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A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants

Phase 1
Completed
Conditions
Healthy Participants
Interventions
Drug: Placebo
Registration Number
NCT04302064
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Brief Summary

To evaluate the safety and tolerability of single and potentially multiple doses of Eplontersen administered subcutaneously (SC) to healthy Japanese participants.

Detailed Description

This is a single-center, double-blinded, placebo-controlled study in up to 44 participants. Participants will be randomized to receive three subcutaneous single-ascending doses and potentially multiple doses of Eplontersen or placebo.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
24
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboSingle dose on Day 1 or multiple doses (every 4 weeks for 12 weeks) of Eplontersen-matching placebo administered SC.
EplontersenEplontersenSingle dose on Day 1 or multiple doses (every 4 weeks for 12 weeks) of Eplontersen administered SC.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants with Changes in Clinically Significant (CS) Laboratory Value AbnormalitiesUp to 92 days
Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by SeverityUp to 92 days
Percentage of Participants with TEAEs Potentially Related to Study DrugUp to 92 days
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

WCCT Global

🇺🇸

Cypress, California, United States

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