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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: |
NCT04226924 |
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Date of registration:
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15/07/2019 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
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Scientific title:
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A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of |
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Date of first enrolment:
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June 15, 2017 |
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Target sample size:
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0 |
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Recruitment status: |
Withdrawn |
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URL:
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https://clinicaltrials.gov/show/NCT04226924 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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Contacts
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Name:
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Bernard Brais, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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McGill University |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Genetically confirmed OPMD with a (GCN)13 size PABPN1 mutation
- A score greater than 235 on the Sydney Swallow Questionnaire at screening
- Confirmation of oropharyngeal dysfunction by abnormal ice-cold water drinking test
result, defined as drinking 80 cc of ice-cold water in = 8 seconds at both drinking
tests (at least 1 week apart) during the screening period
Exclusion Criteria:
- History of pharyngeal myotomy.
- Esophageal dilatation within the last 12 months.
- Treatment with botulinum toxin (any location) within 1 year prior to screening.
- Diagnosis of any other muscle disorder.
- Prior head and neck surgery or radiation.
- Oropharyngeal injury or oropharyngeal cancer.
- Other esophageal disease that may be the cause of the dysphagia.
- Previously diagnosed with diabetes or a hemoglobin A1c (HgbA1c) result > 6.0% at
screening.
- Prior treatment with IV trehalose.
- Known hypersensitivity to trehalose.
- Non-ambulatory (Use of a cane or short leg braces are permitted).
- Prior history of stroke (ischemic or hemorrhagic).
- Pregnancy or breast feeding.
- History of alcohol or drug abuse within the last 5 years.
- Evidence of hepatitis B, hepatitis C, or HIV infection at screening.
- Currently receiving anti-coagulant treatment (e.g., warfarin, enoxaparin) other than
anti-platelet treatments, which are not a reason for exclusion.
- Currently participating in another clinical trial or has completed an interventional
trial less than 90 days prior to planned first dosing.
Age minimum:
50 Years
Age maximum:
70 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Oculopharyngeal Muscular Dystrophy
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Intervention(s)
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Drug: Trehalose
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Primary Outcome(s)
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Drinking Test Time
[Time Frame: 24 weeks]
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Secondary Outcome(s)
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Stair Climb Test
[Time Frame: 24 weeks]
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EuroQol-5D-5L
[Time Frame: 24 weeks]
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Swallowing Quality of Life
[Time Frame: 24 weeks]
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Sydney Swallow Questionnaire
[Time Frame: 24 weeks]
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Timed Up and Go Test
[Time Frame: 24 weeks]
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30-Second Lift Test
[Time Frame: 24 weeks]
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Muscle Strength Testing
[Time Frame: 24 weeks]
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Secondary ID(s)
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BB-OPMD-202
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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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