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Lingual 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
Duke University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Glycogen Storage Disease

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

First Posted
August 3, 2017
Last Updated
July 27, 2021
Sponsor
Duke University
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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

Learn more about this trial

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

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