Cardiac GlycogenosisPhase 1/2NCT07738107
ATR 1072 in Participants With PRKAG2 Syndrome
A Phase 1/2 study testing ATR 1072 in people with Cardiac Glycogenosis and Cardiomyopathy, run by Atrium Therapeutics at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Cardiac Glycogenosis, Cardiomyopathy, Hypertrophic
- What is being tested?
- ATR 1072
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Sep 2026to Oct 2028
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
St Luke's Mid American Heart Institute
Kansas City, Missouri, 64111
Michael Nassif, MD, 816-932-3458, michael.nassif@bjc.org
Micheal Nassif, MD
About this study
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
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.
Read the 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.
- Being tested
- ATR 1072
- Keywords
- AMPK, TfR1, transferrin receptor, ATR 1072, targeted RNA delivery, siRNA, antibody-oligonucleotide conjugate (AOC), glycogen storage disease, PRKAG2 Syndrome, cardiac glycogenosis, cardiomyopathy, PRKAG2, genetic cardiomyopathy, corventis
- Medical subject headings
- Cardiomyopathies, Hypertrophy, Wolff-Parkinson-White Syndrome, Glycogen Storage Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| Part A - MAD Dose Escalation Description Three sequential ascending dose | Experimental | 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). Receives: Drug: ATR 1072 |
| Part B - Expansion Cohort at the Recommended Phase 2 Dose | Experimental | 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. Receives: Drug: ATR 1072 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs)
Incidence and severity of treatment-emergent adverse events (TEAEs).
Measured: From first dose through end of study (approximately 48 weeks per participant)
Secondary outcomes (4)
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.
Measured: Through Week 24
Plasma pharmacokinetic (PK) parameters of ATR 1072
Peak Plasma Concentration (Cmax)
Measured: Through Week 24
Plasma pharmacokinetic parameters of ATR 1072
Area under the plasma concentration versus time curve (AUC)
Measured: Through week 48
Plasma pharmacokinetic parameters of ATR 1072
terminal half-life (t1/2)
Measured: Through week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study start14 Sep 2026
- Primary completionMay 2028 (estimated)
- Study completionOct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cardiac GlycogenosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Sequential Assignment (MAD escalation followed by expansion)
- Masking
- None (open label)
- Enrolment
- 37 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Atrium Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07738107
- Sponsor's protocol ID
- ATR 1072-CS1
- Status last verified
- Sep 2026
- First submitted
- 26 Jun 2026
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: No individual participant data (IPD) sharing plan has been finalized at the time of registration. The sponsor is committed to transparent reporting of study results. IPD sharing practices will be determined in accordance with applicable regulations, sponsor policies, and any applicable collaboration agreements.