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HOPEMD: Continuation Protocol to Protocol BBCO-001

Sponsor
Bioblast Pharma Ltd. (Industry)
Overall Status
Completed
CT.gov ID
NCT02328482
Collaborator
(none)
9
Enrollment
1
Location
2
Arms
35
Actual Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Tehalose 30gr
 Phase 3

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 Design

Study Type:
Interventional
Actual Enrollment :
9 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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
Actual Study Start Date :
Jan 1, 2015
Actual Primary Completion Date :
Oct 1, 2017
Actual Study Completion Date :
Dec 1, 2017

Arms and Interventions

Arm Intervention/Treatment
Experimental: Arm 1

Trehalose 30 g for IV infusion administered every week over an additional 52 weeks

Drug: Tehalose 30gr
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
Other Names:
  • Cabellta

    		•
    No Intervention: Arm 2

    no-treatment concurrent control; follow-up over 52 weeks

    Outcome Measures

    Primary Outcome Measures

    1. Change in disease markers [52 weeks]

      long term effect of Cabaletta on disease progression as measured by the changes in the disease markers

    2. 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

    Secondary Outcome Measures

    1. 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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 80 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Montreal Neurological Institute, McGill University Montreal Quebec Canada H3A 2B4

    Sponsors and Collaborators

    • Bioblast Pharma Ltd.

    Investigators

    • Principal Investigator: Bernard Brais, McGill University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Bioblast Pharma Ltd.
    ClinicalTrials.gov Identifier:
    NCT02328482
    Other Study ID Numbers:
    • BB-OPMD-301
    First Posted:
    Dec 31, 2014
    Last Update Posted:
    Jul 17, 2019
    Last Verified:
    Feb 1, 2018
    Additional relevant MeSH terms:
    Muscular Dystrophies
    Muscular Dystrophy, Oculopharyngeal

    Study Results

    No Results Posted as of Jul 17, 2019