Overview

This trial is active, not recruiting.

Condition muscular dystrophy, oculopharyngeal (opmd)
Treatment cabaletta 30gr
Phase phase 3
Sponsor Bioblast Pharma Ltd.
Start date January 2015
End date August 2017
Trial size 9 participants
Trial identifier NCT02328482, BB-OPMD-301

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Cabaletta 30 g for IV infusion administered every week over an additional 52 weeks
cabaletta 30gr
Cabaletta 30 g for IV infusion administered every week over an additional 52 weeks
(No Intervention)
no-treatment concurrent control; follow-up over 52 weeks

Primary Outcomes

Measure
Change in disease markers
time frame: 52 weeks
Change in swallowing quality of life
time frame: 52 weeks

Secondary Outcomes

Measure
Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g
time frame: 52 weeks

Eligibility Criteria

Male or female participants from 18 years up to 80 years old.

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

Additional Information

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
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.
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Bioblast Pharma Ltd..