Plain-English translation of NCT07738107 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing a new medication called designed specifically for people with PRKAG2 Syndrome, a genetic heart condition that causes abnormal protein accumulation in the heart muscle. The medication works by delivering a special molecule directly to heart tissue to reduce the harmful protein. Researchers want to find out if this treatment is safe and whether it can help improve heart function.
PRKAG2 Syndrome is a rare genetic condition that can lead to serious heart problems like enlarged hearts and dangerous heart rhythms, and there are currently very few treatment options available. This medication represents a new approach that targets the root cause of the disease, offering hope to patients who don't have many choices for treatment.
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You would receive intravenous infusions of the medication every 6 weeks for 48 weeks (about one year), either at the lowest dose level first as part of the safety-testing phase, or at the selected dose if you join the later expansion group. The study involves multiple clinic visits for heart monitoring, blood tests, and possibly heart tissue biopsies. After the 48-week treatment period, you would continue in a follow-up phase for an additional 60 weeks to monitor how you respond after treatment ends.
AI-generated summary from trial data · Sep 19, 2026 · Not medical advice
United States
Phase
Safety & dosing / Early efficacy
Sponsor
Atrium Therapeutics
Enrollment target
~37 participants
Started
September 2026
Primary completion
May 2028
Age range
18 Years – 65 Years
Last updated on clinicaltrials.gov in September 2026.
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Central contact
Patient Recruitment Coordinator
Atrium Therapeutics
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