Study Stopped
DSMB suggested closure due to low enrollment
Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders
2 other identifiers
observational
10
2 countries
11
Brief Summary
The primary purpose of the proposed study is to characterize the oxidative stress and inflammatory cytokine status in UCD during baseline and decompensated states.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Feb 2012
Typical duration for all trials
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2012
CompletedFirst Submitted
Initial submission to the registry
February 8, 2012
CompletedFirst Posted
Study publicly available on registry
March 1, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2015
CompletedOctober 2, 2015
October 1, 2015
2.9 years
February 8, 2012
October 1, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Laboratory values indicating oxidative stress
Laboratory values that indicate oxidative stress include IL-1, IL-2, IL-6, and IL-8. These values will be analyzed as a panel (not individually) comparing baseline values to values during periods of decompensation.
Change from baseline to period of decompensation up to one year
Eligibility Criteria
Urea cycle disorders Inherited metabolic disorders N-acetylglutamate synthase (NAGS) deficiency Carbamyl phosphate synthetase I (CPSI) deficiency Ornithine transcarbamylase (OTC) deficiency Argininosuccinate synthetase (AS) deficiency (Citrullinemia) Argininosuccinate lyase (AL) deficiency (argininosuccinic aciduria Arginase (ARG) deficiency (hyperargininemia) Hyperornithinemia, hyperammonemia and homocitrullinuria (HHH) syndrome, or mitochondrial ornithine carrier (ORNT) deficiency Citrullinemia type II, mitochondrial aspartate/glutamate carrier (CITR) deficiency
You may qualify if:
- Enrolled in Longitudinal Study of Urea Cycle Disorders (RDCRN UCDC #5101)
You may not qualify if:
- UCD patients who have undergone orthotopic liver transplantation
- Significant chronic medical co-morbidity that might confound the analysis as determined by the site investigators.
- Significant co-morbidities include but are not limited to:
- diabetes, liver failure + cirrhosis
- renal failure
- cardiac disease
- chronic inflammatory diseases
- asthma requiring daily long-term control medications
- significant respiratory disease.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
University of California, Los Angeles
Los Angeles, California, 90095, United States
The Children's Hospital, Aurora
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Mount Sinai School of Medicine
New York, New York, 10029, United States
Case Western Medical College
Cleveland, Ohio, 44106, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Children's Hospital and Regional Medical Center
Seattle, Washington, 98105, United States
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
George Diaz
Icahn School of Medicine at Mount Sinai
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- MD
Study Record Dates
First Submitted
February 8, 2012
First Posted
March 1, 2012
Study Start
February 1, 2012
Primary Completion
January 1, 2015
Study Completion
July 1, 2015
Last Updated
October 2, 2015
Record last verified: 2015-10