Recruitment

Recruitment Status
Completed
Estimated Enrollment
14

Inclusion Criteria

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

Exclusion Criteria

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

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

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

Exclusion Criteria

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

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
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