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BMN053 in Duchenne Muscular Dystrophy
NCT01957059Phase 2
Key facts
- Status
- Terminated
- Start
- Jun 2013
- Primary completion
- Aug 2016
- Countries
- 5
- Sponsor
- BioMarin Pharmaceutical
- Modality
- Modified DNA Oligonucleotides, Single-strand DNA, Antisense Oligonucleotides (ASOs)
- Drugs
- BMN-053
- Conditions
- Duchenne muscular dystrophy (DMD)
01
Overview
Phase 2, open-label trial of BMN-053 in Duchenne muscular dystrophy (DMD) (4 arms), sponsored by BioMarin Pharmaceutical. Primary endpoint: 6 Minute Walk Test at 48 weeks. Started Jun 2013; status: terminated.
02
Design
4 arms- Study design
- Non-randomizedOpen-label
Dose escalation phase
Single doses of BMN053 IV and SC.
Regimen selection
Weekly dosing to select optimal regimen.
48-week Treatment Phase
Long-term treatment at selected dose.
Dosing extension
Extension phase.
03
Endpoints
Primary
6 Minute Walk Test
Secondary
Muscle function
Muscle strength
Pulmonary function
Functional outcomes questionnaire
Adverse Events
Safety Laboratory
Cardiac function
Pharmacokinetic parameters
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