Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
Megadott ok: 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.
Key facts
- Where
- San Francisco, Egyesült ÁllamokSee the site
- Phase
- 3. fázis
Where in Egyesült Államok
- Eidos TherapeuticsSan Francisco, Egyesült ÁllamokNo longer taking part
Trial details
- Who runs it
- Eidos Therapeutics, a BridgeBio company
- Trial code
- NCT04418024
- Record updated
- 2024. szeptember 19.
- Original description
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)Read full original descriptionShow less
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects with Symptomatic Transthyretin Amyloid Polyneuropathy (ATTRibute-PN Trial)
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)
Inclusion: 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: 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; Is currently undergoing treatment for ATTR-PN with patisiran, inotersen, or other gene silencing agents, marketed drug products lacking a label indication for ATTR- PN (e.g., diflunisal, doxycycline), natural products or derivatives used as unproven therapies for ATTR-PN (e.g., green tea extract, tauroursodeoxycholic acid [TUDCA]/ursodiol), within 14 days, or 90 days for patisiran and 180 days for inotersen prior to dosing. Prior to screening, tafamidis, if already prescribed to potential subjects as part of their established background therapy, is allowed at the labeled dosage and administration of 20 mg/day for the treatment of ATTR-PN with, i in the opinion of the Investigator, evidence of disease progression while on tafamidis treatment; Has sensorimotor or autonomic neuropathy not related to ATTR-PN; for example, autoimmune disease or monoclonal gammopathy, malignancy, or alcohol abuse; Has Type 1 diabetes; Has active hepatitis B or C or known human immunodeficiency virus (HIV) infection; Has NYHA heart failure classification >Class II; Had a malignancy within 2 years, except for basal or squamous cell carcinoma of; Has had Type 2 diabetes for ≥5 years; Has Vitamin B-12 levels below the lower limit of normal (LLN); Has clinical evidence of untreated hyper/hypothyroidism; Has leptomeningeal TTR amyloidosis.