Safety and Efficacy Study of Intramuscular Uricase-PEG 20

Phase 1

• Conditions: Hyperuricemia; Gout
• Interventions: Biological: Uricase-PEG 20

• Registration Number: NCT01038947
• Lead Sponsor: EnzymeRx

Brief Summary

The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single and multiple intramuscular 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 intramuscular Uricase-PEG 20, the anticipated route of administration to be used in future clinical development in gout.

Study Timeline

• Status Verified: 2009-12
• Study Start: 2009-12
• Study First Submitted: 2009-12-22
• Study First Submitted QC: 2009-12-22
• Last Update Submitted: 2009-12-22
• Study First Posted: 2009-12-24
• Last Update Posted: 2009-12-24
• Primary Completion: 2010-04
• Study Completion: 2010-05

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 28

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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Uricase-PEG 20	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.

Primary Outcome Measures

Name	Time	Method
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)	Up to 35 days after dosing

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics (Uricase-PEG 20 serum concentration)	Up to 35 days after dosing
Pharmacodynamics (plasma uric acid concentration)	Up to 35 days after dosing
Immunogenicity	Up to 35 days after dosing

Trial Locations

Locations (2)

MRA Clinical Research; 🇺🇸 Miami, Florida, United States

Altoona Center for Clinical Research; 🇺🇸 Duncansville, Pennsylvania, United States