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Trehalose for Oculopharyngeal Muscular Dystrophy
NCT04226924Phase 2
Key facts
- Status
- Withdrawn
- Start
- Jun 2017
- Primary completion
- Feb 2018
- Countries
- 1
- Sponsor
- Bioblast Pharma Ltd.
- Modality
- Drug Delivery Systems, DNA Intercalators/Alkylators, Metabolically Activated Prodrugs
- Drugs
- trehalose
- Conditions
- oculopharyngeal muscular dystrophy (OPMD)
01
Overview
Phase 2, randomised, double-blind trial in oculopharyngeal muscular dystrophy (OPMD) comparing Trehalose with Placebo (2 arms). Primary endpoint: Drinking Test Time at 24 weeks. Planned start Jun 2017; status: withdrawn.
02
Design
2 arms- Study design
- RandomizedDouble-blind
Trehalose
Trehalose 0.75 g/kg IV weekly
Placebo
Normal saline IV weekly
03
Who it enrols
Patients with genetically confirmed OPMD (PABPN1 mutation) and confirmed oropharyngeal dysfunction (abnormal drinking test and SSQ > 235). Excludes patients with diabetes, prior pharyngeal myotomy, or non-ambulatory status.
04
Endpoints
Primary
Drinking Test Time
Secondary
Muscle Strength Testing
Stair Climb Test
Timed Up and Go Test
30-Second Lift Test
EuroQol-5D-5L
Swallowing Quality of Life
Sydney Swallow Questionnaire
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