Phase 3 N=25 Treatment

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

Symptomatic Transthyretin Amyloid Cardiomyopathy

Bottom Line

View on ClinicalTrials.gov: NCT04622046 ↗

Enrolled (actual)

Serious AEs

48.0%

Results posted

Dec 2024

Primary outcome: Primary: Part A: Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT) — -3.86 meters

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: ALXN2060 (Drug)
Age: Pediatric, Adult, Older Adult
Sex: All
Sponsor: Alexion Pharmaceuticals, Inc.
Primary completion: Nov 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Part A: Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT)	-3.86	—
PRIMARY Parts A and B: Number of Cardiovascular (CV)-Related Hospitalizations Over A 30-month Period	0.1329	—
PRIMARY All-cause Mortality (ACM) Over A 30-month Period	—	—
SECONDARY Parts A and B: Change From Baseline In Distance Walked During The 6MWT	-29.54; -23.12; -26.27; -24.89; -36.20	—
SECONDARY Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)	0.94; -1.73; 2.71; -5.34; -0.68; -6.97	—
SECONDARY Parts A and B: Number of Participants With Treatment-emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Treatment Discontinuation	25; 12; 2	—
SECONDARY Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration	9.73; 9.13; 10.64; 9.61; 11.16; 11.80	—
SECONDARY Parts A and B: Change From Baseline In TTR Stabilization	97.15; 97.29; 99.09; 98.56; 96.47; 100.89	—

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.

Eligibility Criteria

Inclusion Criteria

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).

Exclusion Criteria

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04622046). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.