The Safety and Tolerability Study With ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease

Summary

This is an open label, dose escalation clinic trial of ER2001 intravenous injection to evaluate safety, tolerability and pharmacokinetics of ascending single and multiple doses of intravenously administered ER2001 in patients with early manifest Huntington's Disease. Furthermore, pharmacodynamics in particular target engagement, and early clinical signs of efficacy will be assessed. This study will evaluate increasing doses of ER2001 in sequential cohorts. ER2001 was escalated over 4 dose levels . The planned duration of this treatment is 14 weeks.

Timeline

Start

2023-04-04

Primary completion

2025-01-31

Completion

2025-09-04

Publications

• Background Zhang L, Wu T, Shan Y, Li G, Ni X, Chen X, Hu X, Lin L, Li Y, Guan Y, Gao J, Chen D, Zhang Y, Pei Z, Chen X. Therapeutic reversal of Huntington's disease by in vivo self-assembled siRNAs. Brain. 2021 Dec 16;144(11):3421-3435. doi: 10.1093/brain/awab354.

Drugs

Indications