Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
Last updated on April 2022Recruitment
- 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