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Trial record 1 of 1 for:    aeglea arginase deficiency protocol
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A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency

This study is currently recruiting participants.
See Contacts and Locations
Verified June 2017 by Aeglea Biotherapeutics
Sponsor:
Information provided by (Responsible Party):
Aeglea Biotherapeutics
ClinicalTrials.gov Identifier:
NCT02488044
First received: June 29, 2015
Last updated: June 23, 2017
Last verified: June 2017
  Purpose
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.

Condition Intervention Phase
Arginase I Deficiency Hyperargininemia Drug: AEB1102 Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Aeglea Biotherapeutics:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 10
Study Start Date: June 2016
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: March 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AEB1102

AEB1102, modified human Arginase I administered IV Part 1 Each patient may receive up to 7 doses given up to every other week over a maximum of 14 weeks.

Part 2 Each patient will receive up to 8 weeks of repeat-dose therapy.

Drug: AEB1102
modified human arginase I
Other Name: Co-ArgI-PEG

  Eligibility

Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Documented diagnosis of Arginase I deficiency
  • Adequate organ function: Hgb ≥ 10 g/dL, ANC ≥ 1.5 x 109/L, plt count ≥ 100,000/µL; liver transaminase levels ≤ 2.5x ULN, total bilirubin ≤ 2.0 mg/dL; serum creatinine <1.5 x ULN
  • If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
  • If a sexually active (male or female), must be surgically sterile, post-menopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration
  • Patient or legal guardian is able and willing to provide written informed consent and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification) prior to any screening procedures

Exclusion Criteria:

  • Transfusion of ≥ 2 u RBC within 60 days
  • Active infection requiring systemic treatment
  • Known infection with HIV, Hep B or Hep C
  • Severe hyperammonemia requiring hospitalization within 14 days. Had more than one episode of hyperammonemia requiring hospitalization within the 30 days prior to enrollment.
  • Current uncontrolled hyperammonemia
  • Has a history of hypersensitivity to PEG or any other component of the AEB1102 (Co-ArgI-PEG) formulation
  • If female, is lactating or breast feeding

PART 2 INCLUSION CRITERION:

1. Did not experience any safety or tolerability event in Part 1 which would preclude continued participation and dosing of AEB1102

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02488044

Contacts
Contact: Shannon Lewis Berlin 1-855-509-9921 clinical@aegleabio.com

Locations
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States
Contact: Roberto Zori, MD         
Principal Investigator: Roberto Zori, MD         
United States, New York
Icahn School of Medicine at Mount Sinai Recruiting
New York, New York, United States
Contact: George Diaz, MD         
Principal Investigator: George Diaz, MD         
Sponsors and Collaborators
Aeglea Biotherapeutics
Investigators
Study Director: Kiran Patki, MD Aeglea Biotherapeutics, Inc.
  More Information

Publications:
Responsible Party: Aeglea Biotherapeutics
ClinicalTrials.gov Identifier: NCT02488044     History of Changes
Other Study ID Numbers: CAEB1102-101A
Study First Received: June 29, 2015
Last Updated: June 23, 2017

Additional relevant MeSH terms:
Hyperargininemia
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on September 19, 2017