Continuation Protocol to Protocol BBCO-001 (HOPEMD)

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

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.

Study Overview

• Status: Completed
• Conditions: Muscular Dystrophy, Oculopharyngeal (OPMD)
• Intervention / Treatment: Drug: Tehalose 30gr

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
• Enrollment (Actual): 9
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Montreal, Quebec, Canada, H3A 2B4
      • Montreal Neurological Institute, McGill University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in disease markers	long term effect of Cabaletta on disease progression as measured by the changes in the disease markers	52 weeks
Change in swallowing quality of life	long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g	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.	52 weeks

Collaborators and Investigators

• Sponsor: Bioblast Pharma Ltd.
• Investigators: Principal Investigator: Bernard Brais, McGill University

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2015
• Primary Completion (Actual): October 1, 2017
• Study Completion (Actual): December 1, 2017

Study Registration Dates

• First Submitted: December 25, 2014
• First Submitted That Met QC Criteria: December 30, 2014
• First Posted (Estimate): December 31, 2014

Study Record Updates

• Last Update Posted (Actual): July 17, 2019
• Last Update Submitted That Met QC Criteria: July 15, 2019
• Last Verified: February 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Oculopharyngeal
• Other Study ID Numbers: BB-OPMD-301