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

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ClinicalTrials.gov Identifier: NCT02488044
Recruitment Status : Recruiting
First Posted : July 2, 2015
Last Update Posted : April 6, 2018
Sponsor:
Information provided by (Responsible Party):
Aeglea Biotherapeutics

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.

Condition or disease Intervention/treatment Phase
Arginase I Deficiency Hyperargininemia Drug: AEB1102 Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
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
Study Start Date : June 2016
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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 :
  1. Number of subjects with a decrease from baseline in plasma arginine level [ Time Frame: Baseline to 2, 4, 6, 8 weeks ]
  2. 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 ]
  3. Number of subjects with a decrease from baseline in plasma guanidino compound levels [ Time Frame: Baseline to 2, 4, 6, 8 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02488044


Contacts
Contact: Aeglea Clinical Department 1.855.509.9921 raredisease@aegleabio.com

Locations
United States, California
Stanford University School of Medicine Recruiting
Stanford, California, United States, 94305
Contact: Gregory Enns, MD         
Principal Investigator: Gregory Enns, MD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32611
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, 10029
Contact: George Diaz, MD         
Principal Investigator: George Diaz, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Gerard Vockley, MD         
Principal Investigator: Gerard Vockley, MD         
United States, Texas
UTSW Recruiting
Dallas, Texas, United States, 75390
Contact: Markey McNutt, MD         
Principal Investigator: Markey McNutt, MD         
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84112
Contact: Nicola Longo, MD         
Principal Investigator: Nicola Longo, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Lawrence Merritt, MD         
Principal Investigator: Lawrence Merritt, MD         
Canada, Ontario
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Andreas Schultze, MD         
Principal Investigator: Andreas Schultze, MD         
Sponsors and Collaborators
Aeglea Biotherapeutics
Investigators
Study Director: Jim Joffrion Aeglea Biotherapeutics, Inc.

Publications:
Responsible Party: Aeglea Biotherapeutics
ClinicalTrials.gov Identifier: NCT02488044     History of Changes
Other Study ID Numbers: CAEB1102-101A
First Posted: July 2, 2015    Key Record Dates
Last Update Posted: April 6, 2018
Last Verified: April 2018

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