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
- Are there results?
- Not yet: the study is under way
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.
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07738107
- Acronym / sponsor ID
- ATR 1072-CS1
- Keywords
- AMPK, TfR1, transferrin receptor, ATR 1072, targeted RNA delivery, siRNA, antibody-oligonucleotide conjugate (AOC), glycogen storage disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 37 participants (estimated)
- Lead sponsor
- Atrium TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 14 Sep 2026 (actual)
- Primary completion
- May 2028 (estimated)
- Study completion
- Oct 2028 (estimated)
- First posted
- 31 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Sequential Assignment (MAD escalation followed by expansion)
| Arm | Type | Receives |
|---|---|---|
| Part A - MAD Dose Escalation Description Three sequential ascending dose | Experimental | Drug: ATR 1072Three 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). |
| Part B - Expansion Cohort at the Recommended Phase 2 Dose | Experimental | Drug: ATR 1072Approximately 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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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)
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ATR 1072ATR 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. | Drug | Part A - MAD Dose Escalation Description Three sequential ascending dose, Part B - Expansion Cohort at the Recommended Phase 2 Dose |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: From first dose through end of study (approximately 48 weeks per participant)
Incidence and severity of treatment-emergent adverse events (TEAEs).
4 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patient Recruitment Coordinator (283-227-6543, clinicaltrials@atrium-tx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) (time frame: From first dose through end of study (approximately 48 weeks per participant)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.