Tominersen in Manifest Huntington's (GENERATION HD1)

Phase 3 Terminated

Manifest Huntington's Disease

NCT ID NCT03761849 ↗

Phase Phase 3

Status Terminated

Sponsor Roche

Enrolment n = 791

Last updated 2026-04-30

Registered 2026-04-30

Target intervention

Tominersen (antisense oligonucleotide targeting HTT)

Primary endpoint

cUHDRS at 101 weeks

Summary

Halted March 2021 — exploratory analyses suggested potential harm from non-allele-specific HTT lowering. Reframed the field around earlier intervention + allele-selective targeting.

Protocol timeline

Phase I

Phase II

Phase III

Phase IV

Current status Terminated

Enrolled n = 791

View on ClinicalTrials.gov ↗

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