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| ID | Type | Description | Link |
|---|---|---|---|
| 1R21AR069880-01 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) | NIH |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RMT therapy using modified RMT device | Device | The exercises provided by the RMT device is intended to strengthen breathing muscles. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 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. | 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change in maximum expiratory pressures (MEP) | Change pretest to posttest | baseline, 12 weeks, 6 months, 9 months |
| Change in 6-minute walk test (6MWT) | Change pretest to posttest |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Harrison Jones, PhD | Division of Head and Neck Surgery & Communication Sciences, Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31303277 | Derived | 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. |
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| ID | Term |
|---|---|
| D006009 | Glycogen Storage Disease Type II |
| ID | Term |
|---|---|
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
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| Sham-RMT therapy using modified RMT device | Device | The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles. |
|
| baseline, 12 weeks, 6 months, 9 months |
| Change in Gait, Stairs, Gower, and Chair (GSGC) scale | Change pretest to posttest | baseline, 12 weeks, 6 months, 9 months |
| Change in peak cough flow (PCF) | Change pretest to posttest | baseline, 12 weeks, 6 months, 9 months |
| Change in Rasch-built Pompe-specific Activity Score (R-PAct) | Change pretest to posttest | baseline, 12 weeks, 6 months, 9 months |
| Change in diaphragm thickness | change pretest to posttest | baseline, 12 weeks, 6 months, 9 months |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |