ATR 1072 in Participants With PRKAG2 Syndrome

NCT07738107 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: ATR 1072-CS1
• Study Type: interventional
• Target: 37
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Cardiac Glycogenosis; Cardiomyopathy; Hypertrophic; Wolff-Parkinson-White Syndrome

Keywords

AMPK; TfR1; transferrin receptor; ATR 1072; targeted RNA delivery; siRNA; antibody-oligonucleotide conjugate (AOC); glycogen storage disease; PRKAG2 Syndrome; cardiac glycogenosis; cardiomyopathy; PRKAG2; genetic cardiomyopathy; corventis

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs)

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

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

Secondary Outcomes (4)

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

  Through Week 24

• Plasma pharmacokinetic (PK) parameters of ATR 1072

  Through Week 24

• Plasma pharmacokinetic parameters of ATR 1072

  Through week 48

• Plasma pharmacokinetic parameters of ATR 1072

  Through week 48

Study Arms (2)

Part A - MAD Dose Escalation Description Three sequential ascending dose

EXPERIMENTAL

Three sequential ascending dose cohorts (Dose Level 1, Dose Level 2 and Dose Level 3) with approximately 4 evaluable participants per cohort. ATR 1072 administered intravenously (IV) every 6 weeks (Q6W) for up to 8 doses (48-week treatment period).

Drug: ATR 1072

Part B - Expansion Cohort at the Recommended Phase 2 Dose

EXPERIMENTAL

Approximately 25 participants enrolled at the recommended phase 2 dose determined in Part A. ATR 1072 administered IV Q6W to further evaluate safety, PK, and pharmacodynamics.

Drug: ATR 1072

Interventions

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.

Part A - MAD Dose Escalation Description Three sequential ascending dose; Part B - Expansion Cohort at the Recommended Phase 2 Dose

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Atrium Therapeutics: lead

MeSH Terms

Conditions

Cardiomyopathies; Hypertrophy; Wolff-Parkinson-White Syndrome; Glycogen Storage Disease

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Pre-Excitation Syndromes; Arrhythmias, Cardiac; Cardiac Conduction System Disease; Heart Defects, Congenital; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Patient Recruitment Coordinator

CONTACT

283-227-6543; clinicaltrials@atrium-tx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: None (Open Label)
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Sequential Assignment (MAD escalation followed by expansion)

Study Record Dates

• First Submitted: June 26, 2026
• First Posted: July 31, 2026
• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): October 1, 2028
• Last Updated: July 31, 2026

Record last verified: 2026-06