Continuation Protocol to Protocol BBCO-001

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Muscular Dystrophy, Oculopharyngeal (OPMD)

Intervention / Treatment
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* Drug: Tehalose 30gr


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br>Trehalose 30 g for IV infusion administered every week over an additional 52 weeks | Drug: Tehalose 30gr<br>* Trehalose 30 g for IV infusion administered every week over an additional 52 weeks<br>* Other names: Cabellta;|
| No Intervention: Arm 2<br>no-treatment concurrent control; follow-up over 52 weeks | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 52 weeks |