drugset / Trial / NCT07344519

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

NCT07344519 ↗

Phase 0 Completed 8 enrolled ExoRNA Bioscience
NaSingle-groupOpen-labelTreatment

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.

Timeline

Start
2024-06-25
Primary completion
2025-06-01
Completion
2026-01-31

Drugs

EvaluationDrugModalityDoseRoute
Subject ER2001 Unknown 0.08 mg/kg Intravenous
Subject ER2001 Unknown 0.16 mg/kg Intravenous
Subject ER2001 Unknown 0.32 mg/kg Intravenous

Indications