Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy Clinical Trial

Official title:
A Phase 3, Open-Label, Multicenter, Single-Arm Study to Evaluate the Efficacy and Safety of Acoramidis in Subjects With Symptomatic Transthyretin Amyloid Polyneuropathy (ATTRibute-PN Study)

Status Withdrawn

Clinical Trial Summary

Phase 3 efficacy and safety of acoramidis in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)

Clinical Trial 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 estimated that around 10,000 people in the world are affected. In ATTR-PN, amyloid builds up in the nerves that detect temperature, pain, and touch. Patients with ATTR-PN can experience a loss of sensation, tingling, numbness, or pain in the hands and feet (also called peripheral neuropathy). In this study Eidos, a BridgeBio Company, is researching the investigational drug acoramidis (AG10) hydrochloride (HCl) 800mg administered orally twice a day. Through the study, Eidos/BridgeBio wants to evaluate the efficacy and safety of acoramidis in patients with ATTR-PN. The primary outcome of the study is to determine the efficacy of acoramidis in the treatment of subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN). At the end of 18 months, participants will be eligible to continue to receive acoramidis to evaluate the long-term safety and tolerability of acoramidis. ;

Study Design

Related Conditions & MeSH terms

Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis
Polyneuropathies
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Clinical Trial Details

NCT number	NCT04882735
Study type	Interventional
Source	Eidos Therapeutics, a BridgeBio company
Contact
Status	Withdrawn
Phase	Phase 3
Start date	September 2021
Completion date	October 2026

View Details

See also
Status	Clinical Trial	Phase
Withdrawn	`NCT04418024` - Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Polyneurophathy	Phase 3
Active, not recruiting	`NCT04601051` - Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)	Phase 1
Recruiting	`NCT03237494` - TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
Recruiting	`NCT05697861` - Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
Withdrawn	`NCT02713880` - Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)