Rare Genetic Diseases Resources

Patient Advocacy, Clinical Trials and Additional Information

At Aeglea we are dedicated to changing patients’ lives. Our credo is to put patients first. Successful research and drug development depends on patient advocacy and close collaboration with all members of a disease community including:

  • Patients and their families and/or caregivers
  • Physicians
  • Metabolic dietitians
  • Allied healthcare providers
  • Advocacy organizations
  • Research institutions

Currently, our patient advocacy programs focus on people affected by a rare, genetic urea cycle disorder called Arginase 1 Deficiency. The Aeglea patient advocacy team connects with patient communities and organizations to ensure their voices are heard within our company and to better understand the needs of people living with Arginase 1 Deficiency.

Current Clinical Trials

We are conducting a clinical trial to assess the safety and efficacy of our enzyme replacement therapy in patients with Arginase I Deficiency.

  • PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-blind, Placebo-controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults With Arginase 1 Deficiency – NCT03921541 – Recruiting
  • An Open-label, Multicentre Extension Study to Evaluate the Long-Term Safety, Tolerability and Effects of Intravenous Pegzilarginase in Patients With Arginase I Deficiency Who Previously Received Treatment in Study CAEB1102-101A – NCT03378531 – Active, not recruiting

Thank you to the patients, families and physicians who contribute to our collective knowledge and to the potential of future therapies by considering involvement in clinical trials.