Trial reportEuropean journal of neurology2026
Effect of Eplontersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy Across Genetic Variants: An Exploratory Analysis From the NEURO-TTRansform Trial.
Trial report in European journal of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (NEURO-TTR Study)
A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
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12 authors.
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Abstract
backgroundThis exploratory analysis of the NEURO-TTRansform Phase 3 trial evaluated the efficacy of eplontersen in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy by genetic variant.
methodsChanges from baseline in NEURO-TTRansform primary endpoints serum transthyretin (TTR) at Week 65, modified Neuropathy Impairment Score+7 (mNIS+7) composite score, and Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score at Week 66 were evaluated in patients with early-onset (aged < 50 years) and late-onset (aged ≥ 50 years) Val30Met (p.Val50Met) or non-Val30Met ATTRv amyloidosis with polyneuropathy. Secondary endpoints from NEURO-TTRansform were also evaluated by genetic variant.
resultsIn total, 144 patients with early-onset (n = 54) or late-onset (n = 31) Val30Met or non-Val30Met (n = 59), ATTRv amyloidosis with polyneuropathy were randomized to eplontersen. A further 60 patients from NEURO-TTR with early-onset (n = 16) or late-onset (n = 17) Val30Met or non-Val30Met (n = 27), served as a historical placebo group. The mean percentage difference (95% confidence interval) in serum TTR was -79.9 (-87.8, -72.0), -85.0 (-93.3, -76.6), and -70.6 (-77.7, -63.5) with eplontersen versus placebo in the early- and late-onset Val30Met, and non-Val30Met groups, respectively. Across subgroups, the change from baseline to Week 66 in mNIS+7 composite score was generally well maintained, and the Norfolk QoL-DN total score improved with eplontersen versus worsening with placebo. The Polyneuropathy Disability score was maintained in most patients. From baseline to Week 65, the modified body mass index was maintained with eplontersen compared to a marked reduction (worsening) for placebo.
conclusionsFindings were suggestive of consistent benefits in reducing neuropathy impairment and improving QoL with eplontersen versus historical placebo, across TTR variants.
trial registrationClinicalTrials.gov: NCT04136184, NCT01737398.
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