Trial Review

Did you know?

The ANZCTR now automatically displays published trial results and simplifies the addition of trial documents such as unpublished protocols and statistical analysis plans.

These enhancements will offer a more comprehensive view of trials, regardless of whether their results are positive, negative, or inconclusive.

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06183931




Registration number
NCT06183931
Ethics application status
Date submitted
15/11/2023
Date registered
28/12/2023
Date last updated
30/10/2024

Titles & IDs
Public title
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
Scientific 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)
Secondary ID [1] 0 0
D6810C00001
Universal Trial Number (UTN)
Trial acronym
DepleTTR-CM
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Transthyretin Amyloid Cardiomyopathy 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ALXN2220
Treatment: Drugs - Placebo

Experimental: ALXN2220 - Participants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 24 months up to a maximum of 48 months.

Placebo comparator: Placebo - Participants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.


Treatment: Drugs: ALXN2220
Participants will receive ALXN2220 via IV infusion.

Treatment: Drugs: Placebo
Participants will receive placebo via IV infusion.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
Timepoint [1] 0 0
Baseline up to the end study (up to Month 48)
Secondary outcome [1] 0 0
Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24
Timepoint [1] 0 0
Baseline, Month 24
Secondary outcome [2] 0 0
Time to CV-related Mortality
Timepoint [2] 0 0
Baseline up to the end of study (up to Month 48)
Secondary outcome [3] 0 0
Change from Baseline in Six-minute Walk Test (6MWT) at Month 24
Timepoint [3] 0 0
Baseline, Month 24
Secondary outcome [4] 0 0
Rate of CV Clinical Events
Timepoint [4] 0 0
Baseline up to the end of study (up to Month 48)
Secondary outcome [5] 0 0
Time to ACM
Timepoint [5] 0 0
Baseline up to the end of study (up to Month 48)

Eligibility
Key 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
Minimum age
18 Years
Maximum age
90 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/01/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/10/2028
Actual
Sample size
Target
1000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Bedford Park
Recruitment hospital [2] 0 0
Research Site - Box Hill
Recruitment hospital [3] 0 0
Research Site - Brisbane
Recruitment hospital [4] 0 0
Research Site - Darlinghurst
Recruitment hospital [5] 0 0
Research Site - Joondalup
Recruitment hospital [6] 0 0
Research Site - Melbourne
Recruitment hospital [7] 0 0
Research Site - Westmead
Recruitment postcode(s) [1] 0 0
5042 - Bedford Park
Recruitment postcode(s) [2] 0 0
3128 - Box Hill
Recruitment postcode(s) [3] 0 0
4102 - Brisbane
Recruitment postcode(s) [4] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [5] 0 0
6027 - Joondalup
Recruitment postcode(s) [6] 0 0
3004 - Melbourne
Recruitment postcode(s) [7] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Minnesota
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
North Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Ohio
Country [15] 0 0
United States of America
State/province [15] 0 0
Oregon
Country [16] 0 0
United States of America
State/province [16] 0 0
Pennsylvania
Country [17] 0 0
United States of America
State/province [17] 0 0
Rhode Island
Country [18] 0 0
United States of America
State/province [18] 0 0
South Carolina
Country [19] 0 0
United States of America
State/province [19] 0 0
Tennessee
Country [20] 0 0
United States of America
State/province [20] 0 0
Texas
Country [21] 0 0
United States of America
State/province [21] 0 0
Utah
Country [22] 0 0
United States of America
State/province [22] 0 0
Virginia
Country [23] 0 0
United States of America
State/province [23] 0 0
Washington
Country [24] 0 0
United States of America
State/province [24] 0 0
Wisconsin
Country [25] 0 0
Argentina
State/province [25] 0 0
Buenos Aires
Country [26] 0 0
Argentina
State/province [26] 0 0
Caba
Country [27] 0 0
Argentina
State/province [27] 0 0
Ciudad Autonoma Buenos Aires
Country [28] 0 0
Argentina
State/province [28] 0 0
Cordoba
Country [29] 0 0
Argentina
State/province [29] 0 0
Pilar
Country [30] 0 0
Austria
State/province [30] 0 0
Graz
Country [31] 0 0
Austria
State/province [31] 0 0
Innsbruck
Country [32] 0 0
Austria
State/province [32] 0 0
Wels
Country [33] 0 0
Austria
State/province [33] 0 0
Wien
Country [34] 0 0
Belgium
State/province [34] 0 0
Bruges
Country [35] 0 0
Belgium
State/province [35] 0 0
Brussels
Country [36] 0 0
Belgium
State/province [36] 0 0
Genk
Country [37] 0 0
Brazil
State/province [37] 0 0
Aracaju
Country [38] 0 0
Brazil
State/province [38] 0 0
Campinas
Country [39] 0 0
Brazil
State/province [39] 0 0
Fortaleza
Country [40] 0 0
Brazil
State/province [40] 0 0
Porto Alegre
Country [41] 0 0
Brazil
State/province [41] 0 0
Ribeirão Preto
Country [42] 0 0
Brazil
State/province [42] 0 0
Rio de Janeiro
Country [43] 0 0
Brazil
State/province [43] 0 0
Sao Paulo
Country [44] 0 0
Brazil
State/province [44] 0 0
São Paulo
Country [45] 0 0
Canada
State/province [45] 0 0
Alberta
Country [46] 0 0
Canada
State/province [46] 0 0
British Columbia
Country [47] 0 0
Canada
State/province [47] 0 0
Manitoba
Country [48] 0 0
Canada
State/province [48] 0 0
Ontario
Country [49] 0 0
Canada
State/province [49] 0 0
Quebec
Country [50] 0 0
China
State/province [50] 0 0
Beijing
Country [51] 0 0
China
State/province [51] 0 0
Changsha
Country [52] 0 0
China
State/province [52] 0 0
Chengdu
Country [53] 0 0
China
State/province [53] 0 0
Chongqing
Country [54] 0 0
China
State/province [54] 0 0
Dalian
Country [55] 0 0
China
State/province [55] 0 0
Fuzhou
Country [56] 0 0
China
State/province [56] 0 0
Guangzhou
Country [57] 0 0
China
State/province [57] 0 0
Guiyang
Country [58] 0 0
China
State/province [58] 0 0
Hangzhou
Country [59] 0 0
China
State/province [59] 0 0
Harbin
Country [60] 0 0
China
State/province [60] 0 0
Hefei
Country [61] 0 0
China
State/province [61] 0 0
Jinan
Country [62] 0 0
China
State/province [62] 0 0
Nanjing
Country [63] 0 0
China
State/province [63] 0 0
Shanghai
Country [64] 0 0
China
State/province [64] 0 0
Suzhou
Country [65] 0 0
China
State/province [65] 0 0
Tianjin
Country [66] 0 0
China
State/province [66] 0 0
Wuhan
Country [67] 0 0
China
State/province [67] 0 0
Xi'an
Country [68] 0 0
Czechia
State/province [68] 0 0
Brno
Country [69] 0 0
Czechia
State/province [69] 0 0
Praha 4
Country [70] 0 0
Czechia
State/province [70] 0 0
Praha
Country [71] 0 0
Denmark
State/province [71] 0 0
Aarhus
Country [72] 0 0
Denmark
State/province [72] 0 0
Copenhagen
Country [73] 0 0
Denmark
State/province [73] 0 0
Odense C
Country [74] 0 0
France
State/province [74] 0 0
Bron
Country [75] 0 0
France
State/province [75] 0 0
Chambray Les Tours
Country [76] 0 0
France
State/province [76] 0 0
Creteil
Country [77] 0 0
France
State/province [77] 0 0
Marseille
Country [78] 0 0
France
State/province [78] 0 0
Montpellier
Country [79] 0 0
France
State/province [79] 0 0
Paris
Country [80] 0 0
France
State/province [80] 0 0
Pessac
Country [81] 0 0
France
State/province [81] 0 0
Poitiers
Country [82] 0 0
France
State/province [82] 0 0
Rennes
Country [83] 0 0
France
State/province [83] 0 0
Saint-Herblain
Country [84] 0 0
France
State/province [84] 0 0
Toulouse
Country [85] 0 0
Germany
State/province [85] 0 0
Berlin
Country [86] 0 0
Germany
State/province [86] 0 0
Bonn
Country [87] 0 0
Germany
State/province [87] 0 0
Cologne
Country [88] 0 0
Germany
State/province [88] 0 0
Essen
Country [89] 0 0
Germany
State/province [89] 0 0
Gießen
Country [90] 0 0
Germany
State/province [90] 0 0
Greifwald
Country [91] 0 0
Germany
State/province [91] 0 0
Göttingen
Country [92] 0 0
Germany
State/province [92] 0 0
Hamburg
Country [93] 0 0
Germany
State/province [93] 0 0
Hannover
Country [94] 0 0
Germany
State/province [94] 0 0
Heidelberg
Country [95] 0 0
Germany
State/province [95] 0 0
Homburg
Country [96] 0 0
Germany
State/province [96] 0 0
Kiel
Country [97] 0 0
Germany
State/province [97] 0 0
Leipzig
Country [98] 0 0
Germany
State/province [98] 0 0
München
Country [99] 0 0
Germany
State/province [99] 0 0
Münster
Country [100] 0 0
Germany
State/province [100] 0 0
Würzburg
Country [101] 0 0
Greece
State/province [101] 0 0
Athens
Country [102] 0 0
Ireland
State/province [102] 0 0
Dublin
Country [103] 0 0
Israel
State/province [103] 0 0
Beer Yaakov
Country [104] 0 0
Israel
State/province [104] 0 0
Haifa
Country [105] 0 0
Israel
State/province [105] 0 0
Jerusalem
Country [106] 0 0
Israel
State/province [106] 0 0
Petah Tikva
Country [107] 0 0
Israel
State/province [107] 0 0
Rehovot
Country [108] 0 0
Italy
State/province [108] 0 0
Bologna
Country [109] 0 0
Italy
State/province [109] 0 0
Ferrara
Country [110] 0 0
Italy
State/province [110] 0 0
Florence
Country [111] 0 0
Italy
State/province [111] 0 0
Forli
Country [112] 0 0
Italy
State/province [112] 0 0
Messina
Country [113] 0 0
Italy
State/province [113] 0 0
Milano
Country [114] 0 0
Italy
State/province [114] 0 0
Napoli
Country [115] 0 0
Italy
State/province [115] 0 0
Padova
Country [116] 0 0
Italy
State/province [116] 0 0
Pavia
Country [117] 0 0
Italy
State/province [117] 0 0
Pisa
Country [118] 0 0
Italy
State/province [118] 0 0
Roma
Country [119] 0 0
Italy
State/province [119] 0 0
Torino
Country [120] 0 0
Italy
State/province [120] 0 0
Torrette
Country [121] 0 0
Italy
State/province [121] 0 0
Trieste
Country [122] 0 0
Japan
State/province [122] 0 0
Bunkyo-ku
Country [123] 0 0
Japan
State/province [123] 0 0
Kumamoto-shi
Country [124] 0 0
Japan
State/province [124] 0 0
Kurume-shi
Country [125] 0 0
Japan
State/province [125] 0 0
Kyoto-shi
Country [126] 0 0
Japan
State/province [126] 0 0
Matsumoto-shi
Country [127] 0 0
Japan
State/province [127] 0 0
Sapporo-shi
Country [128] 0 0
Japan
State/province [128] 0 0
Shinjuku-ku
Country [129] 0 0
Japan
State/province [129] 0 0
Suita-shi
Country [130] 0 0
Korea, Republic of
State/province [130] 0 0
Busan
Country [131] 0 0
Korea, Republic of
State/province [131] 0 0
Seoul
Country [132] 0 0
Korea, Republic of
State/province [132] 0 0
Wonju-si
Country [133] 0 0
Netherlands
State/province [133] 0 0
Groningen
Country [134] 0 0
Netherlands
State/province [134] 0 0
Maastricht
Country [135] 0 0
Netherlands
State/province [135] 0 0
Rotterdam
Country [136] 0 0
Netherlands
State/province [136] 0 0
Utrecht
Country [137] 0 0
New Zealand
State/province [137] 0 0
Auckland
Country [138] 0 0
Norway
State/province [138] 0 0
Lørenskog
Country [139] 0 0
Norway
State/province [139] 0 0
Oslo
Country [140] 0 0
Norway
State/province [140] 0 0
Trondheim
Country [141] 0 0
Poland
State/province [141] 0 0
Gdansk
Country [142] 0 0
Poland
State/province [142] 0 0
Krakow
Country [143] 0 0
Poland
State/province [143] 0 0
Poznan
Country [144] 0 0
Poland
State/province [144] 0 0
Warszawa
Country [145] 0 0
Spain
State/province [145] 0 0
Barcelona
Country [146] 0 0
Spain
State/province [146] 0 0
El Palmar
Country [147] 0 0
Spain
State/province [147] 0 0
Jaen
Country [148] 0 0
Spain
State/province [148] 0 0
L'Hospitalet de Llobregat
Country [149] 0 0
Spain
State/province [149] 0 0
La Coruna
Country [150] 0 0
Spain
State/province [150] 0 0
Madrid
Country [151] 0 0
Spain
State/province [151] 0 0
Majadahonda
Country [152] 0 0
Spain
State/province [152] 0 0
Malaga
Country [153] 0 0
Spain
State/province [153] 0 0
Palma de Mallorca
Country [154] 0 0
Spain
State/province [154] 0 0
Pamplona
Country [155] 0 0
Spain
State/province [155] 0 0
Salamanca
Country [156] 0 0
Spain
State/province [156] 0 0
Santiago De Compostela-Coruña
Country [157] 0 0
Spain
State/province [157] 0 0
Valencia
Country [158] 0 0
Sweden
State/province [158] 0 0
Göteborg
Country [159] 0 0
Sweden
State/province [159] 0 0
Lund
Country [160] 0 0
Sweden
State/province [160] 0 0
Skellefteå
Country [161] 0 0
Sweden
State/province [161] 0 0
Stockholm
Country [162] 0 0
Switzerland
State/province [162] 0 0
Basel
Country [163] 0 0
Switzerland
State/province [163] 0 0
Bern
Country [164] 0 0
Switzerland
State/province [164] 0 0
Luzern
Country [165] 0 0
Switzerland
State/province [165] 0 0
St.Gallen
Country [166] 0 0
Switzerland
State/province [166] 0 0
Zurich
Country [167] 0 0
Taiwan
State/province [167] 0 0
New Taipei City
Country [168] 0 0
Taiwan
State/province [168] 0 0
Taichung
Country [169] 0 0
Taiwan
State/province [169] 0 0
Taipei
Country [170] 0 0
Turkey
State/province [170] 0 0
Ankara
Country [171] 0 0
Turkey
State/province [171] 0 0
Dinar
Country [172] 0 0
Turkey
State/province [172] 0 0
Eskisehir
Country [173] 0 0
Turkey
State/province [173] 0 0
Istanbul
Country [174] 0 0
Turkey
State/province [174] 0 0
Izmir
Country [175] 0 0
Turkey
State/province [175] 0 0
Mersin
Country [176] 0 0
United Kingdom
State/province [176] 0 0
Birmingham
Country [177] 0 0
United Kingdom
State/province [177] 0 0
Cardiff
Country [178] 0 0
United Kingdom
State/province [178] 0 0
Glasgow
Country [179] 0 0
United Kingdom
State/province [179] 0 0
Hexham
Country [180] 0 0
United Kingdom
State/province [180] 0 0
London
Country [181] 0 0
United Kingdom
State/province [181] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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 ACM and CV clinical events.
Trial website
https://clinicaltrials.gov/study/NCT06183931
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alexion Pharmaceuticals, Inc. (Sponsor)
Address 0 0
Country 0 0
Phone 0 0
1-855-752-2356
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06183931