Continuation Protocol to Protocol BBCO-001 (HOPEMD)

Bioblast Pharma

Status and phase: Completed; Phase 3

Conditions: Muscular Dystrophy, Oculopharyngeal (OPMD)

Treatments: Drug: Tehalose 30gr

Study type: Interventional

Funder types: Industry

Identifiers

NCT02328482

BB-OPMD-301

Details and patient eligibility

About

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.

Full 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.

Enrollment: 9 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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9 participants in 2 patient groups

Arm 1

Experimental group

Description:

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

Treatment:

Drug: Tehalose 30gr

Arm 2

No Intervention group

Description:

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