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

Oculopharyngeal Muscular Dystrophy Clinical Trial

— HOPEMD  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02015481
• Other study ID #: BBCO-001
• Status: Completed
• Phase: Phase 2
• First received: December 8, 2013
• Last updated: September 15, 2017
• Start date: February 2014
• Est. completion date: April 2016

Study information

• Verified date: September 2017
• Source: Bioblast Pharma Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: April 2016
• Est. primary completion date: April 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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)

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Oculopharyngeal
• Oculopharyngeal Muscular Dystrophy

Intervention

Drug:
• Cabaletta

Locations

Country	Name	City	State
Canada	Montreal Neurological Institute, McGill University	Montreal	Quebec
Israel	Hadassah medical center	Jerusalem
United States	UCLA	Los Angeles	California
United States	Tahseen Mozaffar	Orange	California

Sponsors (1)

Lead Sponsor	Collaborator
Bioblast Pharma Ltd.

Countries where clinical trial is conducted

United States,  Canada,  Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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