Recruitment

Recruitment Status
Completed
Estimated Enrollment
14

Inclusion Criteria

18 - 80 years (inclusive) of age
Males and females
Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
...
18 - 80 years (inclusive) of age
Males and females
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
Clinically and genetically diagnosed as OPMD

Exclusion Criteria

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

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 Criteria

18 - 80 years (inclusive) of age
Males and females
Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
...
18 - 80 years (inclusive) of age
Males and females
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
Clinically and genetically diagnosed as OPMD

Exclusion Criteria

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

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