Back to resultsContinuation Protocol to Protocol BBCO-001 (HOPEMD)
Primary Purpose
Muscular Dystrophy, Oculopharyngeal (OPMD)
Status
Completed
Phase
Phase 3
Locations
Canada
Study Type
Interventional
Intervention
Tehalose 30gr
Sponsored by
About this trial
This is an interventional treatment trial for Muscular Dystrophy, Oculopharyngeal (OPMD)
Eligibility Criteria
Inclusion Criteria:
- Adult men and women who participated and completed study BBCO-001
- Clinical and genetic diagnosis of OPMD
- Able to provide written informed consent to participate in this study
- Able to understand the requirements of the study and willing to comply with the requirements of the study
Exclusion Criteria:
- Pregnant or lactating
- Currently receiving anticoagulant treatment (e.g., warfarin)
- Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety
- Known hypersensitivity to any ingredient in the Cabaletta IV infusion
- Currently participating in another clinical trial (other than BBCO-001) or have completed an interventional trial less than 30 days prior to the planned treatment start date
Sites / Locations
- Montreal Neurological Institute, McGill University
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
Arm 1
Arm 2
Arm Description
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
no-treatment concurrent control; follow-up over 52 weeks
Outcomes
Primary Outcome Measures
Change in disease markers
long term effect of Cabaletta on disease progression as measured by the changes in the disease markers
Change in swallowing quality of life
long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life
Secondary Outcome Measures
Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g
The safety and tolerability will be evaluated by measuring the adverse events, vital signs, safety labs and physical examination during the entire study period. The number of events of change in the safety evaluations will be compared between the treatment and non treatment groups.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02328482
Brief Title
Continuation Protocol to Protocol BBCO-001
Acronym
HOPEMD
Official Title
A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001
Study Type
Interventional
2. Study Status
Record Verification Date
February 2018
Overall Recruitment Status
Completed
Study Start Date
January 2015 (Actual)
Primary Completion Date
October 2017 (Actual)
Study Completion Date
December 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioblast Pharma Ltd.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
Detailed Description
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
Eligible patients will be randomized to one of the following treatment arms:
Treatment Arm 1: active treatment; continuation of 30 g IV Cabaletta once a week over an additional 52 weeks.
Treatment Arm 2: no-treatment concurrent control; discontinuation of IV Cabaletta and follow-up over 52 weeks.
IV Cabaletta will be administered once a week to patients in Treatment Arm 1. All patients, regardless of treatment arm allocation, will undergo the same safety and efficacy assessments during the monthly site visits.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Oculopharyngeal (OPMD)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
9 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Arm 1
Arm Type
Experimental
Arm Description
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
Arm Title
Arm 2
Arm Type
No Intervention
Arm Description
no-treatment concurrent control; follow-up over 52 weeks
Intervention Type
Drug
Intervention Name(s)
Tehalose 30gr
Other Intervention Name(s)
Cabellta
Intervention Description
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
Primary Outcome Measure Information:
Title
Change in disease markers
Description
long term effect of Cabaletta on disease progression as measured by the changes in the disease markers
Time Frame
52 weeks
Title
Change in swallowing quality of life
Description
long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g
Description
The safety and tolerability will be evaluated by measuring the adverse events, vital signs, safety labs and physical examination during the entire study period. The number of events of change in the safety evaluations will be compared between the treatment and non treatment groups.
Time Frame
52 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:
Adult men and women who participated and completed study BBCO-001
Clinical and genetic diagnosis of OPMD
Able to provide written informed consent to participate in this study
Able to understand the requirements of the study and willing to comply with the requirements of the study
Exclusion Criteria:
Pregnant or lactating
Currently receiving anticoagulant treatment (e.g., warfarin)
Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety
Known hypersensitivity to any ingredient in the Cabaletta IV infusion
Currently participating in another clinical trial (other than BBCO-001) or have completed an interventional trial less than 30 days prior to the planned treatment start date
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bernard Brais
Organizational Affiliation
McGill University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Montreal Neurological Institute, McGill University
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
12. IPD Sharing Statement
Learn more about this trial
Continuation Protocol to Protocol BBCO-001
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