Recruitment

Recruitment Status
Completed
Estimated Enrollment
14

Inclusion Criteria

Patients who provide written informed consent to participate in the study
Body Mass Index (BMI) <30 kg/m2
Males and females
...
Patients who provide written informed consent to participate in the study
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)
18 - 80 years (inclusive) of age
Clinically and genetically diagnosed as OPMD

Exclusion Criteria

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

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

Patients who provide written informed consent to participate in the study
Body Mass Index (BMI) <30 kg/m2
Males and females
...
Patients who provide written informed consent to participate in the study
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)
18 - 80 years (inclusive) of age
Clinically and genetically diagnosed as OPMD

Exclusion Criteria

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

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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