ATR 1072 in Participants With PRKAG2 Syndrome | NCT07738107

NCT07738107

Overview

NCT ID: NCT07738107
Lead Sponsor: Atrium TherapeuticsINDUSTRY
Official 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
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
Interventions

Status and Timeline

Status: Not yet recruiting
Last Update Posted: Jul 31, 2026Actual
Start Date: Oct 2026Estimated
Primary Completion Date: May 2028Estimated
Completion Date

Participation Criteria

Ages Eligible for Study: Adult (18-64)Older Adult (65+)
Sexes Eligible for Study: All
Accepts Healthy Volunteers?: No
Eligibility Criteria: 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

Study Plan

Study Type: Interventional
Phase: Phase 1Phase 2
Enrollment: 37Estimated
Primary Purpose: Treatment
Allocation: Non-Randomized
Interventional Model: Sequential Assignment
Masking

Contacts and Locations

Central Contacts: Patient Recruitment CoordinatorContact
clinicaltrials@atrium-tx.com

Similar Studies

Participant Group/Arms

Part A - MAD Dose Escalation Description Three sequential ascending doseExperimental

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 DoseExperimental

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