ATR 1072 in Participants With PRKAG2 Syndrome

Phase 2RecruitingNCT07738107

Atrium Therapeutics37 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cardiac Glycogenosis Cardiomyopathy Hypertrophic Wolff-Parkinson-White Syndrome

Interventions

ATR 1072DRUG

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.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (1)

St Luke's Mid American Heart Institute

Kansas City, Missouri, United States

RECRUITING

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events (TEAEs)

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

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

Secondary Outcomes

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

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

Time frame: Through Week 24

Plasma pharmacokinetic (PK) parameters of ATR 1072

Peak Plasma Concentration (Cmax)

Time frame: Through Week 24

Plasma pharmacokinetic parameters of ATR 1072

Area under the plasma concentration versus time curve (AUC)

Time frame: Through week 48

Plasma pharmacokinetic parameters of ATR 1072

terminal half-life (t1/2)

Time frame: Through week 48

Central Contacts

Patient Recruitment Coordinator

CONTACT

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

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.