Safety and Efficacy Study of Intravenous Uricase-PEG 20
Primary Purpose
Gout, Hyperuricemia
Status
Unknown status
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Uricase-PEG 20
Sponsored by
About this trial
This is an interventional treatment trial for Gout
Eligibility Criteria
Inclusion Criteria:
- Serum uric acid > 6 mg/dL (men) or > 5 mg/dL (women)
- Clinical laboratory values within normal limits or not clinically significant
- Women should be menopausal or peri-menopausal
Exclusion Criteria:
- Prior exposure to uricase
- History of severe allergic reactions, or any allergy to PEG or pegylated products
- G6PD or catalase deficiency
- Medical condition that may interfere with ability to complete the study (e.g., uncontrolled diabetes or hypertension, congestive heart failure NYHA Class III or IV, history of myocardial infarction, immunosuppression, pregnancy)
Sites / Locations
- MRA Clinical Research
- Altoona Center for Clinical Research
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Uricase-PEG 20
Arm Description
Cohorts will receive ascending doses of Uricase-PEG 20 in a sequential manner
Outcomes
Primary Outcome Measures
Safety (assessment of signs and symptoms and clinical laboratory measurements following administration of Uricase-PEG 20, graded according to the Common Toxicity Criteria for Rheumatology, version 2.0)
Pharmacokinetics (Uricase-PEG 20 serum concentration)
Pharmacodynamics (plasma uric acid concentration)
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01021241
Brief Title
Safety and Efficacy Study of Intravenous Uricase-PEG 20
Official Title
A Cohort Dose-Escalation Phase 1 Study of Intravenous Infusion of Uricase-PEG 20
Study Type
Interventional
2. Study Status
Record Verification Date
February 2010
Overall Recruitment Status
Unknown status
Study Start Date
October 2009 (undefined)
Primary Completion Date
March 2010 (Anticipated)
Study Completion Date
March 2010 (Anticipated)
3. Sponsor/Collaborators
Name of the Sponsor
EnzymeRx
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single intravenous doses of Uricase-PEG 20.
Detailed Description
Uricase is an enzyme (found in most mammals but not humans) that converts poorly soluble uric acid into highly soluble allantoin. Because humans lack uricase, they are prone to develop elevated levels of uric acid, which can form crystals in the joints and soft tissues. In those which chronically elevated uric acid, gout may develop. In the setting of acute rises in uric acid, seen for example in tumor lysis syndrome, uric acid crystals can damage the renal tubules. Uricase-PEG 20 is a recombinant uricase conjugated with multiple PEG molecules designed to prolong the half-life and decrease the immunogenicity of uricase. This study will characterize the safety, pharmacokinetics and pharmacodynamics of intravenous Uricase-PEG 20, the anticipated route of administration to be used in future clinical development in tumor lysis syndrome.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gout, Hyperuricemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Uricase-PEG 20
Arm Type
Experimental
Arm Description
Cohorts will receive ascending doses of Uricase-PEG 20 in a sequential manner
Intervention Type
Biological
Intervention Name(s)
Uricase-PEG 20
Intervention Description
Intravenous infusion of Uricase-PEG 20 over one hour; no premedication
Primary Outcome Measure Information:
Title
Safety (assessment of signs and symptoms and clinical laboratory measurements following administration of Uricase-PEG 20, graded according to the Common Toxicity Criteria for Rheumatology, version 2.0)
Time Frame
Through Day 35 after dosing
Title
Pharmacokinetics (Uricase-PEG 20 serum concentration)
Time Frame
Through Day 35 after dosing
Title
Pharmacodynamics (plasma uric acid concentration)
Time Frame
Through Day 35 after dosing
10. Eligibility
Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
Serum uric acid > 6 mg/dL (men) or > 5 mg/dL (women)
Clinical laboratory values within normal limits or not clinically significant
Women should be menopausal or peri-menopausal
Exclusion Criteria:
Prior exposure to uricase
History of severe allergic reactions, or any allergy to PEG or pegylated products
G6PD or catalase deficiency
Medical condition that may interfere with ability to complete the study (e.g., uncontrolled diabetes or hypertension, congestive heart failure NYHA Class III or IV, history of myocardial infarction, immunosuppression, pregnancy)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anthony S Fiorino, MD, PhD
Organizational Affiliation
EnzymeRx, LLC
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Alan Kivitz, MD
Organizational Affiliation
Altoona Center for Clinical Research
Official's Role
Principal Investigator
Facility Information:
Facility Name
MRA Clinical Research
City
Miami
State/Province
Florida
ZIP/Postal Code
33143
Country
United States
Facility Name
Altoona Center for Clinical Research
City
Duncansville
State/Province
Pennsylvania
ZIP/Postal Code
16635
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
12233890
Citation
Bomalaski JS, Holtsberg FW, Ensor CM, Clark MA. Uricase formulated with polyethylene glycol (uricase-PEG 20): biochemical rationale and preclinical studies. J Rheumatol. 2002 Sep;29(9):1942-9.
Results Reference
background
Links:
URL
http://www.enzymerx.com
Description
Homepage of sponsoring company
URL
http://ash.confex.com/ash/2009/webprogram/Paper21744.html
Description
Abstract describing animal pharmacokinetic studies
Learn more about this trial
Safety and Efficacy Study of Intravenous Uricase-PEG 20
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