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

NCT04622046 | Completed Phase 3 Results FDA Drug

• 1 other identifier: ALXN2060-TAC-302
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 11

Brief 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.

Conditions

Symptomatic Transthyretin Amyloid Cardiomyopathy

Outcome Measures

Primary Outcomes (3)

• Part A: Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT)

  The 6MWT measures how far a participant can walk in 6 minutes. Change from baseline was calculated as the difference between the distance walked during the 6MWT at 12 months and the distance walked during the 6MWT at baseline. To determine the baseline, at least 2 6MWTs were conducted \> 24 hours to ≤ 3 weeks apart prior to the first dose of ALXN2060. The baseline 6MWT is the average of the total distance walked by participants for the 2 qualifying 6MWTs that met all the protocol-defined criteria. Least squares mean change from baseline data were adjusted for baseline measures and visits.

  Baseline, Month 12

• Parts A and B: Number of Cardiovascular (CV)-Related Hospitalizations Over A 30-month Period

  CV-related hospitalization was defined as the mean number of CV-related hospitalizations per participant per year over a 30-month period. CV-related hospitalizations were also reported as adverse events and were reviewed and adjudicated by an independent Clinical Events Committee (CEC).

  30 months

• All-cause Mortality (ACM) Over A 30-month Period

  ACM was assessed as time from the date of first initiation of study treatment to the date of death during a 30-month period, and was analyzed using Kaplan-Meier analysis. Data are reported for the number of participants with ACM over the 30-month period.

  30 months

Secondary Outcomes (5)

• Parts A and B: Change From Baseline In Distance Walked During The 6MWT

  Baseline, Months 6, 9, 18, 24 and 30

• Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)

  Baseline, Months 6, 9, 12, 18, 24 and 30

• Parts A and B: Number of Participants With Treatment-emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Treatment Discontinuation

  Up to Month 30

• Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration

  Baseline, pre-dose on Days 14, 28 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27 and 30

• Parts A and B: Change From Baseline In TTR Stabilization

  Baseline, Pre-dose Days 14, 28 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27 and 30

Study Arms (1)

ALXN2060

EXPERIMENTAL

Participants will receive ALXN2060.

Drug: ALXN2060

Interventions

ALXN2060 DRUG

ALXN2060 tablets will be administered twice daily at a dose of 800 milligrams.

Also known as: AG10, Acoramidis

ALXN2060

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype.
• History of heart failure evidenced by at least 1 prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by signs or symptoms of volume overload or elevated pressures or heart failure symptoms that required or requires ongoing treatment with a diuretic.
• New York Heart Association Class I-III symptoms due to ATTR-CM.
• On stable doses of cardiovascular medical therapy.
• Completed ≥ 150 meters on the 6MWT on 2 tests prior to Day 1.
• Left ventricular (LV) wall (interventricular septum or LV posterior wall) thickness ≥ 12 millimeters.
• Biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic pep (NT-proBNP) level ≥ 300 picograms/milliliter (pg/mL).

You may not qualify if:

• Acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening.
• Hemodynamic instability at screening.
• Likely to undergo heart transplantation within a year of screening.
• Current treatment with marketed drug products and other investigational agents for the treatment of ATTR-CM.
• Current treatment with calcium channel blockers with conduction system effects (for example, verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed.
• Confirmed diagnosis of light-chain (AL) amyloidosis.
• Biomarkers of myocardial wall stress: NT-ProBNP ≥ 8,500 pg/mL.
• Measure of kidney function, estimated glomerular filtration rate by Modification of Diet in Renal Disease formula \< 30 mL/minute/1.73 meters squared.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Eidos Therapeutics, a BridgeBio company: collaborator

Study Sites (11)

Research Site

Bunkyō City, 113-8431, Japan

Location

Research Site

Fukuoka, 812-8582, Japan

Location

Research Site

Kumamoto, 860-8556, Japan

Location

Research Site

Kurume-shi, 830-0011, Japan

Location

Research Site

Matsumoto-shi, 390-8621, Japan

Location

Research Site

Nagoya, 466-8560, Japan

Location

Research Site

Nankoku-shi, 783-8505, Japan

Location

Research Site

Sagamihara-shi, 252-0375, Japan

Location

Research Site

Sapporo, 060-8543, Japan

Location

Research Site

Shinjuku-ku, 160-8582, Japan

Location

Research Site

Suita-shi, 564-8565, Japan

Location

Related Links

• Related Info

MeSH Terms

Interventions

attruby

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2020
• First Posted: November 9, 2020
• Study Start: November 13, 2020
• Primary Completion: November 8, 2023
• Study Completion: August 21, 2025
• Last Updated: June 10, 2026
• Results First Posted: December 16, 2024

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

JP (11)