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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
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.
First Affiliated Hospital of Guangzhou Medical University
Guangzhou, Guangdong, China
Incidence and Severity of adverse events (AEs) and serious adverse events (SAEs)
To evaluate the safety and tolerability of ER2001 injection.
Time frame: Approximately 4.5 months
Peak Plasma Concentration (Cmax)
To assess the Pharmacokinetics (PK) of ER2001 injection.
Time frame: Approximately 4.5 months
Terminal half-life (t1/2)
To assess the Pharmacokinetics (PK) of ER2001 injection.
Time frame: Approximately 4.5 months
Area under the plasma concentration versus time curve from time 0 to the last quantifiable concentration (AUC0-t)
To assess the Pharmacokinetics (PK) of ER2001 injection.
Time frame: Approximately 4.5 months
Maximum concentration (Cmax) in cerebrospinal fluid (CSF)
To assess the Pharmacokinetics (PK) of ER2001 injection.
Time frame: Approximately 4.5 months
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