Clinical Trial: A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 1/2 Open-label Study in Patients With Arginase I Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of Intravenous AEB1102

Brief Summary: A Phase 1/2 Open-label Study in Patients with Arginase I Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of Intravenous AEB1102. This study is designed to evaluate the safety and tolerability of IV administration of AEB1102 for the treatment of pediatric and adult patients with Arginase I deficiency and hyperargininemia. This study 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. A data safety monitoring board (DSMB) will provide independent review of study safety data and recommend whether the sponsor should continue the study as planned, modify the study protocol, or discontinue the study.

Detailed Summary:
Sponsor: Aeglea Biotherapeutics

Current Primary Outcome: Number of subjects with adverse events [ Time Frame: weekly throughout the study, up to 14 weeks ]

Includes significant changes in hematology, chemistry and coagulation laboratory studies as well as in physical exam and vital signs


Original Primary Outcome: Number of subjects with adverse events [ Time Frame: weekly throughout the study, up to 24 weeks ]

Includes significant changes in hematology, chemistry and coagulation laboratory studies as well as in physical exam and vital signs


Current Secondary Outcome:

  • Number of subjects with a decrease from baseline in plasma arginine level [ Time Frame: Baseline to 2, 4, 6, 8 weeks ]
  • Pharmacokinetic profile including Cmax, AUC, Tmax, T1/2 for each subject [ Time Frame: At 15 min, 1, 2, 4, 8, 12, 24, 48, 72, and 120 hours following dose escalation ]


Original Secondary Outcome:

  • Number of subjects with a decrease from baseline in plasma arginine level [ Time Frame: Baseline to 2, 4, 6, 8, 12, 14 weeks ]
  • Pharmacokinetic profile including Cmax, AUC, Tmax, T1/2 for each subject [ Time Frame: At 15 min, 1, 2, 4, 8, 12, 24, 48, 72, and 120 hours following dose escalation ]


Information By: Aeglea Biotherapeutics

Dates:
Date Received: June 29, 2015
Date Started: June 2016
Date Completion: June 2018
Last Updated: February 7, 2017
Last Verified: February 2017