Lingual Muscle Training in Late-Onset Pompe Disease (LOPD) | NCT03255213

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

NCT03255213

Sponsor

Duke University

Status

Completed

Last Update Posted

Jul 28, 2021Actual

Enrollment

5Actual

Phase

Not Applicable

Conditions

Glycogen Storage Disease

Interventions

Lingual Muscle Training

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2021

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Mar 21, 2018Actual

Primary Completion Date

Apr 23, 2020Actual

Completion Date

Apr 23, 2020Actual

First Submitted Date

Aug 3, 2017

First Submission Date that Met QC Criteria

Aug 17, 2017

First Posted Date

Aug 21, 2017Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 27, 2021

Last Update Posted Date

Jul 28, 2021Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

5Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Late Onset Pompe Disease who have tongue weakness	Experimental		Other: Lingual Muscle Training

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Lingual Muscle Training	Other	Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device	Late Onset Pompe Disease who have tongue weakness

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
Change in lingual strength	Change from pre-test to post-test	baseline, 13 weeks

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

12 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Jones Harrison, PhD	Duke University	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Duke University Medical Center and affiliated practices		Durham	North Carolina	27710	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D006008	Glycogen Storage Disease

Ancestor Terms

ID	Term
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided