Clinical Trial

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

Completed Early Phase 1
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Summary
This is an open label, dose escalation clinic trial to evaluate safety, tolerability and pharmacokinetics with ER2001 Intravenous Injection repetitive treatment in Huntington's Disease patients who Completed Study ER2001-001（NCT06024265）. Furthermore, pharmacodynamics in particular target engagement, and clinical signs of efficacy will be assessed. This study will evaluate increasing doses of ER2001 in sequential cohorts. ER2001 was escalated over 3 dose levels . The planned duration of this additional treatment is 6 weeks for one course.
Protocol Amendment History 2 changes
critical Trial completed 2026-05-23
notable Enrollment reduced: 9 -> 8 participants 2026-05-23
Trial Details
NCT Number NCT07344519
Lead Sponsor ExoRNA Bioscience
Conditions Huntington Disease
Enrollment 8 participants
Start Date 2024-06-25
Primary Completion 2025-06-01 (estimated)
Study Completion 2026-01-31 (estimated)
Updated on ClinicalTrials.gov 2026-05-22