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HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

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ClinicalTrials.gov Identifier: NCT04153149
Recruitment Status : Active, not recruiting
First Posted : November 6, 2019
Last Update Posted : November 11, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

Condition or disease Intervention/treatment Phase
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy Drug: Vutrisiran Drug: Sterile Normal Saline (0.9% NaCl) Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 655 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Actual Study Start Date : November 26, 2019
Estimated Primary Completion Date : June 2024
Estimated Study Completion Date : June 2025


Arm Intervention/treatment
Experimental: Vutrisiran 25 mg
Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period.
Drug: Vutrisiran
Vutrisiran 25 mg will be administered by SC injection q3M.
Other Name: ALN-TTRSC02

Placebo Comparator: Placebo
Participants will receive placebo during the double-blind period.
Drug: Sterile Normal Saline (0.9% NaCl)
Sterile normal saline (0.9% NaCl) will be administered by SC injection q3M.




Primary Outcome Measures :
  1. Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) [ Time Frame: 30-36 months ]
    All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) will be compared between treatment groups using an Andersen-Gill model.


Secondary Outcome Measures :
  1. Change from Baseline in 6-Minute Walk Test (6-MWT) at Month 30 [ Time Frame: Baseline, Month 30 ]
  2. Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) at Month 30 [ Time Frame: Baseline, Month 30 ]
    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.

  3. Change from Baseline in Mean Left Ventricular (LV) Wall Thickness by Echocardiographic Assessment at Month 30 [ Time Frame: Baseline, Month 30 ]
  4. Change from Baseline in Global Longitudinal Strain by Echocardiographic Assessment at Month 30 [ Time Frame: Baseline and Month 30 ]
  5. Composite Endpoint of All-Cause Mortality and Recurrent All-cause Hospitalizations and Urgent HF Visits [ Time Frame: 30-36 months ]
    All-cause mortality and recurrent all-cause hospitalizations and urgent HF visits will be compared between treatment groups using an Andersen-Gill model.

  6. All-cause Mortality [ Time Frame: 30-36 months ]
  7. Rate of Recurrent CV Events (CV Hospitalizations and Urgent HF Visits) [ Time Frame: 30-36 months ]
  8. Change from Baseline in N-terminal prohormone B-type Natriuretic Peptide (NTproBNP) at Month 30 [ Time Frame: Baseline, Month 30 ]


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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Has a documented diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy, classified as either hereditary ATTR (hATTR) amyloidosis with cardiomyopathy or wild-type ATTR (wtATTR) amyloidosis with cardiomyopathy meeting pre-specified diagnostic criteria
  • Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF OR clinical evidence of HF

Exclusion Criteria:

  • Has known primary amyloidosis or leptomeningeal amyloidosis
  • Has New York Heart Association (NYHA) Class IV heart failure
  • Has NYHA Class III heart failure AND is at high risk based on pre-specified criteria
  • Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV at the Screening visit
  • Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m^2
  • Has received prior TTR-lowering treatment
  • Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04153149


Locations
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Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04153149    
Other Study ID Numbers: ALN-TTRSC02-003
2019-003153-28 ( EudraCT Number )
First Posted: November 6, 2019    Key Record Dates
Last Update Posted: November 11, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alnylam Pharmaceuticals:
Amyloidosis
TTR-mediated amyloidosis
Amyloidosis, Hereditary
Amyloidosis, Hereditary, Transthyretin-Related
Familial Amyloidosis
Transthyretin amyloid cardiomyopathy
TTR amyloidosis
Amyloidosis, Wild Type
ATTR
Cardiomyopathy
TTR
Transthyretin
RNAi therapeutic
TTR cardiomyopathy
ATTR-CM
Wild-type TTR
V122I
Additional relevant MeSH terms:
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Amyloid Neuropathies, Familial
Amyloidosis
Amyloid Neuropathies
Amyloidosis, Familial
Cardiomyopathies
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors