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ATR 1072 in Participants With PRKAG2 Syndrome

Not Yet RecruitingPhase 1, Phase 2

Sponsor: Atrium Therapeutics

Conditions: Cardiac Glycogenosis, Cardiomyopathy, Hypertrophic, Wolff-Parkinson-White Syndrome

Interventions: ATR 1072

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.

Eligibility overview

Sex: ALL

Age: 18 Years to 65 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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