Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy (NCT04418024) | Clinical Trial Compass
WithdrawnPhase 3
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
Stopped: After a careful review of the currently available treatments worldwide for patients with ATTR-polyneuropathy, Eidos has made the decision to halt the current study design.
United States0Started 2020-10-21
Plain-language summary
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
Who can participate
Age range18 Years – 90 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Be male or female ≥18 to ≤90 years of age;
* Have Stage I or II symptoms (polyneuropathy disability \[PND\] ≤IIIa) of ATTR-PN and an established diagnosis of ATTR-PN as defined by physical exam findings and/or neurophysiological test findings consistent with the diagnosis of ATTR-PN;
* Have an NIS of 5 to 130 (inclusive) during screening;
* Have a nerve conduction studies (NCS) score \[sum of the sural sensory nerve action potential (SNAP), tibial compound muscle action potential (CMAP), ulnar SNAP, ulnar CMAP, and peroneal CMAP\] of ≥2 points during screening. NCS is a component of mNIS+7;
* Have a mutation consistent with ATTR-PN either documented in medical history or confirmed by genotyping obtained at Screening prior to randomization. \*No genetic testing is needed for subjects who are recipients of domino liver transplants;
* Have an anticipated survival of ≥2 years
* Have Karnofsky performance status ≥60 %;
Exclusion Criteria:
* Had a prior liver transplantation or is planning to undergo liver transplantation with a wild-type organ graft as treatment for symptomatic ATTR-PN during the study period.
Note: Recipients of a "domino" liver transplant from an ATTR-PN donor who have developed ATTR-PN mediated by their graft are allowed under this protocol, as long as re-transplantation to treat ATTR-PN is not planned during the study period and meets all other eligibility criteria;
* Has sensorimotor or autonomic neuropathy not related to ATTR-PN; f…
What they're measuring
1
Change from baseline to Month 18 of treatment in Modified Neuropathy Impairment