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Trial details
Trial details
Helios-B : A Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
Current status:
Approved
|
Date registered:
28/01/2021
Trial version(s)
History: 10/07/2020
Current: 10/07/2020
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2020104517
Protocol number
ALN-TTRSC02-003
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
29/05/2020
Primary sponsor
Alnylam Pharmaceuticals, Inc.
Primary sponsor: Country of origin
USA
Public title
Helios-B : A Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
Acronym
HELIOS-B
Scientific title
Helios-B: A Phase 3, Randomized, Doubleblind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
Acronym
HELIOS-B
Brief summary of the study: English
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.
Brief summary of the study: Arabic
ستقوم هذه الدراسة بتقييم فعالية وسلامة فوتريزبران 25 مغم يعطى في شكل حقنة تحت الجلد مرة كل 3 اشهر مقارنة مع الدواء الوهمي في المرضى الذبن يعانون من الداء النشواني مع اعتلال عضلة القلب.
Health conditions/problem studied: Specify
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions: Specify
Arm: Experimental: Vutrisiran 25 mg Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period. Assigned Intervention: 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. Assigned Intervention: Drug: Sterile Normal Saline (0.9% NaCl) Sterile normal saline (0.9% NaCl) will be administered by SC injection q3M.
Key inclusion and exclusion criteria: Inclusion criteria
Inclusion criteria: 1. 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. 2. Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF OR clinical evidence of HF.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
18
Key inclusion and exclusion criteria: Age maximum
85
Key inclusion and exclusion criteria: Exclusion criteria
Exclusion criteria: 1. Has known primary amyloidosis or leptomeningeal amyloidosis 2. Has New York Heart Association (NYHA) Class IV heart failure 3. Has NYHA Class III heart failure AND is at high risk based on pre-specified criteria 4. Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV at the Screening visit 5. Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m^2 6. Has received prior TTR-lowering treatment 7. Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
Randomized controlled trial
Study design: Masking
Blinded (masking used)
Study design: Control
Placebo
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Parallel
IMP has market authorization
No
Name of IMP
Vutrisiran (ALN-TTRSC02)
Type of IMP
Gene therapy
Pharmaceutical class
Synthetic RNA interference (RNAi) therapeutic molecule
Therapeutic indication
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Therapeutic benefit
Vutrisiran utilizes RNAi to prevent the synthesis of both wt and mutant TTR in the liver, the primary source of circulating TTR. TTR reduction with vutrisiran will beneficially impact disease progression in patients with ATTR amyloidosis with cardiomyopathy.
Biospecimen retention
Samples with DNA**
Biospecimen description
Biological specimens will be collected, may include DNA, RNA, or biochemical metabolite assessments as they relate to disease progression, efficacy or safety. The biospecimen repository will also include residual material from routine samples (safety laboratory samples, PK samples, etc.) that are obtained during the study.
Target sample size
600
Actual enrollment target size
Date of first enrollment: Type
Actual
Date of first enrollment: Date
26/11/2019
Date of study closure: Type
Actual
Date of study closure: Date
30/06/2025
Recruitment status
Recruiting
Date of completion
30/06/2024
IPD sharing statement plan
No
IPD sharing statement description
Not Applicable
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Food and Drug Administration
NCT04153149
Eudract Number
2019-003153-28
Sources of Monetary or Material Support
Name
Alnylam Pharmaceuticals, Inc
Secondary Sponsors
Name
NA
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Aziz
El Zoghbi
MCT-CRO
Lebanon
+961 71 008 269
zog_az@mctcro. com
Director of Country Oversight and Manageme nt Africa, Levant and GCC
Scientific
Alnylam Clinical Trial Information Line
Central Contact Person
Not applicable
United States of America
1-877- ALNYLAM
clinicaltrials@aln ylam.com
Alnylam Pharmaceuticals, Inc
Scientific
Alnylam Clinical Trial Information Line
Central Contact Backup
Not applicable
United States of America
1-877-256- 9526
Not Applicable
Alnylam Pharmace uticals, Inc
Scientific
Jean
El Cheikh
American University of Beirut Medical Center
Lebanon
+961 71 407 447
je46@aub.edu.lb
Principal Investigato r
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
American University of Beirut Medical Center
Jean El Cheikh
Hematology/Oncology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
American University of Beirut Medical Center
12/10/2020
Jean El Cheikh
je46@aub.edu.lb
+96171407447
Countries of Recruitment
Name
Lebanon
France
United States of America
Argentina
Australia
Austria
Belgium
Brazil
Canada
Colombia
Bulgaria
Denmark
Germany
Ireland
Italy
Czech Republic
Mexico
Norway
Japan
Peru
Republic of Moldova
Poland
Netherlands
Portugal
Spain
Sweden
United Kingdom
Malaysia
Taiwan
Romania
Croatia
Estonia
Finland
Costa Rica
Greece
Hungary
Latvia
Lithuania
Saudi Arabia
Slovakia
Slovenia
Jordan
Health Conditions or Problems Studied
Condition
Code
Keyword
Transthyretin Amyloidosis with Cardiomyopathy
Heart failure (I50)
Cardiomyopathy
Interventions
Intervention
Description
Keyword
Experimental: Vutrisiran 25 mg
Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period
Vutrisiran, SC, q3M
Placebo Comparator: Placebo
Participants will receive placebo during the double-blind period.
Placebo
Primary Outcomes
Name
Time points
Measure
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.
Key Secondary Outcomes
Name
Time points
Measure
Change from Baseline in 6-Minute Walk Test (6-MWT) at Month 30
[ Time Frame: Baseline, Month 30 ]
Change from baseline in 6-minute walk test (6-MWT)
Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) at Month 30
[ Time Frame: Baseline, Month 30 ]
Change from baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
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 Mean Left Ventricular (LV) Wall Thickness by Echocardiographic Assessment
Change from Baseline in Global Longitudinal Strain by Echocardiographic Assessment at month 30
[ Time Frame: Baseline, Month 30 ]
Change from Baseline in Global Longitudinal Strain by Echocardiographic Assessment
Composite Endpoint of All-Cause Mortality and Recurrent Allcause Hospitalizations and Urgent HF Visits
[ Time Frame: 30-36 months ]
Composite Endpoint of All-Cause Mortality and Recurrent All-cause Hospitalizations and Urgent HF Visits using an Andersen-Gill model.
All-cause Mortality
[ Time Frame: 30-36 months ]
All-cause mortality
Rate of Recurrent CV Events (CV Hospitalizations and Urgent HF Visits)
[ Time Frame: 30-36 months ]
Recurrent CV hospitalizations
Change from Baseline in N-terminal prohormone B-type Natriuretic Peptide (NTproBNP) at month 30
[ Time Frame: Baseline, Month 30 ]
Change from Baseline in N-terminal prohormone Btype Natriuretic Peptide (NTproBNP)
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial