Back to resultsLingual Muscle Training in Late-Onset Pompe Disease (LOPD)
Primary Purpose
Glycogen Storage Disease
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Lingual Muscle Training
Sponsored by
About this trial
This is an interventional treatment trial for Glycogen Storage Disease
Eligibility Criteria
Inclusion criteria:
- Age ≥ 12 years
- Lingual weakness as measured by quantitative muscle testing (≤ 5% lower limit of normal for stratified age)
- Confirmed 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 LMT regimen
- Access to reliable internet connection
- Access to use of electronic device that allows for FaceTime, Jabber, or WebEx use
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
- Any current or past history of seizures.
- Head and neck cancer or radiation treatment to head/neck
- Inability to tolerate IOPI device tongue bulb
- Inability to give legally effective consent
- Inability to read and understand English
Sites / Locations
- Duke University Medical Center and affiliated practices
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Late Onset Pompe Disease who have tongue weakness
Arm Description
Outcomes
Primary Outcome Measures
Safety of lingual muscle training
Safety measured by pain rating scale
Feasibility of lingual muscle training as measured by questionnaires
Secondary Outcome Measures
Change in lingual strength
Change from pre-test to post-test
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03255213
Brief Title
Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
Official Title
Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
March 21, 2018 (Actual)
Primary Completion Date
April 23, 2020 (Actual)
Study Completion Date
April 23, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Duke University
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 Lingual Muscle Therapy (LMT) in patients with Late-Onset Pompe Disease (LOPD) who have tongue weakness. The results of this study will help design future research studies about LMT in LOPD.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glycogen Storage Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Late Onset Pompe Disease who have tongue weakness
Arm Type
Experimental
Intervention Type
Other
Intervention Name(s)
Lingual Muscle Training
Intervention Description
Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device
Primary Outcome Measure Information:
Title
Safety of lingual muscle training
Description
Safety measured by pain rating scale
Time Frame
13 weeks
Title
Feasibility of lingual muscle training as measured by questionnaires
Time Frame
13 weeks
Secondary Outcome Measure Information:
Title
Change in lingual strength
Description
Change from pre-test to post-test
Time Frame
baseline, 13 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria:
Age ≥ 12 years
Lingual weakness as measured by quantitative muscle testing (≤ 5% lower limit of normal for stratified age)
Confirmed 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 LMT regimen
Access to reliable internet connection
Access to use of electronic device that allows for FaceTime, Jabber, or WebEx use
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
Any current or past history of seizures.
Head and neck cancer or radiation treatment to head/neck
Inability to tolerate IOPI device tongue bulb
Inability to give legally effective consent
Inability to read and understand English
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jones Harrison, PhD
Organizational Affiliation
Duke University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Duke University Medical Center and affiliated practices
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
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Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
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