Title
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
Phase
Phase 2Lead Sponsor
Bioblast Pharma Ltd.Study Type
InterventionalStatus
Completed Results PostedIndication/Condition
Oculopharyngeal Muscular DystrophyIntervention/Treatment
trehalose ...Study Participants
25The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
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)
| Event Type | Organ System | Event Term | Cabaletta 30gr |
|---|
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
Penetration aspiration score results assessed by VFS comparing baseline, prior to treatment, to week 24. This is an 8 point scale. The higher the number the greater the risk of aspiration. The result reported is the difference from the baseline scores.
Summary of Quality of Life in Swallowing Disorders total symptom score results over time, change from baseline at weeks 12 and 24 This is a 100 point scale. The higher the number the better the quality of life.
Change from baseline in ice water drinking time, in seconds, at week 24. Times greater than 8 seconds to complete the drinking test are considered abnormal.
