Phase 3 Clinical Study Concept

ASCEND-PRKAG2: Moving precision research forward in inherited cardiomyopathy.

The ASCEND-PRKAG2 study is a sample patient-facing Phase 3 site concept for Atrium Therapeutics. It is designed to illustrate how a complete recruitment experience could be presented for a theoretical PRKAG2 cardiac syndrome program.

Check Your Eligibility Read About the Study
  • ~240 adults targeted
  • 1:1 randomized assignment
  • 18 months treatment period

Eligibility Overview

Review potential entry criteria and key screening expectations before contacting a study center.

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Study Design

Understand how participants may be assigned, monitored, and followed throughout the program.

Explore Design

Site Access

See sample regional sites and pathways for participants, caregivers, and referring physicians.

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Who May Qualify

Potential eligibility for ASCEND-PRKAG2

You or a loved one may be able to participate if all core criteria below are met. Full criteria would be reviewed by the study team during screening.

  • Age 18 to 75 years at screening.
  • Known or suspected pathogenic variant associated with PRKAG2 syndrome.
  • Clinical history of PRKAG2-related cardiac findings, including hypertrophy and/or conduction abnormalities.
  • Able to attend scheduled clinic visits and complete required testing.
  • Willing to follow reproductive safety requirements during participation.

About PRKAG2 and the Program

Why this study matters

The PRKAG2 gene is involved in cellular energy signaling through AMP-activated protein kinase. Pathogenic variants can be associated with inherited cardiac disease patterns, including conduction abnormalities and cardiac hypertrophy.

ASCEND-PRKAG2 is designed to evaluate an investigational therapy in a controlled setting to better understand potential efficacy, safety, and longer-term outcomes in this patient population.

Clinical background reference: MedlinePlus Genetics - PRKAG2

Primary Objective

Assess treatment effect on validated cardiac functional endpoints in PRKAG2 syndrome.

Safety Monitoring

Continuous ECG strategy, laboratory monitoring, and structured adverse-event follow-up.

Participant Experience

Travel support and caregiver inclusion designed to reduce barriers to participation.

Global Scope

Multiregional recruitment framework with harmonized protocol and centralized quality review.

What to Expect

Sample participant journey

  1. Step 1

    Screening

    Up to 6 weeks for consent, baseline history, imaging, rhythm review, and lab confirmation.

  2. Step 2

    Treatment Period

    Approximately 18 months with scheduled visits, study treatment administration, and endpoint testing.

  3. Step 3

    Follow-up

    Post-treatment safety monitoring and durability assessment over a defined follow-up period.

  4. Step 4

    Extension Option

    Eligible participants may be offered entry into an open-label extension, pending approvals.

Common Questions

Frequently asked questions

This sample site illustrates how a Phase 3 PRKAG2 study could communicate goals, eligibility, and participation details to patients, caregivers, and clinicians.

Sample site regions

This mock locator view demonstrates how multicenter coverage may be communicated for patient access planning.

  • Boston, MA
  • Cleveland, OH
  • Houston, TX
  • London, UK
  • Munich, DE
  • Tokyo, JP

Built to support a real-world study launch.

This branded mock site demonstrates the complete structure needed for a modern Phase 3 patient website: narrative clarity, accessibility, responsive performance, and policy-ready footer components.

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