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

NCT02488044 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2019-06-12

No results posted yet for this study

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.

Conditions

  • Arginase I Deficiency
  • Hyperargininemia

Interventions

DRUG

AEB1102

modified human arginase I

Sponsors & Collaborators

  • Aeglea Biotherapeutics

    lead INDUSTRY

Principal Investigators

  • Josie Gayton · Aeglea Biotherapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-06-30
Primary Completion
2019-02-28
Completion
2019-02-28

Countries

  • United States
  • Canada
  • Portugal
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02488044 on ClinicalTrials.gov