Continuation Protocol to Protocol BBCO-001

Muscular Dystrophy, Oculopharyngeal (OPMD) Clinical Trial

— HOPEMD  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02328482
• Other study ID #: BB-OPMD-301
• Status: Completed
• Phase: Phase 3
• Start date: January 2015
• Est. completion date: December 2017

Study information

• Verified date: February 2018
• Source: Bioblast Pharma Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: December 2017
• Est. primary completion date: October 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Oculopharyngeal
• Muscular Dystrophy, Oculopharyngeal (OPMD)

Intervention

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

Locations

Country	Name	City	State
Canada	Montreal Neurological Institute, McGill University	Montreal	Quebec

Sponsors (1)

Lead Sponsor	Collaborator
Bioblast Pharma Ltd.

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in disease markers	long term effect of Cabaletta on disease progression as measured by the changes in the disease markers	52 weeks
Primary	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	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