Plain-English translation of NCT07241104 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This is one of the first times this treatment has been tested in people.
This is the first study of in humans with a rare genetic heart condition called phospholamban R14del dilated cardiomyopathy. Researchers are testing whether this new medication is safe and how the body processes it. The study will start with small doses given as single injections, and then move to people receiving multiple doses over time.
This genetic form of heart disease causes the heart muscle to weaken and enlarge, leading to heart failure that is difficult to treat with standard medications. Researchers believe this medication may help strengthen heart function and improve outcomes for people with this specific genetic mutation.
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The trial has two main phases. First, you would receive a single injection of the medication and be monitored for safety. If that goes well, some participants will then move to a second phase where they receive multiple injections over time. Throughout the study, you'll have regular clinic visits, blood tests, heart imaging (like echocardiograms), and heart rhythm monitoring to check how the medication is working and whether it is safe.
AI-generated summary from trial data · Jul 19, 2026 · Not medical advice
Netherlands