Continuation Protocol to Protocol BBCO-001

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.

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