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.

For more information:

Find resources | Learn about clinical trials | Share your experiences

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.

A Phase 1/2 Study of AEB1102 in Patients with Arginase 1 Deficiency

Drug: AEB1102(Pegzilarginase) – Co-ArgI-PEG modified human Arginase I
Phase: Phase 1, Phase 2
Study Type: Interventional
Endpoint Classification: Safety Study/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Indication: Arginase 1 Deficiency, Hyperargininemia
Status: Active
Additional Information: https://clinicaltrials.gov/ct2/show/NCT02488044?term=aeglea&rank=3

At present, Aeglea Biotherapeutics is not providing experimental therapeutic agents outside of the clinical trial setting. As part of our dedication to changing patients’ lives, we are currently developing a new policy that will address Aeglea’s process to determining patient access to investigational therapeutic agents for compassionate use.