Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (… (NCT02015481) | Clinical Trial Compass
CompletedPhase 2
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
United States, Canada, Israel25 participantsStarted 2014-02
Plain-language summary
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
Who can participate
Age range18 Years – 80 Years
SexALL
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Inclusion Criteria:
* Males and females
* 18 - 80 years (inclusive) of age
* Clinically and genetically diagnosed as OPMD
* Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
* Patients who provide written informed consent to participate in the study
* Body Mass Index (BMI) \<30 kg/m2
Exclusion Criteria:
* Diabetes mellitus type 1 or 2
* Other major diseases, e.g.: renal failure (creatinine clearance \<60ml/min), liver failure and chronic liver diseases (e.g. hepatitis B or C) , HIV carriers, tuberculosis, SLE, rheumatoid polyarthritis, sarcoidosis, collagenosis
* Uncontrolled heart disease , CHF,
* Other neuromuscular diseases
* Other disorders associated with esophageal dysphagia: e.g. severe gastroesophageal reflux (GERD), esophageal stricture due to mechanical or chemical trauma, infection (e.g. esophageal moniliasis), drug-induced dysphagia (e.g. bisphosphonates), esophageal rings and webs, spastic motility disorders of the esophagus.
* History of malignancy (except non-invasive skin malignancy)
* History of neck irradiation
* Pregnant or currently lactating women
* Obesity (BMI≥ 30) and associated morbidity
* Prior pharyngeal myotomy
* Weight loss of more than 10% in the last 12 months.
* Known hypersensitivity to any ingredients in the injection
* Patient receiving anticoagulant treatment (e.g. warfarin)