Safety and Efficacy Study of Intramuscular 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: NCT01038947
Recruitment Status : Unknown
Verified December 2009 by EnzymeRx.
Recruitment status was:  Recruiting
First Posted : December 24, 2009
Last Update Posted : December 24, 2009
Information provided by:

December 22, 2009
December 24, 2009
December 24, 2009
December 2009
April 2010   (Final data collection date for primary outcome measure)
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: Up to 35 days after dosing ]
Same as current
No Changes Posted
  • Pharmacokinetics (Uricase-PEG 20 serum concentration) [ Time Frame: Up to 35 days after dosing ]
  • Pharmacodynamics (plasma uric acid concentration) [ Time Frame: Up to 35 days after dosing ]
  • Immunogenicity [ Time Frame: Up to 35 days after dosing ]
Same as current
Not Provided
Not Provided
Safety and Efficacy Study of Intramuscular Uricase-PEG 20
A Cohort Dose-Escalation Phase 1 Study of Intramuscular Injection of Uricase-PEG 20
The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single and multiple intramuscular doses of Uricase-PEG 20
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 intramuscular Uricase-PEG 20, the anticipated route of administration to be used in future clinical development in gout.
Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Gout
  • Hyperuricemia
Biological: Uricase-PEG 20
Intramuscular injection of Uricase-PEG 20 without premedication
Experimental: Uricase-PEG 20
Cohorts will receive ascending doses of Uricase-PEG 20 in a sequential manner. The study will enroll both single dose cohorts and multi-dose cohorts.
Intervention: Biological: Uricase-PEG 20
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.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Unknown status
Same as current
May 2010
April 2010   (Final data collection date for primary outcome measure)

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)
Sexes Eligible for Study: All
40 Years to 75 Years   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Anthony Fiorino, MD, PhD, EnzymeRx, LLC
Not Provided
Study Director: Anthony S Fiorino, MD, PhD EnzymeRx, LLC
Principal Investigator: Patricia Pardo, MD MRA Clinical Research
December 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP