Eplontersen for Cardiomyopathy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called eplontersen for individuals with ATTR-CM, a heart condition characterized by amyloid protein buildup in the heart. The researchers aim to determine if eplontersen is more effective than a placebo (an inactive substance resembling the drug) when combined with standard care. Participants will receive injections every four weeks. Suitable candidates have been diagnosed with ATTR-CM and have amyloid deposits confirmed by specific tests. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants an opportunity to contribute to potentially groundbreaking treatment advancements.
Will I have to stop taking my current medications?
Yes, if you are currently taking diflunisal, doxycycline, or certain calcium-channel blockers like verapamil or diltiazem, you will need to stop taking them at least 14 days before joining the trial.
Is there any evidence suggesting that eplontersen is likely to be safe for humans?
Research has shown that eplontersen, the treatment under study, has a promising safety record. In earlier studies, patients with hereditary transthyretin amyloidosis received eplontersen, and most tolerated it well. Some deaths occurred, but these resulted from complications of the disease itself, such as heart rhythm issues and brain bleeding, not the treatment.
The current study is in a later stage, indicating that eplontersen has already passed initial safety tests. While no treatment is without risk, eplontersen is considered safe enough for testing in a larger group. It is important to consult a doctor to understand what this means for individual circumstances.12345Why do researchers think this study treatment might be promising for cardiomyopathy?
Eplontersen is unique because it targets the root cause of cardiomyopathy related to transthyretin amyloidosis by using an antisense oligonucleotide approach. Unlike traditional treatments that mainly address symptoms, Eplontersen reduces the production of the transthyretin protein, which is responsible for amyloid buildup in the heart. This targeted mechanism of action has the potential to not only slow down disease progression but also improve heart function, offering hope for better outcomes. Additionally, Eplontersen is administered via a convenient subcutaneous injection once every four weeks, potentially enhancing patient adherence compared to more frequent dosing schedules.
What evidence suggests that eplontersen might be an effective treatment for cardiomyopathy?
Research has shown that eplontersen, which participants in this trial may receive, is designed to slow the progression of cardiomyopathy by reducing the production of transthyretin, a protein. In earlier studies, patients who received eplontersen had lower transthyretin levels in their blood, offering hope for managing the disease. The goal is to reduce the buildup of transthyretin, which can damage heart tissues. Early results suggest that eplontersen may effectively address this issue, potentially improving heart function in people with this condition.12345
Are You a Good Fit for This Trial?
This trial is for adults with ATTR-CM, a type of heart disease caused by amyloid deposits. Participants must have certain heart measurements and confirmed amyloid deposits but can't be on specific heart medications, have had recent major cardiovascular events or procedures, or expect to need a heart transplant or assist device within a year.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive subcutaneous injections of either eplontersen or placebo once every 4 weeks, along with daily supplemental doses of vitamin A
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AKCEA-TTR-LRx
- Eplontersen
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ionis Pharmaceuticals, Inc.
Lead Sponsor
Dr. Brett P. Monia
Ionis Pharmaceuticals, Inc.
Chief Executive Officer since 2020
PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College
Dr. Eric Bastings
Ionis Pharmaceuticals, Inc.
Chief Medical Officer
MD
AstraZeneca
Industry Sponsor
Sir Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Dr. Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology