Can a Gene-Silencing drug tame a rare heart disease?

NCT ID NCT07738107

Not yet recruiting Disease control Sponsor: Atrium Therapeutics Source: ClinicalTrials.gov ↗

First seen Jul 31, 2026 · Last updated Jul 31, 2026

Summary

This trial tests an investigational medicine called ATR 1072 in adults with PRKAG2 syndrome, a genetic condition that causes abnormal heart muscle thickening and rhythm problems. The drug is designed to reduce the activity of the faulty PRKAG2 gene. Researchers are evaluating its safety, how the body processes it, and whether it can improve measures of heart disease.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: ATR 1072, an investigational siRNA therapy given by IV infusion to reduce PRKAG2 gene expression
What this could lead to: If successful, this could lead to a targeted treatment that slows or reverses heart damage in PRKAG2 syndrome, potentially reducing the need for heart transplants.
What could go wrong: This is an early-stage trial with a small number of participants, so safety and effectiveness are not yet proven. The therapy may not work for everyone and could have side effects.

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Conditions

The condition(s) this trial relates to.

cardiomyopathy disorder of glycogen metabolism Hypertrophy PRKAG2-related cardiomyopathy Wolff-Parkinson-White syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CARDIAC GLYCOGENOSIS CARDIOMYOPATHY HYPERTROPHIC WOLFF-PARKINSON-WHITE SYNDROME

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