RightFit
RecruitingPhase 1 / Phase 2

ATR 1072 in Participants With PRKAG2 Syndrome

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 65 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 18 weeks

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "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."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests ATR 1072, an experimental medicine, in adults with PRKAG2 syndrome. Researchers will check its safety and how the body processes it. They will also study its effects on PRKAG2 activity and signs of heart disease.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You are 18 to 65 years old.
  • A genetic test shows a PRKAG2 gene change that causes disease, likely causes disease, or has an unknown effect.
  • You have signs of PRKAG2 syndrome.
  • You can and will follow the study procedures.
  • For Part A, you can and will have small samples of heart muscle removed for testing.

Who can't join

  • You are pregnant or breastfeeding.
  • You have major liver, kidney, blood, heart, or blood vessel problems that may make the study riskier.
  • You have the most severe level of heart failure (NYHA Class IV), or your heart’s main pumping chamber pumps out less than 45% of its blood with each beat.
  • You recently had a major heart or blood vessel event or procedure.
  • You received certain experimental treatments or oligonucleotide treatments (medicines made from short pieces of genetic material) too close to the study’s start.
  • The final exclusion rule is cut off in the provided text.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility 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

Study sites (1)

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