Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose

• Status: Withdrawn
• Trial end date: 2018-08-15
• Target enrollment: 0
• Participant gender: All

Summary

BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioblast Pharma Ltd.

Criteria

Inclusion Criteria:

• Genetically confirmed OPMD with a (GCN)13 size PABPN1 mutation

• A score greater than 235 on the Sydney Swallow Questionnaire at screening

• Confirmation of oropharyngeal dysfunction by abnormal ice-cold water drinking test result, defined as drinking 80 cc of ice-cold water in ≥ 8 seconds at both drinking tests (at least 1 week apart) during the screening period

Exclusion Criteria:

• History of pharyngeal myotomy.

• Esophageal dilatation within the last 12 months.

• Treatment with botulinum toxin (any location) within 1 year prior to screening.

• Diagnosis of any other muscle disorder.

• Prior head and neck surgery or radiation.

• Oropharyngeal injury or oropharyngeal cancer.

• Other esophageal disease that may be the cause of the dysphagia.

• Previously diagnosed with diabetes or a hemoglobin A1c (HgbA1c) result > 6.0% at screening.

• Prior treatment with IV trehalose.

• Known hypersensitivity to trehalose.

• Non-ambulatory (Use of a cane or short leg braces are permitted).

• Prior history of stroke (ischemic or hemorrhagic).

• Pregnancy or breast feeding.

• History of alcohol or drug abuse within the last 5 years.

• Evidence of hepatitis B, hepatitis C, or HIV infection at screening.

• Currently receiving anti-coagulant treatment (e.g., warfarin, enoxaparin) other than anti-platelet treatments, which are not a reason for exclusion.

• Currently participating in another clinical trial or has completed an interventional trial less than 90 days prior to planned first dosing.