Continuation Protocol to Protocol BBCO-001

Bioblast Pharma Ltd.9 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Muscular Dystrophy, Oculopharyngeal (OPMD)

Interventions

Tehalose 30grDRUG

Trehalose 30 g for IV infusion administered every week over an additional 52 weeks

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (1)

Montreal Neurological Institute, McGill University

Montreal, Quebec, Canada

Outcomes

Primary Outcomes

Change in disease markers

long term effect of Cabaletta on disease progression as measured by the changes in the disease markers

Time frame: 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

Time frame: 52 weeks

Secondary Outcomes

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.

Time frame: 52 weeks

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.