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MechanismLAMP-2 modulators |
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Inactive Indication- |
Drug Highest PhasePhase 1 |
First Approval Ctry. / Loc.- |
First Approval Date- |
An Open-Label, Dose Escalation Early Phase 1 Study of ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease
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.
ER2001注射液治疗早期显性亨廷顿舞蹈病的随机、盲法、安慰剂对照的I期临床研究
[Translation] A randomized, blinded, placebo-controlled phase I clinical study of ER2001 injection in the treatment of early-stage dominant Huntington's disease
评价ER2001注射液在早期显性亨廷顿舞蹈病患者中的安全性和耐受性, 药代动力学,药效动力学,临床疗效,免疫原性及潜在的生物标志物等。
[Translation] To evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics, clinical efficacy, immunogenicity and potential biomarkers of ER2001 injection in patients with early-stage manifest Huntington's disease.
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