Status:
WITHDRAWN
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
Lead Sponsor:
Eidos Therapeutics, a BridgeBio company
Conditions:
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eligibility:
All Genders
18-90 years
Phase:
PHASE3
Brief 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)
Detailed Description
\*\* See updated study design under ClinicalTrials.gov Identifier NCT04882735. \*\* Transthyretin amyloid polyneuropathy (ATTR-PN), also called "Familial Transthyretin-Mediated Amyloid Polyneuropathy...
Eligibility Criteria
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;
- Has sensorimotor or autonomic neuropathy not related to ATTR-PN; for example, autoimmune disease or monoclonal gammopathy, malignancy, or alcohol abuse;
- Has Vitamin B-12 levels below the lower limit of normal (LLN);
- Has clinical evidence of untreated hyper/hypothyroidism;
- Has leptomeningeal TTR amyloidosis;
- Has Type 1 diabetes;
- Has had Type 2 diabetes for ≥5 years;
- 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
- 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
Key Trial Info
Start Date :
October 21 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 30 2024
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04418024
Start Date
October 21 2020
End Date
April 30 2024
Last Update
September 19 2024
Active Locations (1)
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1
Eidos Therapeutics
San Francisco, California, United States, 94104