretest to posttest
Change in 6-minute walk test (6MWT)
Change pretest to posttest
Change in Gait, Stairs, Gower, and Chair (GSGC) scale
Change pretest to posttest
Change in peak cough flow (PCF)
Change pretest to posttest
Change in Rasch-built Pompe-specific Activity Score (R-PAct)
Change pretest to posttest
Change in diaphragm thickness
change pretest to posttest
Full Information
NCT ID
NCT02801539
First Posted
June 13, 2016
Last Updated
July 29, 2019
Sponsor
Duke University
Collaborators
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
1. Study Identification
Unique Protocol Identification Number
NCT02801539
Brief Title
Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)
Official Title
Respiratory Muscle Training in Late-Onset Pompe Disease
Study Type
Interventional
2. Study Status
Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
February 2, 2017 (Actual)
Primary Completion Date
October 22, 2018 (Actual)
Study Completion Date
April 17, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Duke University
Collaborators
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glycogen Storage Disease Type II
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
22 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Respiratory muscle training (RMT)
Arm Type
Experimental
Arm Description
Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.
Arm Title
Sham-RMT
Arm Type
Sham Comparator
Arm Description
Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.
Intervention Type
Device
Intervention Name(s)
RMT therapy using modified RMT device
Intervention Description
The exercises provided by the RMT device is intended to strengthen breathing muscles.
Intervention Type
Device
Intervention Name(s)
Sham-RMT therapy using modified RMT device
Intervention Description
The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles.
Primary Outcome Measure Information:
Title
Change in respiratory strength
Description
RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).
Time Frame
baseline, 12 weeks, 6 months, 9 months
Title
Feasibility of sham-RMT, as measured by program adherence
Description
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.
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Change in maximum expiratory pressures (MEP)
Description
Change pretest to posttest
Time Frame
baseline, 12 weeks, 6 months, 9 months
Title
Change in 6-minute walk test (6MWT)
Description
Change pretest to posttest
Time Frame
baseline, 12 weeks, 6 months, 9 months
Title
Change in Gait, Stairs, Gower, and Chair (GSGC) scale
Description
Change pretest to posttest
Time Frame
baseline, 12 weeks, 6 months, 9 months
Title
Change in peak cough flow (PCF)
Description
Change pretest to posttest
Time Frame
baseline, 12 weeks, 6 months, 9 months
Title
Change in Rasch-built Pompe-specific Activity Score (R-PAct)
Description
Change pretest to posttest
Time Frame
baseline, 12 weeks, 6 months, 9 months
Title
Change in diaphragm thickness
Description
change pretest to posttest
Time Frame
baseline, 12 weeks, 6 months, 9 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Harrison Jones, PhD
Organizational Affiliation
Division of Head and Neck Surgery & Communication Sciences, Duke University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
31303277
Citation
Jones HN, Kuchibhatla M, Crisp KD, Hobson Webb LD, Case L, Batten MT, Marcus JA, Kravitz RM, Kishnani PS. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): A protocol for a sham-controlled clinical trial. Mol Genet Metab. 2019 Aug;127(4):346-354. doi: 10.1016/j.ymgme.2019.05.001. Epub 2019 May 8.
Results Reference
derived
Learn more about this trial
Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)
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