Glycogen Storage Disease Type IINCT02801539
Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)
A study testing RMT therapy using modified RMT device in people with Glycogen Storage Disease Type II, run by Duke University at 1 site in United States.
Trial summary
This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glycogen Storage Disease Type II
- What is being tested?
- RMT therapy using modified RMT device
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Feb 2017to 17 Apr 2019
- 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
- Age ≥ 18 years
- 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 RMT regimen
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
- 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
Durham, North Carolina, 27710
About this study
Official title: Respiratory Muscle Training in Late-Onset Pompe Disease
This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to decide if sham-RMT is a useful control condition for RMT and to choose the best ways to measure the health benefits of RMT in LOPD.
- Conditions
- Glycogen Storage Disease Type II
- Being tested
- RMT therapy using modified RMT device, Sham-RMT therapy using modified RMT device
- 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.
RMT therapy using modified RMT deviceDevice
The exercises provided by the RMT device is intended to strengthen breathing muscles.
Sham-RMT therapy using modified RMT deviceDevice
The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles.
| Study group | Role | What they receive |
|---|---|---|
| Respiratory muscle training (RMT) | Experimental | Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy. Receives: Device: RMT therapy using modified RMT device |
| Sham-RMT | Sham comparator | Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy. Receives: Device: Sham-RMT therapy using modified RMT device |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in respiratory strength
RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).
Measured: baseline, 12 weeks, 6 months, 9 months
Feasibility of sham-RMT, as measured by program adherence
As measured by program adherence (good is considered greater than or equal to 80%). Helps determines if a future efficacy trial is warranted and if sham-RMT may be a good control condition for a future trial.
Measured: 12 weeks
Secondary outcomes (6)
Change in maximum expiratory pressures (MEP)
Change pretest to posttest
Measured: baseline, 12 weeks, 6 months, 9 months
Change in 6-minute walk test (6MWT)
Change pretest to posttest
Measured: baseline, 12 weeks, 6 months, 9 months
Change in Gait, Stairs, Gower, and Chair (GSGC) scale
Change pretest to posttest
Measured: baseline, 12 weeks, 6 months, 9 months
Change in peak cough flow (PCF)
Change pretest to posttest
Measured: baseline, 12 weeks, 6 months, 9 months
Change in Rasch-built Pompe-specific Activity Score (R-PAct)
Change pretest to posttest
Measured: baseline, 12 weeks, 6 months, 9 months
Change in diaphragm thickness
change pretest to posttest
Measured: baseline, 12 weeks, 6 months, 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2016 (estimated)
- Study start2 Feb 2017
- Primary completion22 Oct 2018
- Study completion17 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Glycogen Storage Disease Type IIStudies 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
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Responsible party
- Sponsor
- Investigators
- Harrison Jones, PhD, Principal investigator, Division of Head and Neck Surgery & Communication Sciences, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02801539
- Sponsor's protocol ID
- Pro00068728
- Other IDs
- 1R21AR069880-01
- Status last verified
- Jul 2019
- First submitted
- 13 Jun 2016
- Last updated
- 30 Jul 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Jones HN, Kuchibhatla M, Crisp KD, Hobson Webb LD, Case L, Batten MT, Marcus JA, Kravitz RM, Kishnani PS. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): A protocol for a sham-controlled clinical trial. Mol Genet Metab. 2019 Aug;127(4):346-354. doi: 10.1016/j.ymgme.2019.05.001. Epub 2019 May 8.
PubMed 31303277
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Respiratory Muscle Training in Late-Onset Pompe Disease
- ClinicalTrials.gov ID
- NCT02801539
- Acronym / sponsor ID
- Pro00068728
- Conditions
- Glycogen Storage Disease Type II
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Study start
- 2 Feb 2017 (actual)
- Primary completion
- 22 Oct 2018 (actual)
- Study completion
- 17 Apr 2019 (actual)
- First posted
- 16 Jun 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.
This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to decide if sham-RMT is a useful control condition for RMT and to choose the best ways to measure the health benefits of RMT in LOPD.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Respiratory muscle training (RMT) | Experimental | Device: RMT therapy using modified RMT deviceSubjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy. |
| Sham-RMT | Sham comparator | Device: Sham-RMT therapy using modified RMT deviceSubjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18 years
- 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 RMT regimen
Key exclusion criteria (3 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
- Inability to give legally effective consent
- Inability to read and understand English
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 |
|---|---|---|
| RMT therapy using modified RMT deviceThe exercises provided by the RMT device is intended to strengthen breathing muscles. | Device | Respiratory muscle training (RMT) |
| Sham-RMT therapy using modified RMT deviceThe exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles. | Device | Sham-RMT |
Primary outcome
Change in respiratory strength
Time frame: baseline, 12 weeks, 6 months, 9 months
RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).
Feasibility of sham-RMT, as measured by program adherence
Time frame: 12 weeks
As measured by program adherence (good is considered greater than or equal to 80%). Helps determines if a future efficacy trial is warranted and if sham-RMT may be a good control condition for a future trial.
6 secondary outcomes
Change in maximum expiratory pressures (MEP)
Time frame: baseline, 12 weeks, 6 months, 9 months
Change in 6-minute walk test (6MWT)
Time frame: baseline, 12 weeks, 6 months, 9 months
Change in Gait, Stairs, Gower, and Chair (GSGC) scale
Time frame: baseline, 12 weeks, 6 months, 9 months
Change in peak cough flow (PCF)
Time frame: baseline, 12 weeks, 6 months, 9 months
Change in Rasch-built Pompe-specific Activity Score (R-PAct)
Time frame: baseline, 12 weeks, 6 months, 9 months
Change in diaphragm thickness
Time frame: baseline, 12 weeks, 6 months, 9 months
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 30 Jul 2019.
- Derived from this study: PubMed 31303277Jones HN, Kuchibhatla M, Crisp KD, Hobson Webb LD, Case L, Batten MT, Marcus JA, Kravitz RM, Kishnani PS. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): A protocol for a sham-controlled clinical trial. Mol Genet Metab. 2019 Aug;127(4):346-354. doi: 10.1016/j.ymgme.2019.05.001. Epub 2019 May 8.
- 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.
Harrison Jones, PhD principal investigator
Division of Head and Neck Surgery & Communication Sciences, 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 RMT therapy using modified RMT device in people with Glycogen Storage Disease Type II, 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 5 inclusion and 3 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 30 Jul 2019. 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. Some participants in this study may receive a placebo. 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?
Change in respiratory strength (time frame: baseline, 12 weeks, 6 months, 9 months). 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?
On the ClinicalTrials.gov record for NCT02801539, which also lists 1 linked publication.
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 30 Jul 2019.