Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

• Status: Completed
• Trial end date: 2016-04-01
• Target enrollment: 0
• Participant gender: All

Summary

The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioblast Pharma Ltd.

Criteria

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)