Pompe DiseaseNCT02742298
Pompe Disease QMUS and EIM
A study testing Muscle ultrasound and Electrical Impedance Myography in people with Pompe Disease, run by Duke University at 1 site in United States.
Trial summary
Study Objectives: 1. Determine the correlation between quantitative muscle ultrasound (QMUS), electrical impedance myography (EIM) and currently accepted measures of physical function. 2. Determine the reliability of EIM measures performed in the home through use of a hand held device. 3. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease
- What is being tested?
- Muscle ultrasound and Electrical Impedance Myography
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Oct 2016to 31 Jul 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetically confirmed diagnosis of Pompe disease.
Who cannot join
- Age <18 years
- Inability to provide informed consent.
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center
Durham, North Carolina, 27710
About this study
Official title: Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
Study Objectives:
- Determine the correlation between quantitative muscle ultrasound (QMUS), electrical impedance myography (EIM) and currently accepted measures of physical function.
- Determine the reliability of EIM measures performed in the home through use of a hand held device.
- Determine if QMUS and EIM can detect pre-clinical changes in Pompe disease.
- Conditions
- Pompe Disease
- Being tested
- Muscle ultrasound, Electrical Impedance Myography
- Keywords
- Neuromuscular Ultrasound, Electrical Impedance Myography
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Muscle ultrasoundDevice
Ultrasound images of limb muscles will be collected.
Electrical Impedance MyographyOther
A painless current passes through the skin and surface electrodes are used to record the impedance through muscle.
| Study group | Role | What they receive |
|---|---|---|
| QMUS | Other | All patients will undergo serial quantitative muscle ultrasound. Receives: Device: Muscle ultrasound |
| EIM | Other | All patients will undergo serial electrical impedance myography measures. Receives: Other: Electrical Impedance Myography |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change in muscle ultrasound echointensity as compared to baseline
Measured: Baseline, 1-2 years
Percent change in muscle electrical impedance myography as compared to baseline
Measured: Baseline, 1-2 years
Secondary outcomes (1)
Inter-rater reliability of muscle ultrasound and electrical impedance myography measures
Measured: 1-2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2016 (estimated)
- Study start24 Oct 2016
- Primary completion31 Jul 2020
- Study completion31 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe 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
- Device feasibility
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Lisa D Hobson-Webb, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02742298
- Sponsor's protocol ID
- Pro00072199
- Status last verified
- Feb 2020
- First submitted
- 11 Apr 2016
- Last updated
- 25 Sep 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
- ClinicalTrials.gov ID
- NCT02742298
- Acronym / sponsor ID
- Pro00072199
- Conditions
- Pompe Disease
- Keywords
- Neuromuscular Ultrasound, Electrical Impedance Myography
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- 24 Oct 2016 (actual)
- Primary completion
- 31 Jul 2020 (actual)
- Study completion
- 31 Jul 2020 (actual)
- First posted
- 19 Apr 2016 (estimated)
- 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.
Study Objectives:
- Determine the correlation between quantitative muscle ultrasound (QMUS), electrical impedance myography (EIM) and currently accepted measures of physical function.
- Determine the reliability of EIM measures performed in the home through use of a hand held device.
- Determine if QMUS and EIM can detect pre-clinical changes in Pompe disease.
Study design
- Primary purpose
- Device feasibility
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| QMUS | Other | Device: Muscle ultrasoundAll patients will undergo serial quantitative muscle ultrasound. |
| EIM | Other | Other: Electrical Impedance MyographyAll patients will undergo serial electrical impedance myography measures. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Genetically confirmed diagnosis of Pompe disease.
Key exclusion criteria (2 listed)
- Age <18 years
- Inability to provide informed 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 |
|---|---|---|
| Muscle ultrasoundUltrasound images of limb muscles will be collected. | Device | QMUS |
| Electrical Impedance MyographyA painless current passes through the skin and surface electrodes are used to record the impedance through muscle. | Other | EIM |
Primary outcome
Percent change in muscle ultrasound echointensity as compared to baseline
Time frame: Baseline, 1-2 years
Percent change in muscle electrical impedance myography as compared to baseline
Time frame: Baseline, 1-2 years
1 secondary outcome
Inter-rater reliability of muscle ultrasound and electrical impedance myography measures
Time frame: 1-2 years
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 25 Sep 2020.
- 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.
Lisa D Hobson-Webb, MD 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 Muscle ultrasound and Electrical Impedance Myography in people with Pompe 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 1 inclusion and 2 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 25 Sep 2020. 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?
Percent change in muscle ultrasound echointensity as compared to baseline (time frame: Baseline, 1-2 years). 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 25 Sep 2020.