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

Bioblast Pharma

Status and phase

Completed

Phase 2

Conditions

Oculopharyngeal Muscular Dystrophy

Treatments

Drug: Cabaletta

Study type

Interventional

Funder types

Industry

Identifiers

NCT02015481

BBCO-001

Details and patient eligibility

About

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

Enrollment

25 patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

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)