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Enrolling by Invitation

NCT Number: NCT06679946

A Study to Evaluate Vutrisiran in Patients With Transthyretin 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.

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Key information

Conditions

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy Amyloidosis Amyloidosis, Hereditary, Transthyretin-Related Cardiomyopathies Cardiovascular Diseases Heart Diseases Metabolic Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Buenos Aires, Argentina

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Vutrisiran

Drug

Vutrisiran administered SC q3M

Other names: ALN-TTRSC02, AMVUTTRA®

Primary outcomes

  1. Frequency of Adverse Events (AEs)

    Time frame: Up to 36 Months

    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.

Secondary outcomes

  1. Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)

    Time frame: Baseline up to 36 Months

    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.

  2. Change from Baseline in New York Heart Association (NYHA) Class

    Time frame: Baseline up to 36 Months

    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).

  3. Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP)

    Time frame: Baseline up to 36 Months

    NT-proBNP will be assessed through measurement of serum levels.

  4. Change From Baseline in the Cardiac Biomarker: Troponin I

    Time frame: Baseline up to 36 Months

    Troponin I will be assessed through measurement of serum levels.

  5. All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)

    Time frame: Up to 36 Months

    All deaths, hospitalizations and urgent HF visits will be recorded throughout the study as part of SAE and AE monitoring

  6. Change from baseline in serum TTR level

    Time frame: Baseline, Month 6 and Month 12

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study 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)

Important dates

Study start
2024
Primary completion
2028
Study completion
2028
First posted
Nov 8, 2024
Registry last updated
Feb 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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