NCT02328482

Continuation Protocol to Protocol BBCO-001

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Bioblast Pharma Ltd. · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Muscular Dystrophy, Oculopharyngeal (OPMD)

Interventions

Type	Name	Description
DRUG	Tehalose 30gr	Trehalose 30 g for IV infusion administered every week over an additional 52 weeks

Timeline

• Start date: 2015-01-01
• Primary completion: 2017-10-01
• Completion: 2017-12-01
• First posted: 2014-12-31
• Last updated: 2019-07-17

Locations

1 site across 1 country: Canada