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 range50 Years – 70 Years
SexALL
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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 …