NCT04622046

A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM

A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with symptomatic ATTR-CM administered on a background of stable heart failure therapy.

Detailed description

Participants will receive ALXN2060 for 12 months (Part A). Following the last visit (Month 12) of Part A, participants will continue the study in Part B, which will last for an additional 18 months (30 months from Day 1), during which all participants will continue to receive oral treatment with ALXN2060. Following completion of Month 30 assessments in Part B, participants will be offered the opportunity to continue to receive ALXN2060 in the Extension Period, which will last until ALXN2060 is approved in Japan or for up to 24 additional months, whichever occurs first.

Conditions

• Symptomatic Transthyretin Amyloid Cardiomyopathy

Interventions

Type	Name	Description
DRUG	ALXN2060	ALXN2060 tablets will be administered twice daily at a dose of 800 milligrams.

Timeline

• Start date: 2020-11-13
• Primary completion: 2023-11-08
• Completion: 2025-08-21
• First posted: 2020-11-09
• Last updated: 2026-01-09
• Results posted: 2024-12-16

Locations

11 sites across 1 country: Japan

Regulatory

• FDA-regulated drug study