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

NCT03255213 | Completed

• 1 other identifier: Pro00077344
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Glycogen Storage Disease

Outcome Measures

Primary Outcomes (2)

• Safety of lingual muscle training

  Safety measured by pain rating scale

  13 weeks

• Feasibility of lingual muscle training as measured by questionnaires

  13 weeks

Secondary Outcomes (1)

• Change in lingual strength

  baseline, 13 weeks

Study Arms (1)

Late Onset Pompe Disease who have tongue weakness

EXPERIMENTAL

Other: Lingual Muscle Training

Interventions

Lingual Muscle Training OTHER

Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device

Late Onset Pompe Disease who have tongue weakness

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Duke University: lead

Study Sites (1)

Duke University Medical Center and affiliated practices

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Glycogen Storage Disease

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Jones Harrison, PhD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2017
• First Posted: August 21, 2017
• Study Start: March 21, 2018
• Primary Completion: April 23, 2020
• Study Completion: April 23, 2020
• Last Updated: July 28, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)