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

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

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

Study Overview

• Status: Completed
• Conditions: Oculopharyngeal Muscular Dystrophy
• Intervention / Treatment: Drug: Cabaletta

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Montreal, Quebec, Canada, H3A 2B4
      • Montreal Neurological Institute, McGill University
• Israel
  • Jerusalem, Israel, 91120
    • Hadassah Medical Center
• United States
  • California
    • Los Angeles, California, United States, 90095
      • UCLA
    • Orange, California, United States, 92868
      • Tahseen Mozaffar

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cabaletta 30gr.; weekly IV of Cabaletta 30gr.	Drug: Cabaletta

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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

Collaborators and Investigators

• Sponsor: Bioblast Pharma Ltd.
• Investigators: Principal Investigator: Bernard Brais, MD, McGill University; Principal Investigator: Yoseph Caraco, M.D, Hadassah Medical Organization

Study record dates

Study Major Dates

• Study Start: February 1, 2014
• Primary Completion (Actual): April 1, 2016
• Study Completion (Actual): April 1, 2016

Study Registration Dates

• First Submitted: December 8, 2013
• First Submitted That Met QC Criteria: December 13, 2013
• First Posted (Estimate): December 19, 2013

Study Record Updates

• Last Update Posted (Actual): October 17, 2017
• Last Update Submitted That Met QC Criteria: September 15, 2017
• Last Verified: September 1, 2017

More Information

Terms related to this study

• Keywords: Case control, safety, tolerability, efficacy.
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Oculopharyngeal
• Other Study ID Numbers: BBCO-001