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RO7234292 intrathecal PK/PD study in early manifest Huntington's disease
NL-OMON49037Phase 1Completed
- Sponsor
- Roche
- Start
- Jan 2020
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of tominersen in Huntington disease (HD), sponsored by Roche. Primary endpoints: CSF and plasma concentrations of RO7234292, Change from baseline of mutant huntingtin concentrations in CSF and 1 more. Started Jan 2020; status: completed.
02
Drugs
tominersen
03
Conditions
Huntington disease (HD)
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