NCT06024265

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

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

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: ExoRNA Bioscience · Industry
• Sex: All
• Age: 25 Years – 55 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Participants will receive totally 8 intravenous injections of ER2001 on the first day of the study, after 2 weeks they will receive 6 injections at weekly intervals, followed by 1 injections at 6 weeks interval. The treatment period of 14 weeks will be followed by an observation phase of 84 days . A modified 3 plus 3 dose-escalation design will be employed with escalating rules set according to the incidence of dose-limiting toxicity (DLT). The first cohort of 1-6 participants will receive a low dose. After safety analysis the next cohort of 3-6 will received a higher dose. Planned doses for increase in subsequent cohorts are 0.04, 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 0.04mg/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.

Timeline

• Start date: 2023-04-04
• Primary completion: 2025-01-31
• Completion: 2025-09-04
• First posted: 2023-09-06
• Last updated: 2025-12-19

Locations

1 site across 1 country: China