Clinical trial NCT06183931: Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Transthyretin Amyloid Cardiomyopathy
Keywords: Amyloid Depleter
ALXN2220
All-cause mortality (ACM)
Cardiovascular (CV)
Transthyretin Amyloid Cardiomyopathy
Transthyretin amyloid (ATTR)
Transthyretin (TTR)
N-terminal pro b-type natriuretic peptide (NT-proBNP)
NT-proBNP
Amyloidosis
ATTR-CM
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-12-28
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of ATTR-CM with either wild-type or variant TTR genotype
* End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
* NT-proBNP \> 2000 pg/mL at Screening
* Treatment with a loop diuretic for at least 30 days prior to Screening
* History of heart failure NYHA Class II-IV at Screening
* Life expectancy of ≥ 6 months as per the Investigator's judgment
* Males and females of childbearing ability must use contraception

Exclusion Criteria:

* Known leptomeningeal amyloidosis
* Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
* Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
* Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment
* LVEF \< 30% on echocardiography
* Renal failure requiring dialysis or an eGFR \< 20 mL/min/1.73 m2 at Screening
* Polyneuropathy with PND score IV

Primary outcomes:
measure: Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
timeFrame: Baseline up to the end study (up to Month 48)
Secondary outcomes:
measure: Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24
timeFrame: Baseline, Month 24
measure: Time to CV-related Mortality
timeFrame: Baseline up to the end of study (up to Month 48)
measure: Change from Baseline in Six-minute Walk Test (6MWT) at Month 24
timeFrame: Baseline, Month 24
measure: Rate of CV Clinical Events
timeFrame: Baseline up to the end of study (up to Month 48)
measure: Time to ACM
timeFrame: Baseline up to the end of study (up to Month 48)
Other outcomes:
Locations:
city: Irvine
country: United States
facility: Research Site
state: California
city: La Jolla
country: United States
facility: Research Site
state: California
city: Palo Alto
country: United States
facility: Research Site
state: California
city: San Francisco
country: United States
facility: Research Site
state: California
city: Aurora
country: United States
facility: Research Site
state: Colorado
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Jacksonville
country: United States
facility: Research Site
state: Florida
city: Weston
country: United States
facility: Research Site
state: Florida
city: Atlanta
country: United States
facility: Research Site
state: Georgia
city: Atlanta
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facility: Research Site
state: Georgia
city: Chicago
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facility: Research Site
state: Illinois
city: Indianapolis
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city: Boston
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facility: Research Site
state: Massachusetts
city: Boston
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city: Kansas City
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state: Missouri
city: Saint Louis
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state: Missouri
city: New York
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state: New York
city: New York
country: United States
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state: New York
city: Stony Brook
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facility: Research Site
state: New York
city: Chapel Hill
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state: North Carolina
city: Durham
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city: Columbus
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city: Portland
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city: Danville
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state: Pennsylvania
city: Philadelphia
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city: Greenville
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city: Fort Worth
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city: Houston
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city: Falls Church
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country: Austria
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city: Wels
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country: Austria
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city: Wien
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city: Bruges
country: Belgium
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city: Brussels
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city: Genk
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city: Aracaju
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city: Fortaleza
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city: Ribeirão Preto
country: Brazil
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city: Ribeirão Preto
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city: Rio de Janeiro
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city: Sao Paulo
country: Brazil
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city: Sao Paulo
country: Brazil
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city: São Paulo
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city: Calgary
country: Canada
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state: Alberta
city: Winnipeg
country: Canada
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state: Manitoba
city: London
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city: Toronto
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city: Chicoutimi
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city: Montréal
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city: Montréal
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city: Rimouski
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city: Beijing
country: China
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country: China
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country: China
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country: China
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city: Changsha
country: China
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city: Chengdu
country: China
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city: Chengdu
country: China
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city: Chongqing
country: China
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city: Fuzhou
country: China
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city: Guangzhou
country: China
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city: Guiyang
country: China
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city: Hangzhou
country: China
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city: Hangzhou
country: China
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city: Harbin
country: China
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city: Hefei
country: China
facility: Research Site
city: Jinan
country: China
facility: Research Site
city: Nanjing
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Tianjin
country: China
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city: Wuhan
country: China
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city: Xi'an
country: China
facility: Research Site
city: Praha 4
country: Czechia
facility: Research Site
city: Praha
country: Czechia
facility: Research Site
city: Aarhus
country: Denmark
facility: Research Site
city: Odense C
country: Denmark
facility: Research Site
city: Bron
country: France
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city: Chambray Les Tours
country: France
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city: Creteil
country: France
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city: Marseille
country: France
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city: Montpellier
country: France
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city: Paris
country: France
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city: Paris
country: France
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city: Pessac
country: France
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city: Poitiers
country: France
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country: France
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country: France
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city: Toulouse
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city: Berlin
country: Germany
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country: Germany
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country: Germany
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city: Hamburg
country: Germany
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city: Hannover
country: Germany
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city: Heidelberg
country: Germany
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city: Homburg
country: Germany
facility: Research Site
city: Kiel
country: Germany
facility: Research Site
city: Leipzig
country: Germany
facility: Research Site
city: München
country: Germany
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city: Münster
country: Germany
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city: Würzburg
country: Germany
facility: Research Site
city: Athens
country: Greece
facility: Research Site
city: Dublin
country: Ireland
facility: Research Site
city: Beer Yaakov
country: Israel
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city: Haifa
country: Israel
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city: Haifa
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city: Jerusalem
country: Israel
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city: Jerusalem
country: Israel
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city: Petah Tikva
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city: Rehovot
country: Israel
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city: Bologna
country: Italy
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city: Ferrara
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city: Florence
country: Italy
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city: Forli
country: Italy
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city: Messina
country: Italy
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city: Milano
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Padova
country: Italy
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city: Pavia
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city: Roma
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city: Torino
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city: Trieste
country: Italy
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city: Bunkyo-ku
country: Japan
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city: Kumamoto-shi
country: Japan
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city: Kurume-shi
country: Japan
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city: Kyoto-shi
country: Japan
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city: Matsumoto-shi
country: Japan
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city: Sapporo-shi
country: Japan
facility: Research Site
city: Shinjuku-ku
country: Japan
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city: Suita-shi
country: Japan
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city: Busan
country: Korea, Republic of
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city: Seoul
country: Korea, Republic of
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city: Seoul
country: Korea, Republic of
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city: Seoul
country: Korea, Republic of
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city: Seoul
country: Korea, Republic of
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city: Seoul
country: Korea, Republic of
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city: Wonju-si
country: Korea, Republic of
facility: Research Site
city: Groningen
country: Netherlands
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city: Maastricht
country: Netherlands
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city: Rotterdam
country: Netherlands
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city: Utrecht
country: Netherlands
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city: Lørenskog
country: Norway
facility: Research Site
city: Oslo
country: Norway
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city: Trondheim
country: Norway
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country: Poland
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country: Poland
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country: Poland
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country: Spain
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city: Barcelona
country: Spain
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city: El Palmar
country: Spain
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city: Jaen
country: Spain
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city: L'Hospitalet de Llobregat
country: Spain
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city: Madrid
country: Spain
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city: Majadahonda
country: Spain
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city: Malaga
country: Spain
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city: Palma de Mallorca
country: Spain
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city: Pamplona
country: Spain
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city: Salamanca
country: Spain
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city: Santiago De Compostela-Coruña
country: Spain
facility: Research Site
city: Valencia
country: Spain
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city: Göteborg
country: Sweden
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city: Lund
country: Sweden
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city: Skellefteå
country: Sweden
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city: Stockholm
country: Sweden
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city: Basel
country: Switzerland
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city: Bern
country: Switzerland
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city: Luzern
country: Switzerland
facility: Research Site
city: St.Gallen
country: Switzerland
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city: Zurich
country: Switzerland
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city: New Taipei City
country: Taiwan
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city: Taichung
country: Taiwan
facility: Research Site
city: Taichung
country: Taiwan
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
city: Ankara
country: Turkey
facility: Research Site
city: Dinar
country: Turkey
facility: Research Site
city: Eskisehir
country: Turkey
facility: Research Site
city: Istanbul
country: Turkey
facility: Research Site
city: Mersin
country: Turkey
facility: Research Site
city: Birmingham
country: United Kingdom
facility: Research Site
city: Cardiff
country: United Kingdom
facility: Research Site
city: Glasgow
country: United Kingdom
facility: Research Site
city: Hexham
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: Manchester
country: United Kingdom
facility: Research Site
Study officials:
References: