NCT07344519

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

Open-Label, Dose Escalation Early Phase 1 Study of ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease.

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: ExoRNA Bioscience · Industry
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The study 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 (12 weeks). Participants will receive 6 injections at the same dose level used in the NCT06024265 study, once a week over a period of 6 consecutive weeks. Planned doses for increase in subsequent cohorts are 0.08, 0.16, and 0.32 mg/kg. Decision to proceed to the next higher dose cohort will be based upon a safety review of the included participants. Treatment will continue until intolerable toxicity or as per patient preference.

Conditions

• Huntington Disease

Interventions

Type	Name	Description
DRUG	ER2001 injection	The minimum initial dose is 0.08mg/kg, then escalate to 0.16mg/kg, and 0.32mg/kg. The planned duration of the treatment is 6 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.

Timeline

• Start date: 2024-06-25
• Primary completion: 2025-06-01
• Completion: 2026-01-31
• First posted: 2026-01-15
• Last updated: 2026-01-15

Locations

1 site across 1 country: China