Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

Study Overview
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Brief Summary
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The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Official Title
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Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients

Conditions
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Oculopharyngeal Muscular Dystrophy

Intervention / Treatment
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* Drug: Cabaletta


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males and females 18 - 80 years (inclusive) of age Clinically and genetically diagnosed as OPMD 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 Exclusion Criteria: Diabetes mellitus type 1 or 2 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 Uncontrolled heart disease , CHF, Other neuromuscular diseases 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. History of malignancy (except non-invasive skin malignancy) History of neck irradiation Pregnant or currently lactating women Obesity (BMI≥ 30) and associated morbidity Prior pharyngeal myotomy Weight loss of more than 10% in the last 12 months. Known hypersensitivity to any ingredients in the injection Patient receiving anticoagulant treatment (e.g. warfarin)

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cabaletta 30gr.<br>weekly IV of Cabaletta 30gr. | Drug: Cabaletta<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety Lab Evaluations | Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 . | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Drinking Test Score | Change from baseline in ice water drinking time, in seconds, at week 24. Times greater than 8 seconds to complete the drinking test are considered abnormal. | 24 weeks | 
| Videofluoroscopy (VFS) Score | Penetration aspiration score results assessed by VFS comparing baseline, prior to treatment, to week 24. This is an 8 point scale. The higher the number the greater the risk of aspiration. The result reported is the difference from the baseline scores. | 24 Weeks | 
| SWAL-QOL, Swallowing Quality of Life Questionnaire | Summary of Quality of Life in Swallowing Disorders total symptom score results over time, change from baseline at weeks 12 and 24 This is a 100 point scale. The higher the number the better the quality of life. | 28 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Case control, safety, tolerability, efficacy.