Continuation Protocol to Protocol BBCO-001

Phase 3

Completed

• Conditions: Muscular Dystrophy, Oculopharyngeal (OPMD)
• Interventions: Drug: Tehalose 30gr

• Registration Number: NCT02328482
• Lead Sponsor: Bioblast Pharma Ltd.

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.

Study Timeline

• Study First Submitted: 2014-12-25
• Study First Submitted QC: 2014-12-30
• Study First Posted: 2014-12-31
• Study Start: 2015-01
• Primary Completion: 2017-10
• Study Completion: 2017-12
• Status Verified: 2018-02
• Last Update Submitted: 2019-07-15
• Last Update Posted: 2019-07-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm 1	Tehalose 30gr	Trehalose 30 g for IV infusion administered every week over an additional 52 weeks

Primary Outcome Measures

Name	Time	Method
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
Change in disease markers	52 weeks	long term effect of Cabaletta on disease progression as measured by the changes in the disease markers

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Montreal Neurological Institute, McGill University; 🇨🇦 Montreal, Quebec, Canada