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A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency
Sponsor: Immedica Pharma AB
Summary
The purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who complete Study CAEB1102-101A.
Official title: An Open-label, Multicentre Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of AEB1102 in Patients With Arginase 1 Deficiency
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
INTERVENTIONAL
Enrollment
14
Start Date
2017-12-07
Completion Date
2022-12-15
Last Updated
2026-09-24
Healthy Volunteers
No
Conditions
Interventions
AEB1102
modified human arginase I
Locations (7)
Stanford University School of Medicine
Stanford, California, United States
University of Florida
Gainsville, Florida, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
UTSW
Dallas, Texas, United States
The Hospital for Sick Children
Toronto, Ontario, Canada
Centro Hospitalar S. Joao
Porto, Portugal
Great Ormond Street Hospital
London, United Kingdom