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NCT Number: NCT07738107

ATR 1072 in Participants With PRKAG2 Syndrome

ATR 1072 is an investigational medicine being studied in adults with PRKAG2 Syndrome. This study will evaluate the safety of ATR 1072 and how the body processes the drug. Researchers will also assess how ATR 1072 affects PRKAG2 activity and measures of heart disease.

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Key information

Conditions

Cardiac Glycogenosis Arrhythmias, Cardiac Carbohydrate Metabolism, Inborn Errors Cardiac Conduction System Disease Cardiomyopathies Cardiomyopathy Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Heart Defects, Congenital Heart Diseases Hypertrophic Hypertrophy Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Pathological Conditions, Anatomical Pathological Conditions, Signs and Symptoms Pre-Excitation Syndromes Wolff-Parkinson-White Syndrome

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

St Luke's Mid American Heart Institute

Kansas City, Missouri, 64111, United States

Location status: Recruiting

Location contact

Michael Nassif, MD

CONTACT

[email protected]

816-932-3458

Micheal Nassif, MD

PRINCIPAL_INVESTIGATOR

About this study

ATR 1072 is an antibody-oligonucleotide conjugate (AOC) designed to reduce expression of PRKAG2 through targeted delivery of siRNA to cardiac tissue. This first-in-human, Phase 1/2, open-label, multicenter study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of multiple doses of ATR 1072 in adults with PRKAG2 Syndrome. The study consists of a multiple ascending dose portion (Part A) followed by a dose-expansion cohort at the dose selected in (Part A). After completion of a 48-week treatment period, participants in Parts A and B enter a 42-week extension period followed by a post-treatment follow-up of up to 18 weeks. Approximately 37 participants will be enrolled globally.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Adults 18 to 65 years of age.
  • Pathogenic, likely pathogenic, or variant of uncertain significance (VUS) in PRKAG2 confirmed by genetic testing.
  • Clinical manifestations consistent with PRKAG2 Syndrome.
  • Able and willing to comply with study procedures.
  • Able and willing to undergo endomyocardial biopsy procedures (Part A)

Exclusion criteria

  • Pregnancy or breastfeeding.
  • Significant hepatic, renal, hematologic, or cardiovascular abnormalities that may increase study risk.
  • NYHA Class IV heart failure or left ventricular ejection fraction <45%.
  • Recent clinically significant cardiovascular events or procedures.
  • Prior treatment with prohibited investigational therapies or oligonucleotide therapies within protocol-defined washout periods.
  • Any condition that, in the investigator's

Treatment and study plan

ATR 1072

Drug

ATR 1072 is an investigational therapy that delivers an siRNA to reduce PRKAG2 gene expression. Administered as an intravenous (IV) infusion every 6 weeks (Q6W). Formulated as a sterile liquid for injection.

Primary outcomes

  1. Incidence of treatment-emergent adverse events (TEAEs)

    Time frame: From first dose through end of study (approximately 48 weeks per participant)

    Incidence and severity of treatment-emergent adverse events (TEAEs).

Secondary outcomes

  1. Change from baseline in cardiac muscle tissue concentration of ATR 1072 siRNA

    Time frame: Through Week 24

    siRNA content in endomyocardial biopsy samples measured by quantitative bioanalytical methods to characterize cardiac tissue distribution.

  2. Plasma pharmacokinetic (PK) parameters of ATR 1072

    Time frame: Through Week 24

    Peak Plasma Concentration (Cmax)

  3. Plasma pharmacokinetic parameters of ATR 1072

    Time frame: Through week 48

    Area under the plasma concentration versus time curve (AUC)

  4. Plasma pharmacokinetic parameters of ATR 1072

    Time frame: Through week 48

    terminal half-life (t1/2)

Study contacts

Contact information is provided by the study sponsor or research team.

Patient Recruitment Coordinator

CONTACT

[email protected]

283-227-6543

Sponsors and collaborators

Lead sponsor

Atrium Therapeutics

Industry

Registry information

Official study title

A Phase 1/2, Open-Label Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous ATR 1072 for the Treatment of PRKAG2 Syndrome

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Jul 31, 2026
Registry last updated
Sep 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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