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Trial record 1 of 1 for:    NCT04153149
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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 : January 18, 2020
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE November 4, 2019
First Posted Date  ICMJE November 6, 2019
Last Update Posted Date January 18, 2020
Actual Study Start Date  ICMJE November 26, 2019
Estimated Primary Completion Date June 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 4, 2019)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT04153149 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 4, 2019)
  • Change from Baseline in 6-Minute Walk Test (6-MWT) at Month 30 [ Time Frame: Baseline, Month 30 ]
  • 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.
  • Change from Baseline in Mean Left Ventricular (LV) Wall Thickness by Echocardiographic Assessment at Month 30 [ Time Frame: Baseline, Month 30 ]
  • Change from Baseline in Global Longitudinal Strain by Echocardiographic Assessment at Month 30 [ Time Frame: Baseline and Month 30 ]
  • 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.
  • All-cause Mortality [ Time Frame: 30-36 months ]
  • Frequency of Recurrent CV Hospitalizations [ Time Frame: 30-36 months ]
  • Change from Baseline in N-terminal prohormone B-type Natriuretic Peptide (NTproBNP) at Month 30 [ Time Frame: Baseline, Month 30 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Official Title  ICMJE 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)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Intervention  ICMJE
  • Drug: Vutrisiran
    Vutrisiran 25 mg will be administered by SC injection q3M.
    Other Name: ALN-TTRSC02
  • Drug: Sterile Normal Saline (0.9% NaCl)
    Sterile normal saline (0.9% NaCl) will be administered by SC injection q3M.
Study Arms  ICMJE
  • Experimental: Vutrisiran 25 mg
    Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period.
    Intervention: Drug: Vutrisiran
  • Placebo Comparator: Placebo
    Participants will receive placebo during the double-blind period.
    Intervention: Drug: Sterile Normal Saline (0.9% NaCl)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 4, 2019)
600
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2025
Estimated Primary Completion Date June 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04153149
Other Study ID Numbers  ICMJE ALN-TTRSC02-003
2019-003153-28 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: John Vest, MD Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP