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Trial record 9 of 11 for:    AG10

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04622046
Recruitment Status : Active, not recruiting
First Posted : November 9, 2020
Last Update Posted : April 10, 2024
Sponsor:
Collaborator:
Eidos Therapeutics, a BridgeBio company
Information provided by (Responsible Party):
Alexion Pharmaceuticals, Inc.

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.

Condition or disease Intervention/treatment Phase
Symptomatic Transthyretin Amyloid Cardiomyopathy Drug: ALXN2060 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : November 13, 2020
Actual Primary Completion Date : November 8, 2023
Estimated Study Completion Date : September 23, 2025

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: ALXN2060
Participants will receive ALXN2060.
Drug: ALXN2060
ALXN2060 tablets will be administered twice daily at a dose of 800 milligrams.
Other Names:
  • AG10
  • Acoramidis




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

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


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04622046


Locations
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Japan
Research Site
Bunkyo-ku, Japan, 113-8431
Research Site
Fukuoka-shi, Japan, 812-8582
Research Site
Kumamoto-shi, Japan, 860-8556
Research Site
Kurume-shi, Japan, 830-0011
Research Site
Matsumoto-shi, Japan, 390-8621
Research Site
Nagoya-shi, Japan, 466-8560
Research Site
Nankoku-shi, Japan, 783-8505
Research Site
Sagamihara-shi, Japan, 252-0375
Research Site
Sapporo-shi, Japan, 060-8543
Research Site
Shinjuku-ku, Japan, 160-8582
Research Site
Suita-shi, Japan, 564-8565
Sponsors and Collaborators
Alexion Pharmaceuticals, Inc.
Eidos Therapeutics, a BridgeBio company
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Responsible Party: Alexion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04622046    
Other Study ID Numbers: ALXN2060-TAC-302
First Posted: November 9, 2020    Key Record Dates
Last Update Posted: April 10, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cardiomyopathies
Amyloidosis
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases