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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 : Recruiting
First Posted : November 6, 2019
Last Update Posted : November 28, 2019
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)
Estimated Enrollment : 600 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)
Estimated Study Start Date : November 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 Frequency of Recurrent Cardiovascular (CV) Hospitalizations [ Time Frame: 30-36 months ]
    All-cause mortality and frequency of CV hospitalizations 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 [ Time Frame: 30-36 months ]
    All-cause mortality and frequency of recurrent all-cause hospitalizations will be compared between treatment groups using an Andersen-Gill model.

  6. All-cause Mortality [ Time Frame: 30-36 months ]
  7. Frequency of Recurrent CV Hospitalizations [ 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


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526

Locations
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United States, California
Clinical Trial Site Recruiting
La Mesa, California, United States, 91942
United States, District of Columbia
Clinical Trial Site Recruiting
Washington, District of Columbia, United States, 20010
United States, Massachusetts
Clinical Trial Site Not yet recruiting
Boston, Massachusetts, United States, 02118
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: John Vest, MD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04153149     History of Changes
Other Study ID Numbers: ALN-TTRSC02-003
2019-003153-28 ( EudraCT Number )
First Posted: November 6, 2019    Key Record Dates
Last Update Posted: November 28, 2019
Last Verified: November 2019
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:
ATTR
Cardiomyopathy
Amyloidosis
TTR
Transthyretin
TTR-mediated amyloidosis
Amyloidosis, Hereditary
Amyloidosis, Hereditary, Transthyretin-Related
Familial Amyloidosis
RNAi therapeutic
Transthyretin amyloid cardiomyopathy
TTR cardiomyopathy
ATTR-CM
Wild-type TTR
V122I
TTR amyloidosis
Amyloidosis, Wild Type
Additional relevant MeSH terms:
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Amyloid Neuropathies, Familial
Cardiomyopathies
Amyloidosis
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Amyloid Neuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors