Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients (HOPEMD)
|ClinicalTrials.gov Identifier: NCT02015481|
Recruitment Status : Completed
First Posted : December 19, 2013
Results First Posted : August 23, 2017
Last Update Posted : October 17, 2017
|Condition or disease||Intervention/treatment||Phase|
|Oculopharyngeal Muscular Dystrophy||Drug: Cabaletta||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients|
|Study Start Date :||February 2014|
|Actual Primary Completion Date :||April 2016|
|Actual Study Completion Date :||April 2016|
Experimental: Cabaletta 30gr.
weekly IV of Cabaletta 30gr.
- Safety Lab Evaluations [ Time Frame: 24 weeks ]Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
- Drinking Test Score [ Time Frame: 24 weeks ]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.
- Videofluoroscopy (VFS) Score [ Time Frame: 24 Weeks ]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.
- SWAL-QOL, Swallowing Quality of Life Questionnaire [ Time Frame: 28 weeks ]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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02015481
|United States, California|
|Los Angeles, California, United States, 90095|
|Orange, California, United States, 92868|
|Montreal Neurological Institute, McGill University|
|Montreal, Quebec, Canada, H3A 2B4|
|Hadassah medical center|
|Jerusalem, Israel, 91120|
|Principal Investigator:||Yoseph Caraco, M.D||Hadassah Medical Center|
|Principal Investigator:||Bernard Brais, MD||Montreal Neurological Institute, McGill University|