CompletedPhase 3

Continuation Protocol to Protocol BBCO-001

Canada9 participantsStarted 2015-01

Plain-language 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.

Who can participate

Age range18 Years – 80 Years

SexALL

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Inclusion criteria

✓. Adult men and women who participated and completed study BBCO-001
✓. Clinical and genetic diagnosis of OPMD
✓. Able to provide written informed consent to participate in this study
✓. Able to understand the requirements of the study and willing to comply with the requirements of the study

Exclusion criteria

✕. Pregnant or lactating
✕. Currently receiving anticoagulant treatment (e.g., warfarin)
✕. Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety
✕. Known hypersensitivity to any ingredient in the Cabaletta IV infusion
✕. 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

What they're measuring

Change in disease markers

Timeframe: 52 weeks

Change in swallowing quality of life

Timeframe: 52 weeks

Trial details

NCT IDNCT02328482

SponsorBioblast Pharma Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-10

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