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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 : February 15, 2018
Sponsor:
Information provided by (Responsible Party):
Bioblast Pharma Ltd.

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
Muscular Dystrophy, Oculopharyngeal (OPMD) Drug: Cabaletta 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 Type : Interventional  (Clinical Trial)
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 Oculopharyngeal Muscular Dystrophy (OPMD) Patients Who Participated in Study BBCO-001
Actual Study Start Date : January 2015
Actual Primary Completion Date : October 2017
Actual Study Completion Date : December 2017


Arm Intervention/treatment
Experimental: Arm 1
Cabaletta 30 g for IV infusion administered every week over an additional 52 weeks
Drug: Cabaletta 30gr
Cabaletta 30 g for IV infusion administered every week over an additional 52 weeks

No Intervention: Arm 2
no-treatment concurrent control; follow-up over 52 weeks



Primary Outcome Measures :
  1. 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

  2. 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


Secondary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Responsible Party: Bioblast Pharma Ltd.
ClinicalTrials.gov Identifier: NCT02328482     History of Changes
Other Study ID Numbers: BB-OPMD-301
First Posted: December 31, 2014    Key Record Dates
Last Update Posted: February 15, 2018
Last Verified: February 2018

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Oculopharyngeal
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn