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Not yet recruiting NCT07738107

ATR 1072 in Participants With PRKAG2 Syndrome

Phase I / Phase II Interventional Cardiac Glycogenosis Cardiomyopathy Hypertrophic Wolff-Parkinson-White Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ATR 1072.
Who it may be relevant to
Registry conditions: Cardiac Glycogenosis, Cardiomyopathy, Hypertrophic, Wolff-Parkinson-White Syndrome. Basic parameters: 18 years — 65 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

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.

Detailed description

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.

Interventions

  • Drug 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 measures

  • Incidence of treatment-emergent adverse events (TEAEs) [Time frame: From first dose through end of study (approximately 48 weeks per participant)]
Secondary outcome measures (4)
  • Change from baseline in cardiac muscle tissue concentration of ATR 1072 siRNA [Time frame: Through Week 24]
  • Plasma pharmacokinetic (PK) parameters of ATR 1072 [Time frame: Through Week 24]
  • Plasma pharmacokinetic parameters of ATR 1072 [Time frame: Through week 48]
  • Plasma pharmacokinetic parameters of ATR 1072 [Time frame: Through week 48]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07738107 · ATR 1072-CS1

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗