Health TrialsbyHealthPanel

ATR 1072 in Participants With PRKAG2 Syndrome

ConditionsCardiac GlycogenosisCardiomyopathyHypertrophicWolff-Parkinson-White Syndrome
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorAtrium Therapeutics

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • ATR 1072

    Drug

    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.

Treatment groups

37 Participants
are divided into 2 treatment groups
Group A: Part A - MAD Dose Escalation Description Three sequential ascending doseExperimental treatment 1 intervention
Group B: Part B - Expansion Cohort at the Recommended Phase 2 DoseExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of treatment-emergent adverse events (TEAEs)

Incidence and severity of treatment-emergent adverse events (TEAEs).

Time frame
From first dose through end of study (approximately 48 weeks per participant)

Secondary outcomes

1

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.

Time frame
Through Week 24
2

Plasma pharmacokinetic (PK) parameters of ATR 1072

Peak Plasma Concentration (Cmax)

Time frame
Through Week 24
3

Plasma pharmacokinetic parameters of ATR 1072

Area under the plasma concentration versus time curve (AUC)

Time frame
Through week 48
4

Plasma pharmacokinetic parameters of ATR 1072

terminal half-life (t1/2)

Time frame
Through week 48

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Atrium Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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