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

NCT07339514 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 27

Last updated 2026-01-14

No results posted yet for this study

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).

Conditions

  • Huntington Disease

Interventions

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.

Sponsors & Collaborators

  • ExoRNA Bioscience

    lead INDUSTRY

Principal Investigators

  • jinsheng zeng, Ph.D · First Affiliated Hospital of Sun Yat-sen University

  • pingyi xu, Ph.D · First Affiliated Hospital of Guangzhou Medical University

  • huifang shang, Ph.D · West China Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
25 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-02-11
Primary Completion
2026-04-01
Completion
2026-05-30

Countries

  • China

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07339514 on ClinicalTrials.gov