Continuation Protocol to Protocol BBCO-001

NCT ID: NCT02328482

Last Updated: 2019-07-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-01-31
• Study Completion Date: 2017-12-31

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.

Conditions

Muscular Dystrophy, Oculopharyngeal (OPMD)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm 1

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

Group Type: EXPERIMENTAL

Tehalose 30gr

Intervention Type: DRUG

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

Arm 2

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

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Tehalose 30gr

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

Intervention Type: DRUG

Other Intervention Names

Cabellta

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bioblast Pharma Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Bernard Brais

Role: PRINCIPAL_INVESTIGATOR

McGill University

Locations

Montreal Neurological Institute, McGill University

Montreal, Quebec, Canada

Countries

Canada

Other Identifiers

BB-OPMD-301

Identifier Type: -

Identifier Source: org_study_id