Glycogen Storage DiseaseNCT03255213
Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
A study testing Lingual Muscle Training in people with Glycogen Storage Disease, run by Duke University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Glycogen Storage Disease
- What is being tested?
- Lingual Muscle Training
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Mar 2018to 23 Apr 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center and affiliated practices
Durham, North Carolina, 27710
About this study
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
- Being tested
- Lingual Muscle Training
- Medical subject headings
- Glycogen Storage Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lingual Muscle TrainingOther
Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device
| Study group | Role | What they receive |
|---|---|---|
| Late Onset Pompe Disease who have tongue weakness | Experimental | Not described Receives: Other: Lingual Muscle Training |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of lingual muscle training
Safety measured by pain rating scale
Measured: 13 weeks
Feasibility of lingual muscle training as measured by questionnaires
Measured: 13 weeks
Secondary outcomes (1)
Change in lingual strength
Change from pre-test to post-test
Measured: baseline, 13 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2017
- Study start21 Mar 2018
- Primary completion23 Apr 2020
- Study completion23 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage DiseaseStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jones Harrison, PhD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03255213
- Sponsor's protocol ID
- Pro00077344
- Status last verified
- Jul 2021
- First submitted
- 3 Aug 2017
- Last updated
- 28 Jul 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
- ClinicalTrials.gov ID
- NCT03255213
- Acronym / sponsor ID
- Pro00077344
- Conditions
- Glycogen Storage Disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Study start
- 21 Mar 2018 (actual)
- Primary completion
- 23 Apr 2020 (actual)
- Study completion
- 23 Apr 2020 (actual)
- First posted
- 21 Aug 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Late Onset Pompe Disease who have tongue weakness | Experimental | Other: Lingual Muscle Training |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lingual Muscle TrainingLingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device | Other | Late Onset Pompe Disease who have tongue weakness |
Primary outcome
Safety of lingual muscle training
Time frame: 13 weeks
Safety measured by pain rating scale
Feasibility of lingual muscle training as measured by questionnaires
Time frame: 13 weeks
1 secondary outcome
Change in lingual strength
Time frame: baseline, 13 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jones Harrison, PhD principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Lingual Muscle Training in people with Glycogen Storage Disease, run by Duke University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of lingual muscle training (time frame: 13 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jul 2021.