Clinical trial NCT06679946: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Official title: An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Keywords: ATTR
Cardiomyopathy
Amylodosis
TTR
Transthyretin
TTR-mediated amyloidosis
Amyloidosis, Hereditary, Transthyretin-Related
RNAi therapeutic
Transthyretin amyloid cardiomyopathy
TTR cardiomyopathy
ATTR-CM
Wild-type TTR
V122I
TTR amyloidosis
Amyloidosis, Wild Type
hATTR amyloidosis with cardiomyopathy
ATTR amyloidosis with cardiomyopathy
wtATTR amyloidosis with cardiomyopathy
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2024-11-08
Enrollment: 800
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.


Eligibility criteria: Inclusion Criteria:

* Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003

Exclusion Criteria:

* Has permanently discontinued study drug administration while participating in the parent studies, ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003
* Future or current participation in another investigational device or drug study, scheduled to occur during this study
* Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation

Primary outcomes:
measure: Frequency of Adverse Events (AEs)
timeFrame: Up to 36 Months
Secondary outcomes:
measure: Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
timeFrame: Baseline up to 36 Months
measure: Change from Baseline in New York Heart Association (NYHA) Class
timeFrame: Baseline up to 36 Months
measure: Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP)
timeFrame: Baseline up to 36 Months
measure: Change From Baseline in the Cardiac Biomarker: Troponin I
timeFrame: Baseline up to 36 Months
measure: All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)
timeFrame: Up to 36 Months
measure: Change from baseline in serum TTR level
timeFrame: Baseline, Month 6 and Month 12
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Clinical Trial Site
state: Arizona
city: San Diego
country: United States
facility: Clinical Trial Site
state: California
city: Stanford
country: United States
facility: Clinical Trial Site
state: California
city: West Hollywood
country: United States
facility: Clinical Trial Site
state: California
city: Washington
country: United States
facility: Clinical Trial Site
state: District of Columbia
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Indianapolis
country: United States
facility: Clinical Trial Site
state: Indiana
city: Kansas City
country: United States
facility: Clinical Trial Site
state: Kansas
city: Baltimore
country: United States
facility: Clinical Trial Site
state: Maryland
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Rochester
country: United States
facility: Clinical Trial Site
state: Minnesota
city: Saint Louis
country: United States
facility: Clinical Trial Site
state: Missouri
city: Omaha
country: United States
facility: Clinical Trial Site
state: Nebraska
city: New York
country: United States
facility: Clinical Trial Site
state: New York
city: New York
country: United States
facility: Clinical Trial Site
state: New York
city: Durham
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Cleveland
country: United States
facility: Clinical Trial Site
state: Ohio
city: Columbus
country: United States
facility: Clinical Trial Site
state: Ohio
city: Philadelphia
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Nashville
country: United States
facility: Clinical Trial Site
state: Tennessee
city: Dallas
country: United States
facility: Clinical Trial Site
state: Texas
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Salt Lake City
country: United States
facility: Clinical Trial Site
state: Utah
city: Buenos Aires
country: Argentina
facility: Clinical Trial Site
city: Córdoba
country: Argentina
facility: Clinical Trial Site
city: Rosario
country: Argentina
facility: Clinical Trial Site
city: Vienna
country: Austria
facility: Clinical Trial Site
city: Aalst
country: Belgium
facility: Clinical Trial Site
city: Anderlecht
country: Belgium
facility: Clinical Trial Site
city: Dendermonde
country: Belgium
facility: Clinical Trial Site
city: Ghent
country: Belgium
facility: Clinical Trial Site
city: Hasselt
country: Belgium
facility: Clinical Trial Site
city: Leuven
country: Belgium
facility: Clinical Trial Site
city: Liège
country: Belgium
facility: Clinical Trial Site
city: Roeselare
country: Belgium
facility: Clinical Trial Site
city: Ribeirão Preto
country: Brazil
facility: Clinical Trial Site
city: Rio de Janeiro
country: Brazil
facility: Clinical Trial Site
city: São Paulo
country: Brazil
facility: Clinical Trial Site
city: Prague
country: Czechia
facility: Clinical Trial Site
city: Aarhus
country: Denmark
facility: Clinical Trial Site
city: Copenhagen
country: Denmark
facility: Clinical Trial Site
city: Odense
country: Denmark
facility: Clinical Trial Site
city: Créteil
country: France
facility: Clinical Trial Site
city: Marseille
country: France
facility: Clinical Trial Site
city: Paris
country: France
facility: Clinical Trial Site
city: Rennes
country: France
facility: Clinical Trial Site
city: Toulouse
country: France
facility: Clinical Trial Site
city: Bologna
country: Italy
facility: Clinical Trial Site
city: Florence
country: Italy
facility: Clinical Trial Site
city: Pavia
country: Italy
facility: Clinical Trial Site
city: Groningen
country: Netherlands
facility: Clinical Trial Site
city: Maastricht
country: Netherlands
facility: Clinical Trial Site
city: Utrecht
country: Netherlands
facility: Clinical Trial Site
city: Oslo
country: Norway
facility: Clinical Trial Site
city: Creixomil
country: Portugal
facility: Clinical Trial Site
city: Lisbon
country: Portugal
facility: Clinical Trial Site
city: Porto
country: Portugal
facility: Clinical Trial Site
city: Viseu
country: Portugal
facility: Clinical Trial Site
city: Barcelona
country: Spain
facility: Clinical Trial Site
city: Bilbao
country: Spain
facility: Clinical Trial Site
city: Llobregat
country: Spain
facility: Clinical Trial Site
city: Majadahonda
country: Spain
facility: Clinical Trial Site
city: Málaga
country: Spain
facility: Clinical Trial Site
city: Gothenburg
country: Sweden
facility: Clinical Trial Site
city: Solna
country: Sweden
facility: Clinical Trial Site
city: Umeå
country: Sweden
facility: Clinical Trial Site
city: Taipei
country: Taiwan
facility: Clinical Trial Site
city: Bellshill
country: United Kingdom
facility: Clinical Trial Site
state: Scotland
city: Birmingham
country: United Kingdom
facility: Clinical Trial Site
city: Cardiff
country: United Kingdom
facility: Clinical Trial Site
city: Hexham
country: United Kingdom
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
city: Manchester
country: United Kingdom
facility: Clinical Trial Site
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: Fontana M, Berk JL, Gillmore JD, Witteles RM, Grogan M, Drachman B, Damy T, Garcia-Pavia P, Taubel J, Solomon SD, Sheikh FH, Tahara N, Gonzalez-Costello J, Tsujita K, Morbach C, Pozsonyi Z, Petrie MC, Delgado D, Van der Meer P, Jabbour A, Bondue A, Kim D, Azevedo O, Hvitfeldt Poulsen S, Yilmaz A, Jankowska EA, Algalarrondo V, Slugg A, Garg PP, Boyle KL, Yureneva E, Silliman N, Yang L, Chen J, Eraly SA, Vest J, Maurer MS; HELIOS-B Trial Investigators. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. 2025 Jan 2;392(1):33-44. doi: 10.1056/NEJMoa2409134. Epub 2024 Aug 30.