Continuation Protocol to Protocol BBCO-001
HOPEMD
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
1 other identifier
interventional
9
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jan 2015
Typical duration for phase_3
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 25, 2014
CompletedFirst Posted
Study publicly available on registry
December 31, 2014
CompletedStudy Start
First participant enrolled
January 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2017
CompletedJuly 17, 2019
February 1, 2018
2.8 years
December 25, 2014
July 15, 2019
Conditions
Outcome Measures
Primary Outcomes (2)
Change in disease markers
long term effect of Cabaletta on disease progression as measured by the changes in the disease markers
52 weeks
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
52 weeks
Secondary Outcomes (1)
Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g
52 weeks
Study Arms (2)
Arm 1
EXPERIMENTALTrehalose 30 g for IV infusion administered every week over an additional 52 weeks
Arm 2
NO INTERVENTIONno-treatment concurrent control; follow-up over 52 weeks
Interventions
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Montreal Neurological Institute, McGill University
Montreal, Quebec, H3A 2B4, Canada
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Bernard Brais
McGill University
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 25, 2014
First Posted
December 31, 2014
Study Start
January 1, 2015
Primary Completion
October 1, 2017
Study Completion
December 1, 2017
Last Updated
July 17, 2019
Record last verified: 2018-02