A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.

Summary

This is a dose escalation and expansion clinical study to evaluate the safety, tolerability, PK profile and preliminary efficacy of ER2001 Injection vs. placebo in subjects with definitive diagnosis of early manifest HD. The study consists of a dose escalation phase (Part A, an open-label without placebo, which will be carried out firstly) and a dose expansion phase (Part B,randomized, blinded, placebo-controlled), both of which include a screening period (4 week prior to the first administration), a treatment period (for 6 consecutive weeks, once a week \[QW\] for 6 weeks), and a safety follow-up period (24 weeks).

Timeline

Start

2025-02-11

Primary completion

2026-04-01

Completion

2026-05-30

Drugs

Indications