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

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).

NCT Number	NCT07339514
Lead Sponsor	ExoRNA Bioscience
Conditions	Huntington Disease
Enrollment	27 participants
Start Date	2025-02-11
Primary Completion	2026-04-01 (estimated)
Study Completion	2026-05-30 (estimated)
Updated on ClinicalTrials.gov	2026-01-14