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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

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
Composite of Events of All-cause Mortality (ACM) and Total Cardiovascular (CV) Clinical Events
Timepoint [1] 0 0
Baseline up to the end study (up to Month 48)
Secondary outcome [1] 0 0
Composite of Events of ACM and Total Heart Failure (HF)
Timepoint [1] 0 0
Baseline up to the end of study (up to Month 48)
Secondary outcome [2] 0 0
Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24
Timepoint [2] 0 0
Baseline, Month 24
Secondary outcome [3] 0 0
Time to CV-related Mortality
Timepoint [3] 0 0
Baseline up to the end of study (up to Month 48)
Secondary outcome [4] 0 0
Change from Baseline in Six-minute Walk Test (6MWT) at Month 24
Timepoint [4] 0 0
Baseline, Month 24
Secondary outcome [5] 0 0
Number of Participants With CV Clinical Events
Timepoint [5] 0 0
Baseline up to the end of study (up to Month 48)
Secondary outcome [6] 0 0
Time to ACM
Timepoint [6] 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
30/11/2028
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
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06183931