A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers
NCT Number: NCT06527755
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1
Conditions: Transthyretin-mediated Amyloidosis
Interventions: DRUG: Eplontersen Solution for Injection
Sponsor: AstraZeneca
Sex: ALL
Age: ADULT
Enrollment: 12
Study Design: Allocation: RANDOMIZED|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: OTHER
Start Date: 2024-08-06
Completion Date: 2024-12-17
Locations: Research Site, Beijing, 100029, China

Summary:
This is a Phase 1 study to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of eplontersen following single dosing in healthy Chinese participants. The objectives of the study are: to characterize the pharmacokinetic (PK) profiles and the pharmacodynamic (PD) profiles, and to evaluate the safety and tolerability and the immunogenicity of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants.

Primary Outcome Measures: PK parameters: Maximum Observed Concentration(Cmax), To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants, collect PK sample in pre-dose of Day 1 and 0.5 hours, 1 hours, 1.5 hours, 2 hours, 3 hours, 4 hours, 5 hours, 6 hours, 8 hours, 10 hours, 12 hours, 16 hours, 24 hours, 36 hours, 48 hours of post-dose, and in Day 8, Day 15, Day 29, Day 50, Day 71, Day 92|PK parameters: Time to Maximal Concentration (tmax), To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants, collect PK sample in pre-dose of Day 1 and 0.5 hours, 1 hours, 1.5 hours, 2 hours, 3 hours, 4 hours, 5 hours, 6 hours, 8 hours, 10 hours, 12 hours, 16 hours, 24 hours, 36 hours, 48 hours of post-dose, and in Day 8, Day 15, Day 29, Day 50, Day 71, Day 92|PK parameters: Plasma Half-life Associated with the Apparent Terminal Elimination Phase (t½λz), To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants, collect PK sample in pre-dose of Day 1 and 0.5 hours, 1 hours, 1.5 hours, 2 hours, 3 hours, 4 hours, 5 hours, 6 hours, 8 hours, 10 hours, 12 hours, 16 hours, 24 hours, 36 hours, 48 hours of post-dose, and in Day 8, Day 15, Day 29, Day 50, Day 71, Day 92|PK parameters: Area Under Plasma Concentration-time Curve (0-24 hours) (AUC0-24h), To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants, collect PK sample in pre-dose of Day 1 and 0.5 hours, 1 hours, 1.5 hours, 2 hours, 3 hours, 4 hours, 5 hours, 6 hours, 8 hours, 10 hours, 12 hours, 16 hours, 24 hours, 36 hours, 48 hours of post-dose, and in Day 8, Day 15, Day 29, Day 50, Day 71, Day 92|PK parameters: Area Under Plasma Concentration-time Curve (0-168 hours) (AUC0-168h), To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants, collect PK sample in pre-dose of Day 1 and 0.5 hours, 1 hours, 1.5 hours, 2 hours, 3 hours, 4 hours, 5 hours, 6 hours, 8 hours, 10 hours, 12 hours, 16 hours, 24 hours, 36 hours, 48 hours of post-dose, and in Day 8, Day 15, Day 29, Day 50, Day 71, Day 92
Secondary Outcome Measures: The change and percent change from baseline in serum (Transthyretin) TTR levels at specified timepoints, To characterize the pharmacodynamic (PD) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participant, collect PD sample in screening visit, pre-dose of Day 1 and Day 8, Day 15, Day 29, Day 50, Day 71, Day 92