[Translation] Phase I/IIa open-label, single-dose, dose-escalation and dose-expansion study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of YOLT-201 in patients with transthyretin amyloid polyneuropathy (ATTR-PN) and transthyretin amyloid cardiomyopathy (ATTR-CM)
主要目的:
评价在 ATTR-PN 和 ATTR-CM 患者中给予 YOLT-201 后的安全性和耐受性;
次要目的:(1)评价在ATTR-PN和ATTR-CM患者中给予YOLT-201后的药代动力学特征;
(2)评价在ATTR-PN和ATTR-CM患者中给予YOLT-201后的药效学特征;
(3)评价在ATTR-PN和ATTR-CM患者中给予YOLT-201后的初步有效性;
(4)评价在ATTR-PN和ATTR-CM患者中给予YOLT-201后的免疫原性。
探索性目的:
评价在ATTR-CM患者中给予YOLT-201后的99Tcm-PYP摄取
[Translation] Primary objectives:
To evaluate the safety and tolerability of YOLT-201 in patients with ATTR-PN and ATTR-CM;
Secondary objectives: (1) To evaluate the pharmacokinetic characteristics of YOLT-201 in patients with ATTR-PN and ATTR-CM;
(2) To evaluate the pharmacodynamic characteristics of YOLT-201 in patients with ATTR-PN and ATTR-CM;
(3) To evaluate the preliminary efficacy of YOLT-201 in patients with ATTR-PN and ATTR-CM;
(4) To evaluate the immunogenicity of YOLT-201 in patients with ATTR-PN and ATTR-CM.
Exploratory objectives:
To evaluate the uptake of 99Tcm-PYP in patients with ATTR-CM after YOLT-201 administration