Recruiting
Phase 1
Phase 2

ATR 1072

Sponsor:

Atrium Therapeutics

Code:

NCT07738107

Conditions

Cardiac Glycogenosis

Cardiomyopathy

Hypertrophic

Wolff-Parkinson-White Syndrome

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

Interventions

ATR 1072

Study Details

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

Study ID

NCT07738107

Start date

Sep 14, 2026

Status verified date

Sep, 2026

Completion date

Oct, 2028

Anticipated

Primary completion date

May, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

37 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Part A - MAD Dose Escalation Description Three sequential ascending dose

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).

experimental: Part B - Expansion Cohort at the Recommended Phase 2 Dose

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.

Interventions

ATR 1072

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 outcome measure

  • Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: From first dose through end of study (approximately 48 weeks per participant) ]

Central Contacts and Locations

Central contacts

Patient Recruitment Coordinator

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

Locations

St Luke's Mid American Heart Institute

Recruiting

Kansas City, Missouri, United States, 64111

Contacts

Michael Nassif, MD

816-932-3458michael.nassif@bjc.org

Principal Investigator:

Micheal Nassif, MD

More Information

Sponsor

Atrium Therapeutics

Last update posted

Sep 18, 2026

Last verified

Sep, 2026

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-19. This information was provided to ClinicalTrials.gov by Atrium Therapeutics on 2026-09-18.