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Clinical trial Recruiting at 1 site Phase1; Phase2 Interventional Ages 18 to 65

ATR 1072 in PRKAG2 Syndrome

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

Run by Atrium Therapeutics · NCT07738107 · Data updated 2026-11-12

A first-in-human study tests ATR 1072 to see if it is safe and how it behaves in adults with PRKAG2 Syndrome.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Ages18 Years to 65 Years
SexAll
Enrollment37 (estimated)
SponsorAtrium Therapeutics
Industry-sponsored
Locations1 site (Kansas City, Missouri)
StatusRecruiting
StartedSeptember 14, 2026
Main results expectedMay 2028
Study endsOctober 2028
Registry record updatedSeptember 18, 2026
Trial IDNCT07738107 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Adults aged 18–65 with pathogenic, likely pathogenic, or VUS in PRKAG2 and clinical features of PRKAG2 Syndrome who are able to undergo endomyocardial biopsy (Part A).

Probably not a fit

Patients without PRKAG2 genetic findings or those who do not respond to the study drug may not experience benefit and could discontinue due to adverse effects or lack of efficacy.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label, multicenter study will assess safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of multiple doses of ATR 1072 in adults with PRKAG2 syndrome. It consists of a dose-escalation part to identify a suitable dose (Part A) followed by a dose-expansion cohort at that dose. After a 48-week treatment period, participants may enter a 42-week extension and up to 18 weeks of post-treatment follow-up, with approximately 37 participants globally. ATR 1072 is an antibody-oligonucleotide conjugate designed to reduce PRKAG2 expression by delivering siRNA to cardiac tissue, with optional endomyocardial biopsy in Part A to assess tissue-level effects.

Why it matters

Potential benefit: If successful, ATR 1072 could reduce disease-driving PRKAG2 activity and potentially improve heart-related outcomes in PRKAG2 Syndrome.

How similar studies have gone: This is a first-in-human study of ATR 1072; prior related approaches have shown safety signals in early-phase research for targeted RNA-delivery therapies in other conditions, but this exact approach is novel for PRKAG2 Syndrome.

Who can join: the full rules

Show full inclusion / exclusion 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

Where it's running

  • St Luke's Mid American Heart Institute
    Kansas City, Missouri, United States     Recruiting

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
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Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.