A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency
Research type
Research Study
Full title
A Phase 1/2 Open-label Study in Patients with Arginase I Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of Intravenous AEB1102
IRAS ID
236714
Contact name
Suresh Vijay (Vijayaraghavan), Dr
Contact email
Sponsor organisation
Aeglea BioTherapeutics, Inc.
Eudract number
2017-003851-45
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
127774, IND Number
Duration of Study in the UK
0 years, 8 months, 0 days
Research summary
The study sponsor, Aeglea BioTherapeutics, Inc., is developing AEB1102 (pegzilarginase) a recombinant human arginase enzyme to treat excess arginase levels in patients with Arginase 1 Deficiency, a urea cycle disorder caused by a mutation in the arginase 1 gene that results in the inability to degrade the amino acid arginine.
Pegzilarginase is an engineered human arginase 1 enzyme and is intended for use as enzyme replacement therapy to reduce the elevated blood arginine levels to the normal physiological range.
Arginase 1 Deficiency is a debilitating, progressive disease with significant morbidity and early mortality. This Phase 1/2, multicentre, single arm, open label trial is designed to evaluate the safety and tolerability of intravenous administration of pegzilarginase in patients with the deficiency. It will also evaluate the pharmacokinetic and pharmacodynamic effects of repeated doses of pegzilarginase on plasma arginine levels.
The study will enrol approximately 10 patients, adult and paediatric, with Arginase 1 Deficiency in the United States, Canada, and Europe.
It will be conducted in 2 parts: Part 1 (Single Ascending Dose Escalation) and Part 2 (Repeated Dosing). Each part will be preceded by a baseline assessment of arginine levels. All patients who participate in Part 1 may continue AEB1102 dosing in Part 2 if they qualify for continued dosing.REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
18/YH/0046
Date of REC Opinion
12 Mar 2018
REC opinion
Further Information Favourable Opinion