Safety and Efficacy Study of Intravenous Uricase-PEG 20

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01021241
Recruitment Status : Unknown
Verified February 2010 by EnzymeRx.
Recruitment status was:  Active, not recruiting
First Posted : November 26, 2009
Last Update Posted : February 23, 2010
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Brief Summary:
The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single intravenous doses of Uricase-PEG 20.

Condition or disease Intervention/treatment Phase
Gout Hyperuricemia Biological: Uricase-PEG 20 Phase 1

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Cohort Dose-Escalation Phase 1 Study of Intravenous Infusion of Uricase-PEG 20
Study Start Date : October 2009
Estimated Primary Completion Date : March 2010
Estimated Study Completion Date : March 2010

Resource links provided by the National Library of Medicine

Drug Information available for: Rasburicase

Arm Intervention/treatment
Experimental: Uricase-PEG 20
Cohorts will receive ascending doses of Uricase-PEG 20 in a sequential manner
Biological: Uricase-PEG 20
Intravenous infusion of Uricase-PEG 20 over one hour; no premedication

Primary Outcome Measures :
  1. 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 ]
  2. Pharmacokinetics (Uricase-PEG 20 serum concentration) [ Time Frame: Through Day 35 after dosing ]
  3. Pharmacodynamics (plasma uric acid concentration) [ Time Frame: Through Day 35 after dosing ]

Information from the National Library of Medicine

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Ages Eligible for Study:   40 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

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)

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 identifier (NCT number): NCT01021241

United States, Florida
MRA Clinical Research
Miami, Florida, United States, 33143
United States, Pennsylvania
Altoona Center for Clinical Research
Duncansville, Pennsylvania, United States, 16635
Sponsors and Collaborators
Study Director: Anthony S Fiorino, MD, PhD EnzymeRx, LLC
Principal Investigator: Alan Kivitz, MD Altoona Center for Clinical Research

Additional Information:
Responsible Party: Anthony Fiorino, MD, PhD, EnzymeRx, LLC Identifier: NCT01021241     History of Changes
Other Study ID Numbers: ENZ-101
First Posted: November 26, 2009    Key Record Dates
Last Update Posted: February 23, 2010
Last Verified: February 2010

Additional relevant MeSH terms:
Pathologic Processes
Gout Suppressants
Antirheumatic Agents