Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 10:34 PM
Ignite Modification Date:
2025-12-24 @ 10:34 PM
NCT ID:
NCT04882735
Eligibility Criteria:
Inclusion Criteria:
* Male or female ≥18 to ≤90 years of age;
* Have Stage I or II symptoms (polyneuropathy disability \[PND\] ≤IIIb) of ATTR-PN and an established diagnosis of ATTR-PN as defined by physical examination 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 enrollment. No genetic testing is needed for subjects who are recipients of domino liver transplants;
* Have an anticipated survival of \>2 years in the opinion of the investigator;
* 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; for example, due to autoimmune disease or monoclonal gammopathy, malignancy, or alcohol abuse;
* Has Vitamin B-12 levels below the lower limit of normal (LLN) at Screening;
* Has clinical evidence of untreated hyperthyroidism or hypothyroidism;
* Has leptomeningeal TTR amyloidosis;
* Has Type 1 diabetes;
* Has had Type 2 diabetes for ≥5 years;
* Has a documented case of hepatitis B or C at Screening;
* Known history of human immunodeficiency virus (HIV) infection;
* Has NYHA heart failure classification \>Class II;
* Had acute coronary syndrome, uncontrolled cardiac arrhythmia, or a stroke within 90 days prior to Screening;
* Has estimated glomerular filtration rate (eGFR) by Modification of Diet for Renal Disease (MDRD) formula \<30 mL/min/1.73 m2 at Screening;
* Has abnormal liver function tests at Screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3 × upper limit of normal (ULN) or total bilirubin \>3 × ULN;
* Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
* Has known hypersensitivity to acoramidis, its metabolites, or formulation excipients.
* Is currently undergoing treatment for ATTR-PN with tafamidis, or patisiran, inotersen, or other knockdown agents, marketed drug products lacking a labeled 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 14 days for tafamidis or 90days for patisiran and 180 days for inotersen prior to dosing.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
18 Years
Maximum Age:
90 Years
Study:
NCT04882735
Study Brief:
Protocol Section:
NCT04882735