StudyFinder
A Phase 3, Randomized, Multicenter, Double-Blind, PlaceboControlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Status: Recruiting
ACT-EARLY is a research study to see if an investigational drug, acoramidis (AG10), is safe and effective in preventing or delaying symptoms of a disease called Transthyretin Amyloidosis (ATTR).This research study will test whether acoramidis treatment in people who do not have the disease but who carry a genetic predisposition to developing the disease is safe and effective in preventing or delaying ATTR disease (genetic predisposition is also referred to as genetic variant).
Sex: Male or Female
Age: 18 years and over
Healthy Volunteers:
Inclusion Criteria:
Carries a specific change (mutation) in the TTR gene that is known, or strongly believed, to cause disease. However, they currently have no signs or symptoms of that disease.
The participant's age must be close to the age when people with their specific TTR gene variant typically start developing symptoms. Specifically, they must be no more than 10 years younger than that expected age. They can be older than the expected age, as long as they are 75 years old or younger.
Exclusion Criteria:
No current stymptoms caused by ATTR amyloidosis.
No current tratment for ATTR amyloidosisInterventions:
Drug/Device: Acoramidis
Conditions:
Transthyretin Amyloidosis (ATTR)
Keywords:
Acoramidis, Amyloidosis, ATTR Cardiomyopathy (ATTR-CM), ATTR Polyneuropathy (ATTR-PN), Transthyretin Amyloidosis (ATTR)
Contact(s): Kristin Clark - kclark2@hfhs.org
Principal Investigator: Celeste Williams
Principal Investigator ID: cwillia6@hfhs.org
Phase: III
See this study on ClinicalTrials.gov