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

Phase 2

Completed

• Conditions: Oculopharyngeal Muscular Dystrophy
• Interventions: Drug: Cabaletta

• Registration Number: NCT02015481
• Lead Sponsor: Bioblast Pharma Ltd.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-12-08
• Study First Submitted QC: 2013-12-13
• Study First Posted: 2013-12-19
• Study Start: 2014-02
• Primary Completion: 2016-04
• Study Completion: 2016-04
• Results First Submitted: 2017-07-21
• Results First Submitted QC: 2017-07-21
• Results First Posted: 2017-08-23
• Status Verified: 2017-09
• Last Update Submitted: 2017-09-15
• Last Update Posted: 2017-10-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cabaletta 30gr.	Cabaletta	weekly IV of Cabaletta 30gr.

Primary Outcome Measures

Name	Time	Method
Safety Lab Evaluations	24 weeks	Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .

Secondary Outcome Measures

Name	Time	Method
Drinking Test Score	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	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	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.

Trial Locations

Locations (4)

Montreal Neurological Institute, McGill University; 🇨🇦 Montreal, Quebec, Canada

UCLA; 🇺🇸 Los Angeles, California, United States

Hadassah medical center; 🇮🇱 Jerusalem, Israel

Tahseen Mozaffar; 🇺🇸 Orange, California, United States