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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03255213
Recruitment Status : Recruiting
First Posted : August 21, 2017
Last Update Posted : May 15, 2019
Sponsor:
Information provided by (Responsible Party):
Duke University

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.

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Other: Lingual Muscle Training Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
Actual Study Start Date : March 21, 2018
Estimated Primary Completion Date : December 30, 2019
Estimated Study Completion Date : December 30, 2019


Arm Intervention/treatment
Experimental: Late Onset Pompe Disease who have tongue weakness Other: Lingual Muscle Training
Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device




Primary Outcome Measures :
  1. Safety of lingual muscle training [ Time Frame: 13 weeks ]
    Safety measured by pain rating scale

  2. Feasibility of lingual muscle training as measured by questionnaires [ Time Frame: 13 weeks ]

Secondary Outcome Measures :
  1. Change in lingual strength [ Time Frame: baseline, 13 weeks ]
    Change from pre-test to post-test



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

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): NCT03255213


Contacts
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Contact: Amy Walker 919-684-1732 amy.walker1@duke.edu

Locations
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United States, North Carolina
Duke University Medical Center and affiliated practices Recruiting
Durham, North Carolina, United States, 27710
Contact: Amy Walker    919-684-1732    AMY.WALKER1@DUKE.EDU   
Sponsors and Collaborators
Duke University
Investigators
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Principal Investigator: Jones Harrison, PhD Duke University

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Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT03255213     History of Changes
Other Study ID Numbers: Pro00077344
First Posted: August 21, 2017    Key Record Dates
Last Update Posted: May 15, 2019
Last Verified: May 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