TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN)

Conditions

Hereditary transthyretin-mediated amyloidosis with polyneuropathy

Brief summary

Change from baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) compared to the external placebo group from the APOLLO study at Month 9

Detailed description

1. Change from baseline in Norfolk Quality of Life Diabetic Neuropathy (Norfolk QoL-DN) total score compared to the external placebo group from the APOLLO study at Month 9, 2. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 9, 3. Change from baseline in modified body mass index (mBMI) compared to the external placebo group from the APOLLO study at Month 9, 4. Change from baseline in the following parameters compared to the external placebo group from the APOLLO study at Month 18: o mNIS+7; o Norfolk QoL-DN total score; o mBMI; o Rasch-built Overall Disability Scale R-ODS; o Timed 10-meter walk test (10-MWT), 5. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 18, 6. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group at Week 6

Related papers

No related papers found yet.

Interventions

DRUGAmvuttra 25 mg solution for injection in pre-filled syringe

DRUGALN-TTRSC04

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Change from baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) compared to the external placebo group from the APOLLO study at Month 9	—

Secondary

Measure	Time frame
1. Change from baseline in Norfolk Quality of Life Diabetic Neuropathy (Norfolk QoL-DN) total score compared to the external placebo group from the APOLLO study at Month 9, 2. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 9, 3. Change from baseline in modified body mass index (mBMI) compared to the external placebo group from the APOLLO study at Month 9, 4. Change from baseline in the following parameters compared to the external placebo group from the APOLLO study at Month 18: o mNIS+7; o Norfolk QoL-DN total score; o mBMI; o Rasch-built Overall Disability Scale R-ODS; o Timed 10-meter walk test (10-MWT), 5. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 18, 6. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group at Week 6	—

Measure

Time frame

1. Change from baseline in Norfolk Quality of Life Diabetic Neuropathy (Norfolk QoL-DN) total score compared to the external placebo group from the APOLLO study at Month 9, 2. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 9, 3. Change from baseline in modified body mass index (mBMI) compared to the external placebo group from the APOLLO study at Month 9, 4. Change from baseline in the following parameters compared to the external placebo group from the APOLLO study at Month 18: o mNIS+7; o Norfolk QoL-DN total score; o mBMI; o Rasch-built Overall Disability Scale R-ODS; o Timed 10-meter walk test (10-MWT), 5. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 18, 6. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group at Week 6

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Outcome results

None listed