CARDIO-TTRansform
A study to evaluate the efficacy and safety of AKCEA-TTR-LRx in participants with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
AKCEA-TTR-LRx is an RNA inhibitor TTR lowering drug, belonging to the antisense oligonucleotide drug class. This type of drug inhibits the production of the TTR protein. It is administered by subcutaneous injection.
The trial
This is a phase 3 global, double-blind, randomised study to evaluate the efficacy and safety of AKCEA-TTR-LRx compared to placebo in patients with ATTR cardiomyopathy (ATTR-CM).
What the study involves for patients
Participants will receive AKCEA-TTR-LRx or placebo by subcutaneous injection every 4 weeks for 120 weeks. All participants will also receive vitamin A supplements.
Who can take part in the trial
Patients diagnosed with ATTR cardiomyopathy may take part.
Patients may not take part in the study if they have cardiomyopathy not primarily caused by ATTR-CM, AL amyloidosis, prior liver, or heart transplant, current or previous treatment with another RNA inhibitor drug.
For a full list of inclusion and exclusion criteria, see the www.clinicaltrials.gov website.
Recruitment of over 1,000 participants is planned.
