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

Primary Purpose

Symptomatic Transthyretin Amyloid Cardiomyopathy

Status
Active
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
ALXN2060
Sponsored by
Alexion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Symptomatic Transthyretin Amyloid Cardiomyopathy

Eligibility Criteria

20 Years - 90 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Nagoya University Hospital
  • Kurume University Hospital
  • Kyushu University Hospital
  • Sapporo Medical University Hospital
  • Kitasato University Hospital
  • Kochi Medical School Hospital
  • Kumamoto University Hospital
  • Shinshu University Hospital
  • Okayama University Hospital
  • National Cerebral and Cardiovascular Center
  • Juntendo University Hospital
  • Keio University Hospital
  • The University of Tokyo Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ALXN2060

Arm Description

Participants will receive ALXN2060.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
November 6, 2020
Last Updated
December 12, 2022
Sponsor
Alexion
Collaborators
Eidos Therapeutics, a BridgeBio company
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1. Study Identification

Unique Protocol Identification Number
NCT04622046
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)
Study Type
Interventional

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 1, 2020 (Actual)
Primary Completion Date
May 31, 2024 (Anticipated)
Study Completion Date
May 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
Collaborators
Eidos Therapeutics, a BridgeBio company

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Symptomatic Transthyretin Amyloid Cardiomyopathy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
22 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
ALXN2060
Arm Type
Experimental
Arm Description
Participants will receive ALXN2060.
Intervention Type
Drug
Intervention Name(s)
ALXN2060
Other Intervention Name(s)
AG10, Acoramidis
Intervention Description
ALXN2060 tablets will be administered twice daily at a dose of 800 milligrams.
Primary Outcome Measure Information:
Title
Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT)
Time Frame
Baseline, Month 12
Title
All-cause Mortality And Cardiovascular-related Hospitalization Over A 30-month Period
Time Frame
Baseline through Month 30
Secondary Outcome Measure Information:
Title
Change From Baseline To Month 30 Of Treatment In Distance Walked During The 6MWT
Time Frame
Baseline, Month 30
Title
Change From Baseline To Month 12 Of Treatment In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Time Frame
Baseline, Month 12
Title
Change From Baseline To Month 30 Of Treatment In The KCCQ-OS
Time Frame
Baseline, Month 30
Title
Incidence Of Treatment-emergent Serious Adverse Events (SAEs) And Adverse Events (AEs)
Time Frame
Baseline through Month 12
Title
Incidence Of Treatment-emergent SAEs And AEs
Time Frame
Baseline through Month 30
Title
Change From Baseline To Day 28 In Transthyretin (TTR) Stabilization
Time Frame
Baseline, Day 28
Title
Change From Baseline To Month 30 In TTR Stabilization
Time Frame
Baseline, Month 30

10. Eligibility

Sex
All
Minimum Age & Unit of Time
20 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Nagoya University Hospital
City
Aichi
Country
Japan
Facility Name
Kurume University Hospital
City
Fukuoka
Country
Japan
Facility Name
Kyushu University Hospital
City
Fukuoka
Country
Japan
Facility Name
Sapporo Medical University Hospital
City
Hokkaido
Country
Japan
Facility Name
Kitasato University Hospital
City
Kanagawa
Country
Japan
Facility Name
Kochi Medical School Hospital
City
Kochi
Country
Japan
Facility Name
Kumamoto University Hospital
City
Kumamoto
Country
Japan
Facility Name
Shinshu University Hospital
City
Nagano
Country
Japan
Facility Name
Okayama University Hospital
City
Okayama
Country
Japan
Facility Name
National Cerebral and Cardiovascular Center
City
Osaka
Country
Japan
Facility Name
Juntendo University Hospital
City
Tokyo
Country
Japan
Facility Name
Keio University Hospital
City
Tokyo
Country
Japan
Facility Name
The University of Tokyo Hospital
City
Tokyo
Country
Japan

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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