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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03759379




Registration number
NCT03759379
Ethics application status
Date submitted
28/11/2018
Date registered
30/11/2018

Titles & IDs
Public title
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Scientific title
HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Secondary ID [1] 0 0
2018-002098-23
Secondary ID [2] 0 0
ALN-TTRSC02-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyloidosis, Hereditary 0 0
Transthyretin Amyloidosis 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 - Patisiran
Treatment: Drugs - Vutrisiran

Experimental: Vutrisiran + Vutrisiran (HELIOS-A) - Participants will receive vutrisiran 25 mg subcutaneous (SC) injection once every 3 months (q3M) for 18 months during the Treatment Period followed by vutrisiran 50 mg SC injection once every 6 months (q6M) or vutrisiran 25 mg q3M during the Randomized Treatment Extension (RTE) Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing.

Active comparator: Patisiran + Vutrisiran (HELIOS-A) - Participants will receive patisiran 0.3 mg/kg intravenous (IV) infusion once every 3 weeks (q3w) for 18 months during the Treatment Period followed by vutrisiran 50 mg SC injection once q6M or vutrisiran 25 mg q3M during the RTE Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing.


Treatment: Drugs: Patisiran
Patisiran will be administered by IV infusion.

Treatment: Drugs: Vutrisiran
Vutrisiran will be administered by SC injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [1] 0 0
Baseline, Month 9
Secondary outcome [1] 0 0
Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [1] 0 0
Baseline, Month 9
Secondary outcome [2] 0 0
Change From Baseline in the Timed 10-Meter Walk Test (10-MWT) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [2] 0 0
Baseline, Month 9
Secondary outcome [3] 0 0
Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 18 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [3] 0 0
Baseline, Month 18
Secondary outcome [4] 0 0
Change From Baseline in Norfolk QoL-DN Total Score at Month 18 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [4] 0 0
Baseline, Month 18
Secondary outcome [5] 0 0
Change From Baseline in the 10-MWT at Month 18 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [5] 0 0
Baseline, Month 18
Secondary outcome [6] 0 0
Change From Baseline in the Modified Body Mass Index (mBMI) at Month 18 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [6] 0 0
Baseline, Month 18
Secondary outcome [7] 0 0
Change From Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Month 18 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]
Timepoint [7] 0 0
Baseline, Month 18
Secondary outcome [8] 0 0
Percent Reduction in Serum Transthyretin (TTR) Levels Through Month 18 Between the Vutrisiran Group (HELIOS-A) and the Patisiran Group (HELIOS-A)
Timepoint [8] 0 0
Up to Month 18

Eligibility
Key inclusion criteria
* Male or female of 18 to 85 years of age (inclusive);
* Has a diagnosis of hATTR amyloidosis with transthyretin (TTR) mutation;
* Has adequate neurologic impairment score (NIS);
* Has adequate polyneuropathy disability (PND) score;
* Has adequate Karnofsky Performance Status (KPS).
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Had a prior liver transplant or is likely to undergo liver transplantation during the study;
* Has known other (non-hATTR) forms of amyloidosis or leptomeningeal amyloidosis;
* Has New York Heart Association heart failure classification >2;
* Clinically significant liver function test abnormalities;
* Has known human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) infection;
* Received an experimental drug within 30 days of dosing;
* Received prior TTR-lowering treatment;
* Has other known causes of neuropathy.

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
Open (masking not used)
Who is / are masked / blinded?



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
14/02/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2026
Actual
Sample size
Target
Accrual to date
Final
164
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Box Hill
Recruitment hospital [2] 0 0
Clinical Trial Site - Westmead
Recruitment hospital [3] 0 0
Clinical Trial Site - Woolloongabba
Recruitment postcode(s) [1] 0 0
- Box Hill
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment postcode(s) [3] 0 0
- Woolloongabba
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oregon
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
Argentina
State/province [14] 0 0
Buenos Aires
Country [15] 0 0
Belgium
State/province [15] 0 0
Bruxelles
Country [16] 0 0
Belgium
State/province [16] 0 0
Leuven
Country [17] 0 0
Brazil
State/province [17] 0 0
Rio De Janeiro
Country [18] 0 0
Bulgaria
State/province [18] 0 0
Sofia
Country [19] 0 0
Canada
State/province [19] 0 0
Montréal
Country [20] 0 0
Canada
State/province [20] 0 0
Vancouver
Country [21] 0 0
Cyprus
State/province [21] 0 0
Nicosia
Country [22] 0 0
France
State/province [22] 0 0
Bordeaux
Country [23] 0 0
France
State/province [23] 0 0
Créteil
Country [24] 0 0
France
State/province [24] 0 0
Marseille
Country [25] 0 0
France
State/province [25] 0 0
Paris
Country [26] 0 0
Germany
State/province [26] 0 0
Mainz
Country [27] 0 0
Germany
State/province [27] 0 0
Münster
Country [28] 0 0
Greece
State/province [28] 0 0
Athens
Country [29] 0 0
Italy
State/province [29] 0 0
Messina
Country [30] 0 0
Italy
State/province [30] 0 0
Pavia
Country [31] 0 0
Italy
State/province [31] 0 0
Rome
Country [32] 0 0
Japan
State/province [32] 0 0
Kumamoto
Country [33] 0 0
Japan
State/province [33] 0 0
Nagano
Country [34] 0 0
Japan
State/province [34] 0 0
Nagoya
Country [35] 0 0
Japan
State/province [35] 0 0
Osaka
Country [36] 0 0
Korea, Republic of
State/province [36] 0 0
Junggu
Country [37] 0 0
Malaysia
State/province [37] 0 0
Kuala Lumpur
Country [38] 0 0
Mexico
State/province [38] 0 0
Mexico City
Country [39] 0 0
Netherlands
State/province [39] 0 0
Groningen
Country [40] 0 0
Portugal
State/province [40] 0 0
Lisboa
Country [41] 0 0
Portugal
State/province [41] 0 0
Porto
Country [42] 0 0
Spain
State/province [42] 0 0
Barcelona
Country [43] 0 0
Spain
State/province [43] 0 0
Huelva
Country [44] 0 0
Spain
State/province [44] 0 0
Madrid
Country [45] 0 0
Spain
State/province [45] 0 0
Valencia
Country [46] 0 0
Sweden
State/province [46] 0 0
Solna
Country [47] 0 0
Sweden
State/province [47] 0 0
Umeå
Country [48] 0 0
Taiwan
State/province [48] 0 0
Taipei City
Country [49] 0 0
Taiwan
State/province [49] 0 0
Taipei
Country [50] 0 0
United Kingdom
State/province [50] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the United States (US) and/or the European Union (EU).

Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03759379