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Continuation Protocol to Protocol BBCO-001 (HOPEMD)

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ClinicalTrials.gov Identifier: NCT02328482
Recruitment Status : Completed
First Posted : December 31, 2014
Last Update Posted : July 17, 2019
Sponsor:
Information provided by (Responsible Party):
Bioblast Pharma Ltd.

Tracking Information
First Submitted Date  ICMJE December 25, 2014
First Posted Date  ICMJE December 31, 2014
Last Update Posted Date July 17, 2019
Actual Study Start Date  ICMJE January 2015
Actual Primary Completion Date October 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 30, 2014)
  • 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 Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 30, 2014)
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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Continuation Protocol to Protocol BBCO-001
Official Title  ICMJE 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
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophy, Oculopharyngeal (OPMD)
Intervention  ICMJE Drug: Tehalose 30gr
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
Other Name: Cabellta
Study Arms  ICMJE
  • Experimental: Arm 1
    Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
    Intervention: Drug: Tehalose 30gr
  • No Intervention: Arm 2
    no-treatment concurrent control; follow-up over 52 weeks
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 21, 2016)
9
Original Estimated Enrollment  ICMJE
 (submitted: December 30, 2014)
68
Actual Study Completion Date  ICMJE December 2017
Actual Primary Completion Date October 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02328482
Other Study ID Numbers  ICMJE BB-OPMD-301
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Bioblast Pharma Ltd.
Study Sponsor  ICMJE Bioblast Pharma Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Bernard Brais McGill University
PRS Account Bioblast Pharma Ltd.
Verification Date February 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP