Clinical Trial: Continuation Protocol to Protocol BBCO-001
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in Oculophar
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 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.
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.
Sponsor: Bioblast Pharma Ltd.
Current Primary Outcome:
- Change in disease markers [ Time Frame: 52 weeks ]long term effect of Cabaletta on disease progression as measured by the changes in the disease markers
- Change in swallowing quality of life [ Time Frame: 52 weeks ]long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life
Original Primary Outcome: Same as current
Current Secondary Outcome: Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g [ Time Frame: 52 weeks ]
Original Secondary Outcome: Same as current
Information By: Bioblast Pharma Ltd.
Dates:
Date Received: December 25, 2014
Date Started: January 2015
Date Completion: December 2017
Last Updated: November 21, 2016
Last Verified: November 2016