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Trial record 1 of 1 for:    NCT03759379
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HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

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ClinicalTrials.gov Identifier: NCT03759379
Recruitment Status : Recruiting
First Posted : November 30, 2018
Last Update Posted : April 16, 2019
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran or the reference comparator patisiran during the Treatment Period. The Treatment Period is followed by a Treatment Extension Period during which all participants in the patisiran group will switch to vutrisiran. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the co-primary and most other efficacy endpoints.

Condition or disease Intervention/treatment Phase
Amyloidosis, Hereditary Transthyretin Amyloidosis Drug: Patisiran Drug: Vutrisiran (ALN-TTRSC02) Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : January 18, 2019
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : May 2024


Arm Intervention/treatment
Experimental: Vutrisiran (ALN-TTRSC02)
Participants will receive vutrisiran during the Treatment and Treatment Extension Periods.
Drug: Vutrisiran (ALN-TTRSC02)
Vutrisiran will be administered by subcutaneous (SC) injection.

Active Comparator: Patisiran
Participants will receive patisiran during the Treatment Period and will switch to vutrisiran during the Treatment Extension Period.
Drug: Patisiran
Patisiran will be administered by intravenous (IV) infusion.

Drug: Vutrisiran (ALN-TTRSC02)
Vutrisiran will be administered by subcutaneous (SC) injection.




Primary Outcome Measures :
  1. Change from Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 [ Time Frame: Baseline, Month 9 ]
    The mNIS+7 is a composite score that quantifies motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.

  2. Change from Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score at Month 9 [ Time Frame: Baseline, Month 9 ]
    The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.


Secondary Outcome Measures :
  1. Change from Baseline in the Timed 10-Meter Walk Test (10-MWT) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ]
  2. Change from Baseline in the Modified Body Mass Index (mBMI) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ]
  3. Change from Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ]
    The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.

  4. Percentage Reduction in Serum Transthyretin (TTR) Levels at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ]
  5. Frequency of All-Cause Deaths and/or All-Cause Hospitalizations Up to Month 18 [ Time Frame: Up to Month 18 ]
  6. Frequency of All-Cause Deaths and/or All-Cause Hospitalizations in Participants with Cardiac Involvement Up to Month 18 [ Time Frame: Up to Month 18 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).

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.

Information from the National Library of Medicine

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): NCT03759379


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526

Locations
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United States, California
Clinical Trial Site Recruiting
San Diego, California, United States, 92120
United States, Colorado
Clinical Trial Site Recruiting
Aurora, Colorado, United States, 80045
United States, Maryland
Clinical Trial Site Recruiting
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Clinical Trial Site Recruiting
Boston, Massachusetts, United States, 02118
United States, Missouri
Clinical Trial Site Recruiting
Saint Louis, Missouri, United States, 63130
United States, Ohio
Clinical Trial Site Recruiting
Columbus, Ohio, United States, 43210
United States, Pennsylvania
Clinical Trial Site Recruiting
Philadelphia, Pennsylvania, United States, 19140
United States, Texas
Clinical Trial Site Recruiting
Fort Worth, Texas, United States, 75246
Bulgaria
Clinical Trial Site Recruiting
Sofia, Bulgaria
France
Clinical Trial Site Recruiting
Le Kremlin-Bicêtre, France
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: John Vest, MD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03759379     History of Changes
Other Study ID Numbers: ALN-TTRSC02-002
2018-002098-23 ( EudraCT Number )
First Posted: November 30, 2018    Key Record Dates
Last Update Posted: April 16, 2019
Last Verified: April 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Amyloidosis
Amyloid Neuropathies, Familial
Amyloidosis, Familial
Proteostasis Deficiencies
Metabolic Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Amyloid Neuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors