Testing a new gene therapy for heart disease caused by amyloid protein buildup

Plain-English translation of NCT06128629 on ClinicalTrials.gov ↗ · Source last updated March 2026 · Translation generated June 2026 · How we translate trials

Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.

What is this trial?

This study is testing whether , an experimental gene therapy, can help treat a rare inherited heart disease where abnormal protein builds up and damages the heart. About 1,200 people will receive either a single dose of this medication or a placebo (salt water), and researchers will compare how well each group does over time. The goal is to see if this treatment can improve heart function and quality of life for people living with this condition.

This condition is life-threatening and currently has limited treatment options. Researchers believe this medication may work differently than existing treatments by addressing the root cause of the problem, and this trial will test whether it's safe and effective enough to help patients.

Do you qualify?

You likely qualify if…

you have been diagnosed with transthyretin amyloidosis (a rare genetic disease causing abnormal protein to damage the heart)
you have heart failure symptoms that are stable and well-managed with current medications
your blood test results show a specific marker of heart stress within the range the study is looking for
you are willing to take vitamin A supplements throughout the study period

You likely don't qualify if…

you have hepatitis B, hepatitis C, or HIV infection
you are currently taking or have recently taken other amyloidosis medications (patisiran, inotersen, eplontersen, or vutrisiran)
you have severe kidney disease or liver disease
you have active cancer or a history of cancer within the past 3 years
you are unable to manage your blood pressure or are in the most severe stage of heart failure

What participation looks like

You will receive a single intravenous infusion (through a vein in your arm) of either the experimental medication or a placebo — you won't know which one. Researchers will then monitor you over time with blood tests and heart function assessments to see how you respond. You'll need to commit to vitamin A supplementation for the duration of the study and maintain regular follow-up visits with the research team.

AI-generated summary from trial data · Jun 16, 2026 · Not medical advice

Trial locations(132 sites)

United States

University of Arizona, Tucson, Arizona

Cedars-Sinai Medical Center, Los Angeles, California

Western Greater Los Angeles VA Medical Center, Los Angeles, California

Stanford University, Palo Alto, California

University of California, San Diego (UCSD) - Medical Center, San Diego, California