Status:
WITHDRAWN
Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)
Lead Sponsor:
Eidos Therapeutics, a BridgeBio company
Conditions:
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eligibility:
All Genders
18-90 years
Phase:
PHASE3
Brief Summary
Phase 3 efficacy and safety of acoramidis in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
Detailed Description
Transthyretin amyloid polyneuropathy (ATTR-PN), also called "Familial Transthyretin-Mediated Amyloid Polyneuropathy (FAP)" is a hereditary condition caused by mutations in the TTR gene. It is estimate...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
September 8 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2026
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04882735
Start Date
September 8 2021
End Date
October 1 2026
Last Update
September 19 2024
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