Muscular Dystrophy, Oculopharyngeal (OPMD)
Conditions
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.
Interventions
DRUGTehalose 30gr
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
Sponsors
Bioblast Pharma Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Adult men and women who participated and completed study BBCO-001 2. Clinical and genetic diagnosis of OPMD 3. Able to provide written informed consent to participate in this study 4. Able to understand the requirements of the study and willing to comply with the requirements of the study
Exclusion criteria
1. Pregnant or lactating 2. Currently receiving anticoagulant treatment (e.g., warfarin) 3. Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety 4. Known hypersensitivity to any ingredient in the Cabaletta IV infusion 5. 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in disease markers | 52 weeks | long term effect of Cabaletta on disease progression as measured by the changes in the disease markers |
| Change in swallowing quality of life | 52 weeks | long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g | 52 weeks | 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. |
Countries
Canada
Outcome results
None listed