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

• ClinicalTrials.gov Identifier: NCT04622046
• Recruitment Status: Active, not recruiting
• First Posted: November 9, 2020
• Last Update Posted: April 10, 2024
• Sponsor: Alexion Pharmaceuticals, Inc.
• Collaborator: Eidos Therapeutics, a BridgeBio company
• Information provided by (Responsible Party): Alexion Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: November 6, 2020
• First Posted Date: November 9, 2020
• Last Update Posted Date: April 10, 2024
• Actual Study Start Date: November 13, 2020
• Actual Primary Completion Date: November 8, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 6, 2020)

• Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT) [ Time Frame: Baseline, Month 12 ]
• All-cause Mortality And Cardiovascular-related Hospitalization Over A 30-month Period [ Time Frame: Baseline through Month 30 ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 6, 2020)

• Change From Baseline To Month 30 Of Treatment In Distance Walked During The 6MWT [ Time Frame: Baseline, Month 30 ]
• Change From Baseline To Month 12 Of Treatment In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) [ Time Frame: Baseline, Month 12 ]
• Change From Baseline To Month 30 Of Treatment In The KCCQ-OS [ Time Frame: Baseline, Month 30 ]
• Incidence Of Treatment-emergent Serious Adverse Events (SAEs) And Adverse Events (AEs) [ Time Frame: Baseline through Month 12 ]
• Incidence Of Treatment-emergent SAEs And AEs [ Time Frame: Baseline through Month 30 ]
• Change From Baseline To Day 28 In Transthyretin (TTR) Stabilization [ Time Frame: Baseline, Day 28 ]
• Change From Baseline To Month 30 In TTR Stabilization [ Time Frame: Baseline, Month 30 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
• Official Title: 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)
• 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.
• 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.
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Symptomatic Transthyretin Amyloid Cardiomyopathy

Intervention

Drug: ALXN2060

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

Other Names:

• AG10
• Acoramidis

Study Arms

Experimental: ALXN2060

Participants will receive ALXN2060.

Intervention: Drug: ALXN2060

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: April 9, 2024): 25
• Original Estimated Enrollment (submitted: November 6, 2020): 22
• Estimated Study Completion Date: September 23, 2025
• Actual Primary Completion Date: November 8, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype.
2. 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.
3. New York Heart Association Class I-III symptoms due to ATTR-CM.
4. On stable doses of cardiovascular medical therapy.
5. Completed ≥ 150 meters on the 6MWT on 2 tests prior to Day 1.
6. Left ventricular (LV) wall (interventricular septum or LV posterior wall) thickness ≥ 12 millimeters.
7. Biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic pep (NT-proBNP) level ≥ 300 picograms/milliliter (pg/mL).

Exclusion Criteria:

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Japan

Administrative Information

• NCT Number: NCT04622046
• Other Study ID Numbers: ALXN2060-TAC-302
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Alexion Pharmaceuticals, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Alexion Pharmaceuticals, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Eidos Therapeutics, a BridgeBio company
• Investigators: Not Provided
• PRS Account: Alexion Pharmaceuticals, Inc.
• Verification Date: April 2024