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

Primary Purpose

Oculopharyngeal Muscular Dystrophy

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Cabaletta
Sponsored by
Bioblast Pharma Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Oculopharyngeal Muscular Dystrophy focused on measuring Case control, safety, tolerability, efficacy.

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • UCLA
  • Tahseen Mozaffar
  • Montreal Neurological Institute, McGill University
  • Hadassah medical center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Cabaletta 30gr.

Arm Description

weekly IV of Cabaletta 30gr.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
December 8, 2013
Last Updated
September 15, 2017
Sponsor
Bioblast Pharma Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT02015481
Brief Title
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
Acronym
HOPEMD
Official Title
Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
Study Type
Interventional

2. Study Status

Record Verification Date
September 2017
Overall Recruitment Status
Completed
Study Start Date
February 2014 (undefined)
Primary Completion Date
April 2016 (Actual)
Study Completion Date
April 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioblast Pharma Ltd.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Oculopharyngeal Muscular Dystrophy
Keywords
Case control, safety, tolerability, efficacy.

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cabaletta 30gr.
Arm Type
Experimental
Arm Description
weekly IV of Cabaletta 30gr.
Intervention Type
Drug
Intervention Name(s)
Cabaletta
Primary Outcome Measure Information:
Title
Safety Lab Evaluations
Description
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
Time Frame
24 weeks
Secondary Outcome Measure Information:
Title
Drinking Test Score
Description
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.
Time Frame
24 weeks
Title
Videofluoroscopy (VFS) Score
Description
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.
Time Frame
24 Weeks
Title
SWAL-QOL, Swallowing Quality of Life Questionnaire
Description
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.
Time Frame
28 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yoseph Caraco, M.D
Organizational Affiliation
Hadassah Medical Organization
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Bernard Brais, MD
Organizational Affiliation
McGill University
Official's Role
Principal Investigator
Facility Information:
Facility Name
UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Tahseen Mozaffar
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Montreal Neurological Institute, McGill University
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
Facility Name
Hadassah medical center
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel

12. IPD Sharing Statement

Learn more about this trial

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

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