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

NCT06024265 · Status: COMPLETED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-12-19

No results posted yet for this study

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.

Conditions

  • Huntington Disease

Interventions

DRUG

ER2001 injection

The minimum initial dose is 0.04mg/kg, then escalate to 0.08mg/kg, 0.16mg/kg and 0.32mg/kg. The planned duration of the treatment is 14 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 3, 4, 5, 6, 7, 8, and 14.

Sponsors & Collaborators

  • ExoRNA Bioscience

    lead INDUSTRY

Principal Investigators

  • Xia Meng, Ph.D · ExoRNA Bioscience

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2023-04-04
Primary Completion
2025-01-31
Completion
2025-09-04

Countries

  • China

Study Locations

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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 NCT06024265 on ClinicalTrials.gov