NCT07339514

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

A Randomized, Double-Blind, Placebo-Controlled Phase I Clinical Study of ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease.

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 27 (estimated)
• Sponsor: ExoRNA Bioscience · Industry
• Sex: All
• Age: 25 Years – 55 Years
• Healthy volunteers: Not accepted

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).

Detailed description

Two dose levels will be included in the dose escalation phase, and subjects will be in cohorts (Cohort 1, and Cohort 2) in sequence to receive the investigational drug. Either 3 or 6 subjects will be enrolled in each cohort. Dose escalation from the current cohort to the next cohort will be determined through joint review by the sponsor and the IDMC, typically based on the number of DLT(dose-limiting toxicity)s observed in subjects. If one DLT occurs in a cohort, additional 3 subjects will be enrolled in that cohort, with a total of 6 subjects. If one DLT occurs in the low-dose cohort while the administration is ongoing in the high-dose cohort, recruitment of subjects in the high-dose cohort should be held until all enrolled subjects have completed their dosing regimen and a safety assessment of at least 7 days after completion of the treatment. In addition, the IDMC will hold a meeting to decide whether further actions are necessary, e.g., dose interruption or reduction for subjects in the high-dose cohort. If two DLTs occur in a cohort, the dose will be discontinued in that cohort and any active higher dose cohorts. No more than one subject within the same cohort should initiate the treatment on the same day. Based on review of safety, tolerability, PK and exploratory PD results in the dose escalation phase, dose expansion will be performed in 1 or 2 cohort(s) at the recommended dose levels. For the dose expansion phase, 9 subjects (including 3 subjects in the placebo control group) will be enrolled for each dose level cohort.

Conditions

• Huntington Disease

Interventions

Type	Name	Description
DRUG	ER2001 intravenous injection	The drug dose is 0.08mg/kg, or 0.32mg/kg. The planned duration of each patient's treatment is 6 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.
DRUG	Placebo	Placebo Injection, The planned duration of the treatment is 6 weeks, and Placebo will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.

Timeline

• Start date: 2025-02-11
• Primary completion: 2026-04-01
• Completion: 2026-05-30
• First posted: 2026-01-14
• Last updated: 2026-01-14

Locations

3 sites across 1 country: China