Muscle Weakness Clinical Trial
— ExercisPompeOfficial title:
Safety and Effectiveness of Resistance Exercise Training in Patients With Late Onset Pompe Disease- A Pilot Study
Verified date | September 2019 |
Source | University of California, Irvine |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this research study is to determine if exercise will help improve muscle strength, endurance, and quality of life in individuals with Pompe disease. This is a research study to further define the outcome of patients with Pompe disease treated with a combined diet and exercise therapy.
Status | Completed |
Enrollment | 10 |
Est. completion date | September 24, 2019 |
Est. primary completion date | August 6, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years to 75 Years |
Eligibility |
Inclusion Criteria: 1. Able to fully consent to the protocol. 2. Physically able to perform resistance exercises for 12 weeks. 3. Patients with a confirmed diagnosis of Pompe disease. 4. Age 16 years to 75 years. 5. Currently receiving ERT. 6. Patient on ERT for at least 1year. Exclusion Criteria: 1. Patients with no confirmed diagnosis of Pompe disease, either by GAA enzyme deficiency from any tissue and/or molecular testing revealing two GAA gene mutations 2. Unable to walk or cycle 3. Unable to consent to the study/ procedures 4. Women who are pregnant or breastfeeding 5. Heart disease 6. Patients with any metal inside their body such as metallic clips used for vascular repairs and/or implanted devices such as cardiac pacemakers which would prevent them from doing the MRI. |
Country | Name | City | State |
---|---|---|---|
United States | UCI ICTS (Institute for Clinical and Translational Science) | Irvine | California |
Lead Sponsor | Collaborator |
---|---|
University of California, Irvine |
United States,
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Winkel LP, Van den Hout JM, Kamphoven JH, Disseldorp JA, Remmerswaal M, Arts WF, Loonen MC, Vulto AG, Van Doorn PA, De Jong G, Hop W, Smit GP, Shapira SK, Boer MA, van Diggelen OP, Reuser AJ, Van der Ploeg AT. Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up. Ann Neurol. 2004 Apr;55(4):495-502. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Isometric muscle strength change from week 8 through week 32 | Eight months | ||
Secondary | 6 Minute Walk Test change from week 8 through week 32 | Eight months | ||
Secondary | Maximum Inspiratory Pressures percent change from week 8 through week 32 | Eight months | ||
Secondary | MRC score change from week 8 through week 32 | Eight months | ||
Secondary | Muscle volume and texture change from week 8 through week 32 using MR Imaging | Eight months | ||
Secondary | Glycogen content change in quadriceps muscle from week 8 through week 32 | Eight months | ||
Secondary | GAA enzyme activity change from week 8 through week 32 | Eight months | ||
Secondary | HEX4 change from week 8 through week 32 | Eight months | ||
Secondary | Other spirometry parameters change from week 8 through week 32 | Eight months |
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