Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

Transthyretin Amyloid Cardiomyopathy Clinical Trial

— DepleTTR-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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06183931
• Other study ID #: D6810C00001
• Status: Recruiting
• Phase: Phase 3
• Start date: January 11, 2024
• Est. completion date: October 31, 2028

Study information

• Verified date: June 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: Alexion Pharmaceuticals, Inc. (Sponsor)
• Phone: 1-855-752-2356
• Email: clinicaltrials[@]alexion.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The primary objective of this study is to assess 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 composite endpoint of all-cause mortality (ACM) and total cardiovascular (CV) clinical events.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1000
• Est. completion date: October 31, 2028
• Est. primary completion date: July 31, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 90 Years

Eligibility

Inclusion Criteria:

• Centrally confirmed diagnosis of ATTR-CM with either wild-type or variant TTR genotype

• End-diastolic interventricular septal wall thickness = 11 mm for women or = 12 mm for men 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

Related Conditions & MeSH terms

• Amyloidosis
• Cardiomyopathies
• Transthyretin Amyloid Cardiomyopathy

Intervention

Drug:
• ALXN2220
Participants will receive ALXN2220 via IV infusion.
• Placebo
Participants will receive placebo via IV infusion.

Locations

Country	Name	City	State
Argentina	Research Site	Buenos Aires
Argentina	Research Site	Buenos Aires
Argentina	Research Site	Caba
Argentina	Research Site	Caba
Argentina	Research Site	Ciudad Autonoma Buenos Aires
Argentina	Research Site	Cordoba
Argentina	Research Site	Pilar
Australia	Research Site	Bedford Park
Australia	Research Site	Box Hill
Australia	Research Site	Brisbane
Australia	Research Site	Darlinghurst
Australia	Research Site	Joondalup
Australia	Research Site	Melbourne
Australia	Research Site	Westmead
Austria	Research Site	Graz
Austria	Research Site	Innsbruck
Austria	Research Site	Wels
Austria	Research Site	Wien
Austria	Research Site	Wien
Belgium	Research Site	Bruges
Belgium	Research Site	Brussels
Belgium	Research Site	Genk
Brazil	Research Site	Campinas
Brazil	Research Site	Fortaleza
Brazil	Research Site	Porto Alegre
Brazil	Research Site	Ribeirão Preto
Brazil	Research Site	Ribeirão Preto
Brazil	Research Site	Rio de Janeiro
Brazil	Research Site	Rio de Janeiro
Brazil	Research Site	Sao Paulo
Brazil	Research Site	Sao Paulo
Brazil	Research Site	São Paulo
Brazil	Research Site	São Paulo
Brazil	Research Site	São Paulo
Canada	Research Site	Calgary	Alberta
Canada	Research Site	Chicoutimi	Quebec
Canada	Research Site	London	Ontario
Canada	Research Site	Montréal	Quebec
Canada	Research Site	Montréal	Quebec
Canada	Research Site	Rimouski	Quebec
Canada	Research Site	Toronto	Ontario
Canada	Research Site	Vancouver	British Columbia
Canada	Research Site	Winnipeg	Manitoba
China	Research Site	Beijing
China	Research Site	Beijing
China	Research Site	Beijing
China	Research Site	Beijing
China	Research Site	Beijing
China	Research Site	Changsha
China	Research Site	Chengdu
China	Research Site	Chengdu
China	Research Site	Chongqing
China	Research Site	Dalian
China	Research Site	Fuzhou
China	Research Site	Guangzhou
China	Research Site	Guiyang
China	Research Site	Hangzhou
China	Research Site	Hangzhou
China	Research Site	Hangzhou
China	Research Site	Harbin
China	Research Site	Hefei
China	Research Site	Jinan
China	Research Site	Nanjing
China	Research Site	Shanghai
China	Research Site	Suzhou
China	Research Site	Tianjin
China	Research Site	Wuhan
China	Research Site	Wuhan
China	Research Site	Xi'an
Czechia	Research Site	Brno
Czechia	Research Site	Praha
Czechia	Research Site	Praha 4
Denmark	Research Site	Aarhus
Denmark	Research Site	Copenhagen
Denmark	Research Site	Odense C
France	Research Site	Bron
France	Research Site	Chambray Les Tours
France	Research Site	Créteil Cédex
France	Research Site	Marseille
France	Research Site	Montpellier
France	Research Site	Paris
France	Research Site	Paris Cedex 13
France	Research Site	Pessac
France	Research Site	Poitiers
France	Research Site	Rennes Cedex 9
France	Research Site	Saint-Herblain
France	Research Site	Toulouse
Germany	Research Site	Bonn
Germany	Research Site	Cologne
Germany	Research Site	Essen
Germany	Research Site	Gießen
Germany	Research Site	Göttingen
Germany	Research Site	Greifwald
Germany	Research Site	Hamburg
Germany	Research Site	Hannover
Germany	Research Site	Heidelberg
Germany	Research Site	Homburg
Germany	Research Site	Kiel
Germany	Research Site	Leipzig
Germany	Research Site	München
Germany	Research Site	Münster
Germany	Research Site	Würzburg
Germany	Research Site	Würzburg
Greece	Research Site	Athens
Greece	Research Site	Athens
Ireland	Research Site	Dublin
Ireland	Research Site	Dublin
Israel	Research Site	Beer Yaakov
Israel	Research Site	Haifa
Israel	Research Site	Haifa
Israel	Research Site	Jerusalem
Israel	Research Site	Jerusalem
Israel	Research Site	Petah Tikva
Israel	Research Site	Rehovot
Italy	Research Site	Bologna
Italy	Research Site	Ferrara
Italy	Research Site	Florence
Italy	Research Site	Forli
Italy	Research Site	Messina
Italy	Research Site	Milano
Italy	Research Site	Napoli
Italy	Research Site	Padova
Italy	Research Site	Pavia
Italy	Research Site	Pisa
Italy	Research Site	Roma
Italy	Research Site	Torino
Italy	Research Site	Trieste
Japan	Research Site	Bunkyo-ku
Japan	Research Site	Kumamoto-shi
Japan	Research Site	Kurume-shi
Japan	Research Site	Kyoto-shi
Japan	Research Site	Matsumoto-shi
Japan	Research Site	Sapporo-shi
Japan	Research Site	Shinjuku-ku
Japan	Research Site	Suita-shi
Korea, Republic of	Research Site	Busan
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Wonju-si
Netherlands	Research Site	Groningen
Netherlands	Research Site	Maastricht
Netherlands	Research Site	Rotterdam
Netherlands	Research Site	Utrecht
New Zealand	Research Site	Auckland
Norway	Research Site	Lørenskog
Norway	Research Site	Oslo
Norway	Research Site	Trondheim
Poland	Research Site	Gdansk
Poland	Research Site	Krakow
Poland	Research Site	Poznan
Poland	Research Site	Warszawa
Poland	Research Site	Warszawa
Spain	Research Site	Barcelona
Spain	Research Site	El Palmar
Spain	Research Site	Jaen
Spain	Research Site	L'Hospitalet de Llobregat
Spain	Research Site	La Coruna
Spain	Research Site	Madrid
Spain	Research Site	Majadahonda
Spain	Research Site	Malaga
Spain	Research Site	Palma de Mallorca
Spain	Research Site	Pamplona
Spain	Research Site	Salamanca
Spain	Research Site	Santiago De Compostela-Coruña
Spain	Research Site	Valencia
Sweden	Research Site	Göteborg
Sweden	Research Site	Lund
Sweden	Research Site	Skellefteå
Sweden	Research Site	Stockholm
Switzerland	Research Site	Basel
Switzerland	Research Site	Bern
Switzerland	Research Site	Luzern
Switzerland	Research Site	St.Gallen
Switzerland	Research Site	Zurich
Taiwan	Research Site	New Taipei City
Taiwan	Research Site	Taichung
Taiwan	Research Site	Taichung
Taiwan	Research Site	Taipei
Taiwan	Research Site	Taipei
Turkey	Research Site	Ankara
Turkey	Research Site	Dinar
Turkey	Research Site	Eskisehir
Turkey	Research Site	Istanbul
Turkey	Research Site	Istanbul
Turkey	Research Site	Izmir
Turkey	Research Site	Mersin
United Kingdom	Research Site	Birmingham
United Kingdom	Research Site	Cardiff
United Kingdom	Research Site	Glasgow
United Kingdom	Research Site	Hexham
United Kingdom	Research Site	London
United Kingdom	Research Site	London
United Kingdom	Research Site	London
United Kingdom	Research Site	Manchester
United States	Research Site	Atlanta	Georgia
United States	Research Site	Atlanta	Georgia
United States	Research Site	Aurora	Colorado
United States	Research Site	Boston	Massachusetts
United States	Research Site	Boston	Massachusetts
United States	Research Site	Chapel Hill	North Carolina
United States	Research Site	Charleston	South Carolina
United States	Research Site	Chicago	Illinois
United States	Research Site	Chicago	Illinois
United States	Research Site	Cleveland	Ohio
United States	Research Site	Columbus	Ohio
United States	Research Site	Danville	Pennsylvania
United States	Research Site	Durham	North Carolina
United States	Research Site	Falls Church	Virginia
United States	Research Site	Fort Worth	Texas
United States	Research Site	Greenville	South Carolina
United States	Research Site	Houston	Texas
United States	Research Site	Indianapolis	Indiana
United States	Research Site	Irvine	California
United States	Research Site	Jacksonville	Florida
United States	Research Site	Kansas City	Kansas
United States	Research Site	Kansas City	Missouri
United States	Research Site	La Jolla	California
United States	Research Site	Madison	Wisconsin
United States	Research Site	Minneapolis	Minnesota
United States	Research Site	Minneapolis	Minnesota
United States	Research Site	Nashville	Tennessee
United States	Research Site	New York	New York
United States	Research Site	New York	New York
United States	Research Site	Palo Alto	California
United States	Research Site	Philadelphia	Pennsylvania
United States	Research Site	Portland	Oregon
United States	Research Site	Providence	Rhode Island
United States	Research Site	Richmond	Virginia
United States	Research Site	Rochester	Minnesota
United States	Research Site	Saint Louis	Missouri
United States	Research Site	Salt Lake City	Utah
United States	Research Site	San Francisco	California
United States	Research Site	Seattle	Washington
United States	Research Site	Stony Brook	New York
United States	Research Site	Washington	District of Columbia
United States	Research Site	Weston	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Austria,  Belgium,  Brazil,  Canada,  China,  Czechia,  Denmark,  France,  Germany,  Greece,  Ireland,  Israel,  Italy,  Japan,  Korea, Republic of,  Netherlands,  New Zealand,  Norway,  Poland,  Spain,  Sweden,  Switzerland,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Composite of Events of All-cause Mortality (ACM) and Total Cardiovascular (CV) Clinical Events		Baseline up to the end study (up to Month 48)
Secondary	Composite of Events of ACM and Total Heart Failure (HF)		Baseline up to the end of study (up to Month 48)
Secondary	Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24		Baseline, Month 24
Secondary	Time to CV-related Mortality		Baseline up to the end of study (up to Month 48)
Secondary	Change from Baseline in Six-minute Walk Test (6MWT) at Month 24		Baseline, Month 24
Secondary	Number of Participants With CV Clinical Events		Baseline up to the end of study (up to Month 48)
Secondary	Time to ACM		Baseline up to the end of study (up to Month 48)