

The CARDIO-TTRansform study is currently enrolling participants to evaluate the safety and efficacy of eplontersen, an investigational antisense RNA-targeted therapy, for people living with hereditary and wild-type cardiac amyloidosis.
In a phase I, randomized, placebo-controlled study, eplontersen given at a 45 mg, 60 mg or 90 mg dose, by subcutaneous injection every four weeks in 36 healthy volunteers achieved a mean reduction in serum TTR of 86%, 91% and 94%, respectively, compared to baseline.
Interested in learning more about the CARDIO-TTRansform study?
Information regarding the Ionis CARDIO-TTRansform trial can be downloaded below or Click Here
Notable publications about ATTR-CM and eplontersen:
- Patient-reported burden of hereditary transthyretin amyloidosis on functioning and well-being [Lovley; 2021]
- Evaluation of the Efficacy and Safety of AKCEA-TTR-LRx (ION-682884) in Patients with Transthyretin-mediated Amyloid Cardiomyopathy: The CARDIO-TTRansform Study [Maurer; 2021] [Note: AKCEA-TTR-LRx is now eplontersen]
- Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness [Nativi-Nicolau; 2021]
- Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data [Viney; 2020]
- Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy [Ackermann; 2012]
- Rate of Progression of Transthyretin Amyloidosis [Benson; 2011]
- Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides [Benson; 2006]
If you know of or have patients that may benefit from joining the CARDIO-TTRansform study, please contact us at [email protected] or 844-413-0219. We look forward to hearing from you and working with you throughout this trial.