Conditions: Huntington Disease
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 36
Healthy volunteers: no

Brief summary:
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362331 (SNP2). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP2-001.

Detailed description:

Arms:
arm 1: None

Interventions: intervention 1: WVE-120102
Intervention descriptions: intervention 1: WVE-120102 is a stereopure antisense oligonucleotide. It is administered monthly via intrathecal injection.

Protocol document:
Prot_SAP_000.pdf: