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
|Condition or disease||Intervention/treatment||Phase|
|Transthyretin Amyloidosis (ATTR) With Cardiomyopathy||Drug: Vutrisiran Drug: Sterile Normal Saline (0.9% NaCl)||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||600 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
Experimental: Vutrisiran 25 mg
Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period.
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.
- 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.
- 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 ]
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
|Contact: Alnylam Clinical Trial Information Line||1-877-ALNYLAMemail@example.com|
|Contact: Alnylam Clinical Trial Information Line||1-877-256-9526|
|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|
|Study Director:||John Vest, MD||Alnylam Pharmaceuticals|