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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04153149




Registration number
NCT04153149
Ethics application status
Date submitted
4/11/2019
Date registered
6/11/2019
Date last updated
13/11/2024

Titles & IDs
Public title
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Scientific 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)
Secondary ID [1] 0 0
2019-003153-28
Secondary ID [2] 0 0
ALN-TTRSC02-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vutrisiran
Treatment: Drugs - Sterile Normal Saline (0.9% NaCl)

Experimental: Vutrisiran 25 mg - Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind (DB) period. After the DB period, participants enter the open-label randomized treatment extension (RTE) period to receive vutrisiran 25 mg SC q3M or vutrisiran 50 mg SC every 6 months (q6M). After implementation of Amendment 4, participants from the DB period will enter the open-label treatment extension (OLE) period to receive vutrisiran 25 mg SC q3M and participants who were previously receiving vutrisiran 50 mg q6M in the RTE period, will be transitioned to vutrisiran 25 mg q3M in the OLE period.

Placebo comparator: Placebo - Participants will receive placebo during the double-blind period. After the DB period, participants enter the RTE period to receive vutrisiran 25 mg SC q3M or vutrisiran 50 mg SC q6M. After implementation of Amendment 4, participants from the DB period will enter the OLE period to receive vutrisiran 25 mg SC q3M and participants who were previously receiving vutrisiran 50 mg q6M in the RTE period, will be transitioned to vutrisiran 25 mg q3M in the OLE period.


Treatment: Drugs: Vutrisiran
Vutrisiran will be administered by SC injection.

Treatment: Drugs: Sterile Normal Saline (0.9% NaCl)
Sterile normal saline (0.9% NaCl) will be administered by SC injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population
Timepoint [1] 0 0
Up to Month 36
Primary outcome [2] 0 0
Composite Endpoint of All-Cause Mortality and Recurrent CV Events (CV Hospitalizations and Urgent HF Visits) in the Vutrisiran Monotherapy Subgroup
Timepoint [2] 0 0
Up to Month 36
Secondary outcome [1] 0 0
Change from Baseline in 6-Minute Walk Test (6-MWT) in the Overall Population and Vutrisiran Monotherapy Subgroup
Timepoint [1] 0 0
Baseline to Month 30
Secondary outcome [2] 0 0
Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) in the Overall Population and Vutrisiran Monotherapy Subgroup
Timepoint [2] 0 0
Baseline to Month 30
Secondary outcome [3] 0 0
All-cause Mortality in the Overall Population and Vutrisiran Monotherapy Subgroup
Timepoint [3] 0 0
Up to 42 months
Secondary outcome [4] 0 0
Change from Baseline in New York Heart Association (NYHA) Class in the Overall Population and Vutrisiran Monotherapy Subgroup
Timepoint [4] 0 0
Baseline to Month 30

Eligibility
Key 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
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/11/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2026
Actual
Sample size
Target
Accrual to date
Final
655
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Adelaide
Recruitment hospital [2] 0 0
Clinical Trial Site - Melbourne
Recruitment hospital [3] 0 0
Clinical Trial Site - Sydney
Recruitment hospital [4] 0 0
Clinical Trial Site - Westmead
Recruitment hospital [5] 0 0
Clinical Trial Site - Woolloongabba
Recruitment postcode(s) [1] 0 0
- Adelaide
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- Melbourne
Recruitment postcode(s) [3] 0 0
- Sydney
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment postcode(s) [5] 0 0
- Woolloongabba
Recruitment outside Australia
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California
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District of Columbia
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Middlesbrough

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alnylam Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT04153149
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Alnylam Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04153149