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01
Overview
Phase 2, randomised, double-blind trial of votoplam in Huntington's disease (HD) (2 arms), sponsored by Novartis. Primary endpoints: Treatment-emergent Adverse Events at 54 months and Blood Total Huntingtin Protein at 52 months. Started Aug 2023; status: active, not recruiting.
02
Design
2 arms- Study design
- RandomizedDouble-blind
Votoplam 5 mg
Participants receive votoplam 5 mg once daily orally for 48 months
Votoplam 10 mg
Participants receive votoplam 10 mg once daily orally for 48 months
03
Who it enrols
Participants who completed the Treatment Period in parent study PTC518-CNS-002-HD
04
Endpoints
Primary
Treatment-emergent Adverse Events
Blood Total Huntingtin Protein
Secondary
Caudate Volume
Composite Unified Huntington's Disease Rating Scale
Cerebrospinal Fluid Mutant Huntingtin Protein
Blood Mutant Huntingtin Protein
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