drugset / Trial / NCT03728634

Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).

NCT03728634 ↗

Phase 1/2 Completed 47 enrolled Ionis Pharmaceuticals, Inc.
RandomizedParallel-groupDouble-blindTreatment

Summary

To evaluate the safety and tolerability, as well as the pharmacokinetic and pharmacodynamic profiles of single and multiple doses of Eplontersen administered subcutaneously to healthy volunteers and patients with Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).

Timeline

Start
2018-12-21
Primary completion
2020-02-20
Completion
2020-02-20

Drugs

EvaluationDrugModalityDoseRoute
Subject Eplontersen Antisense oligonucleotide 45 mg Subcutaneous
Subject Eplontersen Antisense oligonucleotide 60 mg Subcutaneous
Subject Eplontersen Antisense oligonucleotide 90 mg Subcutaneous
Subject Eplontersen Antisense oligonucleotide 120 mg Subcutaneous