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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-516568-27 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vutrisiran 25 mg | Experimental | Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vutrisiran | Drug | Vutrisiran administered SC q3M |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | Up to 36 Months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Phoenix | Arizona | 85054 | United States | ||
| Clinical Trial Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39213194 | Result | 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. |
| Label | URL |
|---|---|
| ALN-TTR02-011 ClinicalTrials.gov posting | View source |
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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.
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| 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). | 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. | Baseline up to 36 Months |
| Change From Baseline in the Cardiac Biomarker: Troponin I | Troponin I will be assessed through measurement of serum levels. | 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 | Up to 36 Months |
| Change from baseline in serum TTR level | Baseline, Month 6 and Month 12 |
| Beverly Hills |
| California |
| 90211 |
| United States |
| Clinical Trial Site | La Jolla | California | 92037 | United States |
| Clinical Trial Site | Stanford | California | 94305 | United States |
| Clinical Trial Site | Washington D.C. | District of Columbia | 20010 | United States |
| Clinical Trial Site | Chicago | Illinois | 60637 | United States |
| Clinical Trial Site | Glenview | Illinois | 60026 | United States |
| Clinical Trial Site | Indianapolis | Indiana | 46202 | United States |
| Clinical Trial Site | Kansas City | Kansas | 66160 | United States |
| Clinical Trial Site | Baltimore | Maryland | 21287 | United States |
| Clinical Trial Site | Boston | Massachusetts | 02115 | United States |
| Clinical Trial Site | Boston | Massachusetts | 02118 | United States |
| Clinical Trial Site | Rochester | Minnesota | 55905 | United States |
| Clinical Trial Site | St Louis | Missouri | 63110 | United States |
| Clinical Trial Site | Omaha | Nebraska | 68198 | United States |
| Clinical Trial Site | New York | New York | 10029 | United States |
| Clinical Trial Site | New York | New York | 10032 | United States |
| Clinical Trial Site | Durham | North Carolina | 27710 | United States |
| Clinical Trial Site | Cleveland | Ohio | 44195 | United States |
| Clinical Trial Site | Columbus | Ohio | 43214 | United States |
| Clinical Trial Site | Philadelphia | Pennsylvania | 19104 | United States |
| Clinical Trial Site | Nashville | Tennessee | 37232 | United States |
| Clinical Trial Site | Dallas | Texas | 75246 | United States |
| Clinical Trial Site | Salt Lake City | Utah | 84132 | United States |
| Clinical Trial Site | Buenos Aires | C1093AAS | Argentina |
| Clinical Trial Site | Buenos Aires | C1199ABB | Argentina |
| Clinical Trial Site | Buenos Aires | C1428 | Argentina |
| Clinical Trial Site | Córdoba | X5000KEH | Argentina |
| Clinical Trial Site | Rosario | S2000DEJ | Argentina |
| Clinical Trial Site | Rosario | S2000DSR | Argentina |
| Clinical Trial Site | Rosario | S2000PBJ | Argentina |
| Clinical Trial Site | Vienna | 1090 | Austria |
| Clinical Trial Site | Vienna | 1210 | Austria |
| Clinical Trial Site | Aalst | 9300 | Belgium |
| Clinical Trial Site | Anderlecht | 1070 | Belgium |
| Clinical Trial Site | Dendermonde | 9200 | Belgium |
| Clinical Trial Site | Ghent | 9000 | Belgium |
| Clinical Trial Site | Hasselt | 3500 | Belgium |
| Clinical Trial Site | Leuven | 3000 | Belgium |
| Clinical Trial Site | Liège | 4000 | Belgium |
| Clinical Trial Site | Roeselare | 8800 | Belgium |
| Clinical Trial Site | Ribeirão Preto | 14026-900 | Brazil |
| Clinical Trial Site | Ribeirão Preto | 14051-140 | Brazil |
| Clinical Trial Site | Rio de Janeiro | 22280-020 | Brazil |
| Clinical Trial Site | São Paulo | 04012-180 | Brazil |
| Clinical Trial Site | São Paulo | 05403-000 | Brazil |
| Clinical Trial Site | Prague | 120 00 | Czechia |
| Clinical Trial Site | Prague | 128 21 | Czechia |
| Clinical Trial Site | Prague | 140 21 | Czechia |
| Clinical Trial Site | Aarhus N | 8200 | Denmark |
| Clinical Trial Site | København Ø | 2100 | Denmark |
| Clinical Trial Site | Odense C | 5000 | Denmark |
| Clinical Trial Site | Créteil | 94000 | France |
| Clinical Trial Site | Marseille | 13005 | France |
| Clinical Trial Site | Paris | 75018 | France |
| Clinical Trial Site | Rennes | 35000 | France |
| Clinical Trial Site | Toulouse | 31059 | France |
| Clinical Trial Site | Bologna | 40138 | Italy |
| Clinical Trial Site | Florence | 50134 | Italy |
| Clinical Trial Site | Pavia | 27100 | Italy |
| Clinical Trial Site | Groningen | 9713 GZ | Netherlands |
| Clinical Trial Site | Maastricht | 6229 HX | Netherlands |
| Clinical Trial Site | Utrecht | 3584 CX | Netherlands |
| Clinical Trial Site | Oslo | 372 | Norway |
| Clinical Trial Site | Creixomil | 4835-044 | Portugal |
| Clinical Trial Site | Lisbon | 1649-035 | Portugal |
| Clinical Trial Site | Porto | 4099-001 | Portugal |
| Clinical Trial Site | Viseu | 3504-509 | Portugal |
| Clinical Trial Site | Seoul | 03080 | South Korea |
| Clinical Trial Site | Seoul | 03722 | South Korea |
| Clinical Trial Site | Seoul | 06351 | South Korea |
| Clinical Trial Site | Barcelona | 08035 | Spain |
| Clinical Trial Site | Bilbao | 48013 | Spain |
| Clinical Trial Site | L'Hospitalet de Llobregat | 8907 | Spain |
| Clinical Trial Site | Madrid | 28222 | Spain |
| Clinical Trial Site | Málaga | 29010 | Spain |
| Clinical Trial Site | Gothenburg | 413 45 | Sweden |
| Clinical Trial Site | Solna | 171 76 | Sweden |
| Clinical Trial Site | Umeå | 907 37 | Sweden |
| Clinical Trial Site | Taipei | 112 | Taiwan |
| Clinical Trial Site | Bellshill | ML4 3NJ | United Kingdom |
| Clinical Trial Site | Birmingham | B15 2SQ | United Kingdom |
| Clinical Trial Site | Cardiff | CF15 9SS | United Kingdom |
| Clinical Trial Site | Hexham | NE46 1QJ | United Kingdom |
| Clinical Trial Site | London | NW3 2QG | United Kingdom |
| Clinical Trial Site | London | SE1 1YR | United Kingdom |
| Clinical Trial Site | Manchester | M15 6SE | United Kingdom |
| ALN-TTR02-014 ClinicalTrials.gov posting | View source |
| ALN-TTRSC02-003 ClinicalTrials.gov posting | View source |
| ID | Term |
|---|---|
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| D009202 | Cardiomyopathies |
| D000686 | Amyloidosis |
| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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