Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients (HOPEMD)
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|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|
|Primary Completion Date :||April 2016|
|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|