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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)

HELIOS-A: A Phase 3 Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Alnylam Japan Co., Ltd.

Pacific Century Place Marunouchi 11th Floor, 1-11-1 Marunouchi, Chiyoda-ku, Tokyo

JPTiken@alnylam.com

CMIC Co., Ltd. (ICCC)

Hamamatsucho Bldg., 1-1-1 Shibaura Minato-ku, Tokyo

+81-3-6779-8000

TTR02-006_SC02-002@cmic.co.jp

completed

July. 31, 2019

Interventional

This is a global, Phase 3 randomized, open-label study designed to evaluate efficacy, safety, and pharmacokinetics (PK)/(pharmacodynamics (PD) of ALN-TTRSC02 in adult patients with hATTR amyloidosis. Patients will be randomized 3:1 to ALN TTRSC02 or patisiran, a reference comparator.

treatment purpose

3

-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)

-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

Both

Hereditary Transthyretin Amyloidosis with polyneuropathy

investigational material(s)
Generic name etc : ALN-TTRSC02
INN of investigational material : vutrisiran
Therapeutic category code : 129 Other agents affecting peripheral nervous system
Dosage and Administration for Investigational material : 25mg (0.5 mL) of ALN-TTRSC02 administered as a subcutaneous injection once every 3 months

control material(s)
Generic name etc : Patisiran Sodium (JAN)
INN of investigational material : Patisiran
Therapeutic category code : 129 Other agents affecting peripheral nervous system
Dosage and Administration for Investigational material : 0.3mg/kg of Patisiran is administered by intravenous drip infusion once every 3 weeks

efficacy
-Change from Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 [Time Frame: Baseline, Month 9]
-Change from Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score at Month 9 [Time Frame: Baseline, Month 9]

safety
efficacy
exploratory
pharmacokinetics
pharmacodynamics
-Change from Baseline in the Timed 10-Meter Walk Test (10-MWT) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18]
-Change from Baseline in the Modified Body Mass Index (mBMI) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ]
-Change from Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ]
-Percentage Reduction in Serum Transthyretin (TTR) Levels at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ]
-Frequency of All-Cause Deaths and/or All-Cause Hospitalizations Up to Month 18 [ Time Frame: Up to Month 18 ]
-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 ]

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April. 24, 2019

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April. 24, 2019