Continuation Protocol to Protocol BBCO-001

• Status: Completed
• Trial end date: 2017-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioblast Pharma Ltd.

Criteria

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