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

NCT ID NCT07738107

Disease control Sponsor: Atrium Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 31, 2026 · Last updated Sep 18, 2026 · Updated 1 time

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 37 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2026

Expected to finish

Oct 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Adults 18 to 65 years of age. 2. Pathogenic, likely pathogenic, or variant of uncertain significance (VUS) in PRKAG2 confirmed by genetic testing. 3. Clinical manifestations consistent with PRKAG2 Syndrome. 4. Able and willing to comply with study procedures. 5. Able and willing to undergo endomyocardial biopsy procedures (Part A) Exclusion Criteria: 1. Pregnancy or breastfeeding. 2. Significant hepatic, renal, hematologic, or cardiovascular abnormalities that may increase study risk. 3. NYHA Class IV heart failure or left ventricular ejection fraction \<45%. 4. Recent clinically significant cardiovascular events or procedures. 5. Prior treatment with prohibited investigational therapies or oligonucleotide therapies within protocol-defined washout periods. 6. Any condition that, in the investigator's

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • St Luke's Mid American Heart Institute

    RECRUITING

    Kansas City, Missouri, 64111, United States

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