Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose (NCT04226924) | Clinical Trial Compass
WithdrawnPhase 2
Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
Stopped: Lack of funding for company
Canada0Started 2017-06-15
Plain-language summary
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.
Who can participate
Age range
50 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.