Recruitment

Recruitment Status
Completed
Estimated Enrollment
14

Inclusion Criterias

18 - 80 years (inclusive) of age
Body Mass Index (BMI) <30 kg/m2
Males and females
...
18 - 80 years (inclusive) of age
Body Mass Index (BMI) <30 kg/m2
Males and females
Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
Clinically and genetically diagnosed as OPMD
Patients who provide written informed consent to participate in the study

Exclusion Criterias

Pregnant or currently lactating women
Weight loss of more than 10% in the last 12 months.
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.
...
Pregnant or currently lactating women
Weight loss of more than 10% in the last 12 months.
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.
Other neuromuscular diseases
Uncontrolled heart disease , CHF,
History of malignancy (except non-invasive skin malignancy)
Known hypersensitivity to any ingredients in the injection
Diabetes mellitus type 1 or 2
History of neck irradiation
Patient receiving anticoagulant treatment (e.g. warfarin)
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
Obesity (BMI≥ 30) and associated morbidity
Prior pharyngeal myotomy

Summary

Conditions
Oculopharyngeal Muscular Dystrophy
Type
Interventional
Phase
Phase 2
Design
  • Allocation: N/A
  • Intervention Model: Single Group Assignment
  • Masking: None (Open Label)
  • Primary Purpose: Treatment

Participation Requirements

Age
Between 18 years and 80 years
Gender
Both males and females

Inclusion Criterias

18 - 80 years (inclusive) of age
Body Mass Index (BMI) <30 kg/m2
Males and females
...
18 - 80 years (inclusive) of age
Body Mass Index (BMI) <30 kg/m2
Males and females
Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
Clinically and genetically diagnosed as OPMD
Patients who provide written informed consent to participate in the study

Exclusion Criterias

Pregnant or currently lactating women
Weight loss of more than 10% in the last 12 months.
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.
...
Pregnant or currently lactating women
Weight loss of more than 10% in the last 12 months.
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.
Other neuromuscular diseases
Uncontrolled heart disease , CHF,
History of malignancy (except non-invasive skin malignancy)
Known hypersensitivity to any ingredients in the injection
Diabetes mellitus type 1 or 2
History of neck irradiation
Patient receiving anticoagulant treatment (e.g. warfarin)
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
Obesity (BMI≥ 30) and associated morbidity
Prior pharyngeal myotomy

Locations

Montreal, Quebec, H3A 2B4
Orange, California, 92868
Los Angeles, California, 90095
Jerusalem, 91120
...
Montreal, Quebec, H3A 2B4
Orange, California, 92868
Los Angeles, California, 90095
Jerusalem, 91120

Tracking Information

NCT #
NCT02015481
Collaborators
Not Provided
Investigators
  • Principal Investigator: Yoseph Caraco, M.D Hadassah Medical Center Principal Investigator: Bernard Brais, MD McGill University
  • Yoseph Caraco, M.D Hadassah Medical Center Principal Investigator: Bernard Brais, MD McGill University