The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
UCLA
Los Angeles, California, United States
Tahseen Mozaffar
Orange, California, United States
Montreal Neurological Institute, McGill University
Montreal, Quebec, Canada
Hadassah medical center
Jerusalem, Israel
Safety Lab Evaluations
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
Time frame: 24 weeks
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.
Time frame: 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.
Time frame: 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.
Time frame: 28 weeks
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