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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02357225 |
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Date of registration:
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23/01/2015 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Pilot Study of Pyridostigmine in Pompe Disease
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Scientific title:
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Evaluation of Respiratory and Skeletal Muscle Functions in Response to Acetylcholinesterase Inhibitors in Pompe Disease |
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Date of first enrolment:
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August 2015 |
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Target sample size:
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2 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT02357225 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Early Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Barry J Byrne, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Florida |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Males or females between 8 and 60 years of age;
2. Diagnosis of Pompe disease (protein assay, genotyping, and positive clinical signs)
3. No contraindication to pyridostigmine
Exclusion Criteria:
1. Already receive pyridostigmine as part of their normal clinical care at screening
2. Are pregnant - participants will receive a urine pregnancy test at screening
3. Have received acute administration of antibiotic, corticosteroid, or neuromuscular
blockade medications within 30 days prior to screening
4. Any other concurrent medical condition which, in the opinion of the study team, would
make the subject inappropriate to participate in the assessments
Age minimum:
8 Years
Age maximum:
60 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Drug: Pyridostigmine Bromide
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Primary Outcome(s)
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Change in skeletal muscle function (6 Minute Walk Test)(QMT)
[Time Frame: Baseline, Day 90]
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Change in respiratory function (maximal inspiratory pressure, maximal expiratory pressure, and vital capacity)
[Time Frame: Baseline, Day 90]
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Change in quality of life [short form 36 (SF-36)]
[Time Frame: Baseline, Day 90]
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Evaluate the acute effects of pyridostigmine on neuromuscular junction transmission (Single-fiber EMG)
[Time Frame: Baseline]
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Secondary ID(s)
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IRB201200154
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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