Transthyretin Amyloidosis (ATTR) With CardiomyopathyPhase 3NCT06679946
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
A Phase 3 study testing Vutrisiran in people with Transthyretin Amyloidosis (ATTR) With Cardiomyopathy, run by Alnylam Pharmaceuticals at 87 sites in 17 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
- What is being tested?
- Vutrisiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 700 participants
- Where
- 87 sitesin 17 countries
- Study dates
- 3 Dec 2024to 31 Mar 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
87 sites in 17 countries. Contact the study team or a site near you to ask about taking part.
87 sites in 17 countries
United States24 sites
Clinical Trial Site
Baltimore, Maryland, 21287
Clinical Trial Site
Beverly Hills, California, 90211
Clinical Trial Site
Boston, Massachusetts, 02115
Clinical Trial Site
Boston, Massachusetts, 02118
Clinical Trial Site
Chicago, Illinois, 60637
Clinical Trial Site
Cleveland, Ohio, 44195
Clinical Trial Site
Columbus, Ohio, 43214
Clinical Trial Site
Dallas, Texas, 75246
Clinical Trial Site
Durham, North Carolina, 27710
Clinical Trial Site
Glenview, Illinois, 60026
Clinical Trial Site
Indianapolis, Indiana, 46202
Clinical Trial Site
Kansas City, Kansas, 66160
Clinical Trial Site
La Jolla, California, 92037
Clinical Trial Site
Nashville, Tennessee, 37232
Clinical Trial Site
New York, New York, 10029
Clinical Trial Site
New York, New York, 10032
Clinical Trial Site
Omaha, Nebraska, 68198
Clinical Trial Site
Philadelphia, Pennsylvania, 19104
Clinical Trial Site
Phoenix, Arizona, 85054
Clinical Trial Site
Rochester, Minnesota, 55905
Clinical Trial Site
Salt Lake City, Utah, 84132
Clinical Trial Site
St Louis, Missouri, 63110
Clinical Trial Site
Stanford, California, 94305
Clinical Trial Site
Washington D.C., District of Columbia, 20010
Belgium8 sites
Clinical Trial Site
Aalst, 9300
Clinical Trial Site
Anderlecht, 1070
Clinical Trial Site
Dendermonde, 9200
Clinical Trial Site
Ghent, 9000
Clinical Trial Site
Hasselt, 3500
Clinical Trial Site
Leuven, 3000
Clinical Trial Site
Liège, 4000
Clinical Trial Site
Roeselare, 8800
Argentina7 sites
Clinical Trial Site
Buenos Aires, C1093AAS
Clinical Trial Site
Buenos Aires, C1199ABB
Clinical Trial Site
Buenos Aires, C1428
Clinical Trial Site
Córdoba, X5000KEH
Clinical Trial Site
Rosario, S2000DEJ
Clinical Trial Site
Rosario, S2000DSR
Clinical Trial Site
Rosario, S2000PBJ
United Kingdom7 sites
Clinical Trial Site
Bellshill, ML4 3NJ
Clinical Trial Site
Birmingham, B15 2SQ
Clinical Trial Site
Cardiff, CF15 9SS
Clinical Trial Site
Hexham, NE46 1QJ
Clinical Trial Site
London, NW3 2QG
Clinical Trial Site
London, SE1 1YR
Clinical Trial Site
Manchester, M15 6SE
Brazil5 sites
Clinical Trial Site
Ribeirão Preto, 14026-900
Clinical Trial Site
Ribeirão Preto, 14051-140
Clinical Trial Site
Rio de Janeiro, 22280-020
Clinical Trial Site
São Paulo, 04012-180
Clinical Trial Site
São Paulo, 05403-000
France5 sites
Clinical Trial Site
Créteil, 94000
Clinical Trial Site
Marseille, 13005
Clinical Trial Site
Paris, 75018
Clinical Trial Site
Rennes, 35000
Clinical Trial Site
Toulouse, 31059
Spain5 sites
Clinical Trial Site
Barcelona, 08035
Clinical Trial Site
Bilbao, 48013
Clinical Trial Site
L'Hospitalet de Llobregat, 8907
Clinical Trial Site
Madrid, 28222
Clinical Trial Site
Málaga, 29010
Portugal4 sites
Clinical Trial Site
Creixomil, 4835-044
Clinical Trial Site
Lisbon, 1649-035
Clinical Trial Site
Porto, 4099-001
Clinical Trial Site
Viseu, 3504-509
Czechia3 sites
Clinical Trial Site
Prague, 120 00
Clinical Trial Site
Prague, 128 21
Clinical Trial Site
Prague, 140 21
Denmark3 sites
Clinical Trial Site
Aarhus N, 8200
Clinical Trial Site
København Ø, 2100
Clinical Trial Site
Odense C, 5000
Italy3 sites
Clinical Trial Site
Bologna, 40138
Clinical Trial Site
Florence, 50134
Clinical Trial Site
Pavia, 27100
Netherlands3 sites
Clinical Trial Site
Groningen, 9713 GZ
Clinical Trial Site
Maastricht, 6229 HX
Clinical Trial Site
Utrecht, 3584 CX
South Korea3 sites
Clinical Trial Site
Seoul, 03080
Clinical Trial Site
Seoul, 03722
Clinical Trial Site
Seoul, 06351
Sweden3 sites
Clinical Trial Site
Gothenburg, 413 45
Clinical Trial Site
Solna, 171 76
Clinical Trial Site
Umeå, 907 37
Austria2 sites
Clinical Trial Site
Vienna, 1090
Clinical Trial Site
Vienna, 1210
About this study
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)
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.
- Being tested
- Vutrisiran
- 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
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Cardiomyopathies, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VutrisiranDrug
Also called ALN-TTRSC02, AMVUTTRA®
Vutrisiran administered SC q3M
| Study group | Role | What they receive |
|---|---|---|
| Vutrisiran 25 mg | Experimental | Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M). Receives: Drug: Vutrisiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events (AEs)
An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
Measured: Up to 36 Months
Secondary outcomes (6)
Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary [OS]). Scores are transformed to a range of 0-100, in which higher scores reflect better health status.
Measured: Baseline up to 36 Months
Change from Baseline in New York Heart Association (NYHA) Class
NYHA classification grades the severity of heart failure symptoms into the following stages: I (no symptoms; ordinary physical activity such as walking and climbing stairs does not cause fatigue or dyspnea), II (symptoms with ordinary physical activity; walking or climbing stairs rapidly, walking uphill, walking or stair climbing after meals, in cold weather, in wind or when under emotional stress causes undue fatigue or dyspnea), III (symptoms with less than ordinary physical activity; walking 1 to 2 blocks on the level and climbing more than 1 flight of stairs in normal conditions causes undue fatigue or dyspnea), IV (symptoms at rest; inability to carry on any physical activity without fatigue or dyspnea).
Measured: Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP)
NT-proBNP will be assessed through measurement of serum levels.
Measured: Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: Troponin I
Troponin I will be assessed through measurement of serum levels.
Measured: Baseline up to 36 Months
All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)
All deaths, hospitalizations and urgent HF visits will be recorded throughout the study as part of SAE and AE monitoring
Measured: Up to 36 Months
Change from baseline in serum TTR level
Measured: Baseline, Month 6 and Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2024
- Study start3 Dec 2024
- Primary completion31 Mar 2028 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transthyretin Amyloidosis (ATTR) With CardiomyopathyStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 700 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06679946
- Sponsor's protocol ID
- ALN-TTRSC02-007
- Other IDs
- 2024-516568-27
- Status last verified
- Oct 2026
- First submitted
- 6 Nov 2024
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Access to data may be declined where there is likelihood a patient could be identified or other feasibility issue, where there is a potential conflict of interest, a planned business activities or an actual or potential competitive risk. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Timeframes for data access may vary and can take up to 6 months or more. Requests for access to data can be submitted via the website www.vivli.org. Questions can also be directed to datasharing@alnylam.com.
Publications
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.
PubMed 39213194