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 ]

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.


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